Medicamentos biologicos en glomerulonefritis

Medicamentos biologicos en glomerulonefrtiis

1. ClinicalTrials.gov Search Results 06/12/2023
Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
1 A Randomized, Controlled Clinical Study
of Rituximab in Treatment of Primary IgA
Nephropathy
Active, not
recruiting
No Results
Available
•IgA Nephropathy •Drug: rituximab group Phase:
Phase 4
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Single (Participant)
•Primary Purpose: Treatment
Outcome Measures:
•Changes in proteinuria levels
over 1 year compared with
baseline
•The proportion of 50% reduction
in mean urinary protein
compared with baseline over 1
year
•The proportion of 50% reduction
in mean urinary protein
compared with baseline over 6
months
•Changes in proteinuria levels
over 6 months compared with
baseline
•Changes in eGFR levels over 1
year compared with baseline
•Changes of Gd-IgA1 levels
•Incidence of adverse events
•Incidence of ESRD
•Proportion of eGFR decreased
by 50 % or serum creatine
doubled compared with baseline
•Incidence rate of infection
Enrollment:
116
Age:
18 Years to 75 Years
(Adult, Older Adult)
Study Start:
June 20, 2020
Last Update Posted:
April 21, 2023
•Ruijin Hospital, Shanghai Jiao Tong
University School of Medicine,
Shanghai, Shanghai, China
- Page 1 of 33 -

2. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
2 An Open-Label, Nonrandomized,
Multicenter Extension Study to Evaluate
the Long-term Safety and Efficacy
of Pegcetacoplan in Participants
With C3 Glomerulopathy or Immune-
Complex Membranoproliferative
Glomerulonephritis
Not yet
recruiting
No Results
Available
•C3G
•IC-MPGN
•C3 Glomerulopathy
•C3 Glomerulonephritis
•Complement 3
Glomerulopathy
•Complement 3
Glomerulopathy (C3G)
•Complement 3
Glomerulonephritis
•Dense Deposit Disease
•DDD
•Membranoproliferative
Glomerulonephritis
•Membranoproliferative
Glomerulonephritis (MPGN)
•Immune Complex
Membranoproliferative
Glomerulonephritis (IC-
MPGN)
•Drug: Pegcetacoplan Phase:
Phase 3
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
Proportion of participants with
a reduction in urine protein-to-
creatinine ratio (uPCR) of at least
50% from the pretreatment value
over time.
Enrollment:
100
Age:
12 Years and older (Child,
Adult, Older Adult)
Study Start:
May 2023
Last Update Posted:
April 14, 2023
3 A Study of ANX009 in Adult Participants
With Lupus Nephritis
Recruiting No Results
Available
•Lupus Nephritis •Biological: ANX009 Phase:
Phase 1
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Number of Participants With
Treatment-Emergent Adverse
Events (TEAEs)
•Change From Baseline in Free
Complement Component 1q
(C1q) Concentrations in Serum
Over Time
•Change From Baseline in
Complement Factor C4
Concentration and its Activation
Product (Complement
Component 4d [C4d]) in Plasma
Over Time
Enrollment:
7
Age:
18 Years to 75 Years
(Adult, Older Adult)
Study Start:
August 9, 2022
Last Update Posted:
March 22, 2023
•Annexon Investigational Site 203,
Angeles City, Philippines
•Annexon Investigational Site 204, Iloilo
City, Philippines
•Annexon Investigational Site 201,
Manila, Philippines
•Annexon Investigational Site 202,
Quezon city, Philippines
•Annexon Investigational Site 101,
Taichung City, Taiwan
•Annexon Investigational Site 102,
Taipei, Taiwan
•Annexon Investigational Site 103,
Taoyuan County, Taiwan
- Page 2 of 33 -

3. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
4 Study of Efficacy and Safety of Iptacopan
in Participants With IC-MPGN
Not yet
recruiting
No Results
Available
•IC-MPGN •Drug: Placebo
•Drug: iptacopan
Phase:
Phase 3
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Triple (Participant,
Care Provider, Investigator)
•Primary Purpose: Treatment
Outcome Measures:
•Log-transformed ratio to baseline
in UPCR (sampled from a
24-hour urine collection) at 6
months.
•Log-transformed ratio to baseline
in UPCR at the 12-month visit
(both study treatment arms)
•Log-transformed ratio to 6-month
visit in UPCR at the 12-month
visit in the placebo arm.
•Change from baseline in eGFR
•Change in eGFR from the 6-
month visit to the 12- month visit
of the placebo arm
•Proportion of patients achieved a
composite renal endpoint
•Proportion of patients achieving
a composite renal endpoint from
the 6-month visit to the 12-month
visit of the placebo arm
•Change from baseline in the
Functional Assessment of
Chronic Illness Therapy-Fatigue
(FACIT-Fatigue) score.
•Change in the FACIT-Fatigue
score from the 6-month visit to
the 12-month visit at the placebo
arm
•Number of participants with
abnormal vital signs, ECGs and
safety laboratory measurements
•Number of participants with
study drug discontinuation due to
an AE
•Number of participants with
clinically significant changes
in heart rate, blood pressure,
echocardiography parameters
and NT-proBPN in adolescent
patients
Enrollment:
68
Age:
12 Years to 60 Years
(Child, Adult)
Study Start:
July 31, 2023
Last Update Posted:
March 23, 2023
- Page 3 of 33 -

4. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
5 Study of NM8074 in Adult C3
Glomerulopathy Patients
Not yet
recruiting
No Results
Available
•C3 Glomerulopathy •Drug: NM8074 Phase:
•Phase 1
•Phase 2
Study Design:
•Allocation: Non-Randomized
•Intervention Model: Sequential
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Monitoring for incidence of
Adverse Events (AEs)/Serious
Adverse Events (SAEs)
•Change from Baseline or
Percent Change from Baseline
in Urine Protein to Creatine
Concentration Ratio (UPCR)
•Change from Baseline or
Percent Change from Baseline
in Urine Albumin to Creatinine
Concentration Ratio (UACR)
•Ratio to Baseline of UPCR and
UACR
•Change from Baseline or
Percent Change from Baseline in
Estimated Glomerular Filtration
Rate (eGFR)
•Percent Change from Baseline
in Alternative Pathway (AP)
of Complement Activity as
Compared to Percent Change
from Baseline in Classical
Pathway (CP) of Complement
Activity as Measured by Percent
Change in Levels of Membrane
Attack Complex (MAC)
•Percent Change from Baseline
in Alternative Pathway (AP)
of Complement Activity as
Compared to Percent Change
from Baseline in Classical
Pathway (CP) of Complement
Activity as Measured by
Percent Change in Levels of
Complement Component C3b
•Change from Baseline or
Percent Change from Baseline in
Serum C3 Levels
•Change from Baseline or
Percent Change from Baseline in
Glomerular Inflammation
•Change from Baseline or
Percent Change from Baseline
in Quality of Life (QoL) Assessed
via the Functional Assessment of
Chronic Illness Therapy (FACIT)-
Fatigue Scale, Version 4.
•and 7 more
Enrollment:
18
Age:
18 Years to 65 Years
(Adult, Older Adult)
Study Start:
September 2023
Last Update Posted:
May 12, 2023
- Page 4 of 33 -

5. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
6 Efficacy, Safety, Pharmacokinetics, and
Pharmacodynamics of KP104 to Treat
Glomerulonephritis
Not yet
recruiting
No Results
Available
•Glomerulonephritis •Drug: KP104 Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Sequential
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Percent change from Baseline in
24-hour urinary protein creatinine
ratio (UPCR) at Week 24 (C3G)
for weekly maintenance dosing
•Percent change from Baseline
in 24-hour UPCR at Week 48
(IgAN) for weekly maintenance
dosing
•Percent change from Baseline
in 24-hour UPCR at Week 23
(C3G) for biweekly maintenance
dosing
•Percent change from Baseline
in 24-hour UPCR at Week 47
(IgAN) for biweekly maintenance
dosing
•Number of participants reporting
Treatment-Emergent Adverse
Events (TEAEs)
•Number of participants reporting
Treatment-Emergent Serious
Adverse Events (TESAEs)
•Number of participants reporting
AEs of Special Interest (AESIs)
•Change from Baseline in Rabbit
red blood cell (RBC) assay
•Change from Baseline in
complement component of C3b
activity assay
•Change from baseline in free
serum C5 levels
•and 4 more
Enrollment:
52
Age:
18 Years to 75 Years
(Adult, Older Adult)
Study Start:
July 1, 2023
Last Update Posted:
March 1, 2023
- Page 5 of 33 -

6. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
7 Safety and Pharmacokinetic Study of
Subcutaneous ALXN1720 in Participants
With Proteinuria
Active, not
recruiting
No Results
Available
•Proteinuria •Drug: ALXN1720 Phase:
Phase 1
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Serum Concentration of
ALXN1720
•Number of Participants With
Treatment-Emergent Adverse
Events
•Serum Concentration of
Free and Total Complement
Component 5 (C5)
•Number of Participants With
Antidrug Antibodies (ADAs) to
ALXN1720
Enrollment:
12
Age:
19 Years to 65 Years
(Adult, Older Adult)
Study Start:
June 29, 2022
Last Update Posted:
June 8, 2023
•Clinical Trial Site 2, Anyang-Si,
Gyeonggi-do, Korea, Republic of
•Clinical Trial Site 1, Seoul, Korea,
Republic of
- Page 6 of 33 -

7. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
8 BCX9930 for the Treatment of C3G,
IgAN, and PMN (RENEW)
Terminated No Results
Available
•Complement 3
Glomerulopathy
•Immunoglobulin A
Nephropathy
•Membranous Nephropathy
•Drug: BCX9930 Phase:
Phase 2
Study Design:
•Allocation: Non-Randomized
•Intervention Model: Parallel
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Percent change from baseline
in 24-hour urine protein-to-
creatinine ratio (uPCR)
•Proportion of subjects with
a uPCR response: # 50%
reduction from baseline, # 500
mg/g, or # 200 mg/g
•Percent change from baseline in
24-hour urinary protein excretion
•Change from baseline in
estimated glomerular filtration
rate
•Change from baseline in serum
albumin
•Proportion of subjects with
protein # 3.5 g in a 24-hour urine
collection and serum albumin #
2.5 g/dL
•Proportion of subjects with a
morphologic response
•Treatment-emergent adverse
event and serious adverse event
Enrollment:
2
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
October 29, 2021
Last Update Posted:
January 23, 2023
•Investigative Site, Poitiers, France
•Investigative Site, Toulouse, France
•Investigative Site, Bari, Italy
•Investigative Site, Bergamo, Italy
•Investigative Site, Brescia, Italy
•Investigative Site, Turin, Italy
•Investigative Site #1, Barcelona, Spain
•Investigative Site #2, Barcelona, Spain
•Investigative Site #1, Madrid, Spain
•Investigative Site #2, Madrid, Spain
•Investigative Site, Oxford, United
Kingdom
- Page 7 of 33 -

8. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
9 Study of ALXN2050 in Proliferative Lupus
Nephritis (LN) and Immunoglobulin A
Nephropathy (IgAN)
Recruiting No Results
Available
•Lupus Nephritis
•Immunoglobulin A
Nephropathy
•IgAN
•LN
•Drug: ALXN2050
•Drug: Placebo
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Quadruple (Participant,
Care Provider, Investigator,
Outcomes Assessor)
•Primary Purpose: Treatment
Outcome Measures:
•Both Cohorts: Percentage
Change In Proteinuria From
Baseline To Week 26
•Both Cohorts: Percentage
Change In Proteinuria From
Baseline To Week 50
•Both Cohorts: Participants
Achieving > 30% And > 50%
Reduction In Proteinuria
At Week 26 And Week 50
Compared To Baseline
•Both Cohorts: Change
From Baseline In Estimated
Glomerular Filtration Rate
(eGFR) At Week 26 And Week
50
•LN Cohort: Participants Meeting
The Criteria For Complete Renal
Response At Week 26 And
Week 50
•LN Cohort: Participants Meeting
The Criteria For Partial Renal
Response At Week 26 And
Week 50
•LN Cohort: Time To The First
Occurrence Of Urine Protein To
Creatinine Ratio (UPCR) # 0.5
Gram/Gram (g/g) As Measured
By Spot Urine Sample
•LN Cohort: Participants
Achieving Corticosteroid Taper
To 7.5 mg/Day At Weeks 12, 26,
And 50
•LN Cohort: Participants
Experiencing A Renal Flare
Through Week 50
•LN Cohort: Participants
Experiencing An Extrarenal
Systemic Lupus Erythematosus
(SLE) Flare Through Week 50
•and 6 more
Enrollment:
126
Age:
18 Years to 75 Years
(Adult, Older Adult)
Study Start:
January 14, 2022
Last Update Posted:
June 2, 2023
•Clinical Trial Site, Huntsville, Alabama,
United States
•Clinical Trial Site, Fairfield, California,
United States
•Clinical Trial Site, Northridge, California,
United States
•Clinical Trial Site, Northridge, California,
United States
•Clinical Trial Site, Vacaville, California,
United States
•Clinical Trial Site, Gainesville, Florida,
United States
•Clinical Trial Site, Des Moines, Iowa,
United States
•Clinical Trial Site, Kansas City, Missouri,
United States
•Clinical Trial Ssite, Albuquerque, New
Mexico, United States
•Clinical Trial Site, New York, New York,
United States
•and 50 more
- Page 8 of 33 -

9. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
10 Study of ARO-C3 in Adult Healthy
Volunteers and Patients With
Complement Mediated Renal Disease
Recruiting No Results
Available
•C3 Glomerulopathy
•IgA Nephropathy
•Drug: ARO-C3
•Drug: Placebo
Phase:
Phase 1
Study Design:
•Allocation: Randomized
•Intervention Model: Sequential
Assignment
•Masking: Quadruple (Participant,
Care Provider, Investigator,
Outcomes Assessor)
•Primary Purpose: Treatment
Outcome Measures:
•Number of Participants with
Adverse Events (AEs) and/or
Serious Adverse Events (SAEs)
at Day 169
•Pharmacokinetics (PK) of ARO-
C3: Maximum Observed Plasma
Concentration (Cmax)
•PK of ARO-C3: Area under
the Plasma Concentration
Versus Time Curve from Zero to
24Hours (AUC0-24)
•PK of ARO-C3: Area Under
the Plasma Versus Time
Concentration Curve from Zero
to the Last Quantifiable Plasma
Concentration (AUClast)
•PK of ARO-C3: Area Under
the Plasma Concentration
Versus Time Curve from Zero
Extrapolated to Infinity (AUCinf)
PK of ARO-C3:
•PK of ARO-C3: Terminal
Elimination Half-Life (t1/2)
•PK of ARO-C3: Apparent Total
Body Clearance of ARO-C3 from
Plasma (CL)
•PK of ARO-C3: Volume of
Distribution (Vz/F)
•Pharmacodynamics (PD):
Change From Baseline in
Complement 3 (C3) up to Day
169
•PD: Percent Change From
Baseline in C3 up to Day 169
Enrollment:
60
Age:
18 Years to 70 Years
(Adult, Older Adult)
Study Start:
February 2, 2022
Last Update Posted:
April 10, 2023
•Research Site 1, Camperdown, New
South Wales, Australia
•Research Site 2, Clayton, Victoria,
Australia
•Research Site 2, Busan, Korea,
Republic of
•Research Site 1, Busan, Korea,
Republic of
•Research Site 3, Daegu, Korea,
Republic of
•Research Site 4, Goyang, Korea,
Republic of
•Research Site 5, Soeul, Korea, Republic
of
•Research Site, Auckland, New Zealand
•Research Site 1, Bangkok, Thailand
•Research Site 2, Bangkok, Thailand
•Research Site 3, Chiang Mai, Thailand
- Page 9 of 33 -

10. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
11 Phase III Study Assessing the Efficacy
and Safety of Pegcetacoplan in Patients
With C3 Glomerulopathy or Immune-
Complex Membranoproliferative
Glomerulonephritis
Recruiting No Results
Available
•C3G
•IC-MPGN
•C3 Glomerulopathy
•C3 Glomerulonephritis
•Complement 3
Glomerulopathy
•Complement 3
Glomerulopathy (C3G)
•Complement 3
Glomerulonephritis
•Dense Deposit Disease
•DDD
•Membranoproliferative
Glomerulonephritis
•Membranoproliferative
Glomerulonephritis (MPGN)
•Immune Complex
Membranoproliferative
Glomerulonephritis (IC-
MPGN)
•Drug: Pegcetacoplan
•Other: Placebo
Phase:
Phase 3
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Triple (Participant,
Care Provider, Investigator)
•Primary Purpose: Treatment
Outcome Measures:
•The proportion of subjects with a
reduction from baseline in urine
protein-to-creatinine ratio (uPCR)
of at least 50% at Week 26
•The proportion of subjects with
eGFR values that are stable or
improved
•For subjects with evaluable renal
biopsies, the change in the C3G
histologic index activity score
(adults only)
•The proportion of subjects with
evaluable renal biopsies showing
decreases in C3c staining on
renal biopsy (adults only)
Enrollment:
90
Age:
12 Years and older (Child,
Adult, Older Adult)
Study Start:
November 12, 2021
Last Update Posted:
March 30, 2023
•University of Alabama at Birmingham,
Birmingham, Alabama, United States
•Academic Medical Research Institute,
Los Angeles, California, United States
•Keck School of Medicine, University
of Southern California, Los Angeles,
California, United States
•UCI Center for Clinical Research,
Orange, California, United States
•Children's Hospital Colorado, Aurora,
Colorado, United States
•University of Florida, Gainesville,
Florida, United States
•Emory University School of Medicine,
Atlanta, Georgia, United States
•The University of Iowa, Iowa City, Iowa,
United States
•Renal and Transplant Associates
of New England, PC, Springfield,
Massachusetts, United States
•University of Michigan Medical Center,
Ann Arbor, Michigan, United States
•and 68 more
- Page 10 of 33 -

11. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
12 Study of Efficacy and Safety of Iptacopan
in Patients With C3 Glomerulopathy.
Recruiting No Results
Available
•C3G •Drug: Placebo
•Drug: iptacopan
Phase:
Phase 3
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Triple (Participant,
Care Provider, Investigator)
•Primary Purpose: Treatment
Outcome Measures:
•Adult cohort: Log-transformed
ratio to baseline in UPCR
(sampled from a 24-hour urine
collection)
•Adolescent cohort: Log-
transformed ratio to baseline in
UPCR (sampled from a 24-hour
urine collection)
•Change from baseline in log-
transformed UPCR at the 12-
month visit (both study treatment
arms).
•Change in log-transformed
UPCR from the 6-month visit to
the 12-month visit in the placebo
arm
•Change from baseline in eGFR.
•Proportion of participants who
meet the criteria for achieving a
composite renal endpoint
•Adult cohort: Change from
baseline in disease total activity
score in a renal biopsy.
•Change from baseline in the
Functional Assessment of
Chronic Illness Therapy-Fatigue
(FACIT-Fatigue) score.
•Number of participants with
abnormal clinically significant
vital signs, ECGs and safety
laboratory measurements
•Number of participants with
study drug discontinuation due to
an AE
•and 5 more
Enrollment:
83
Age:
12 Years to 60 Years
(Child, Adult)
Study Start:
July 28, 2021
Last Update Posted:
May 30, 2023
•Novartis Investigative Site, Aurora,
Colorado, United States
•Novartis Investigative Site,
Lawrenceville, Georgia, United States
•Novartis Investigative Site, Iowa City,
Iowa, United States
•Novartis Investigative Site, Baltimore,
Maryland, United States
•Novartis Investigative Site, Boston,
Massachusetts, United States
•Novartis Investigative Site, Albany, New
York, United States
•Novartis Investigative Site, New York,
New York, United States
•Novartis Investigative Site, Temple,
Texas, United States
•Novartis Investigative Site, Caba,
Buenos Aires, Argentina
•Novartis Investigative Site, Buenos
Aires, Argentina
•and 57 more
- Page 11 of 33 -

12. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
13 Study Assessing the Safety and Efficacy
of Pegcetacoplan in Post-Transplant
Recurrence of C3G or IC-MPGN
Recruiting No Results
Available
•C3G
•IC-MPGN
•Renal Transplant
•Complement 3
Glomerulopathy
•Complement 3
Glomerulopathy (C3G)
•Dense Deposit Disease
(DDD)
•Membranoproliferative
Glomerulonephritis
•Membranoproliferative
Glomerulonephritis (MPGN)
•Immune Complex
Membranoproliferative
Glomerulonephritis (IC-
MPGN)
•C3 Glomerulopathy
•C3 Glomerulonephritis
•Complement 3
Glomerulonephritis
•Drug: Pegcetacoplan Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•The primary efficacy endpoint is
the proportion of subjects with
reduction in C3c staining on
renal biopsy after 12 weeks of
treatment with pegcetacoplan.
•The proportion of subjects with
reduction in C3c staining on
renal biopsy
•The proportion of subjects with
stabilization or improvement in
estimated glomerular filtration
rate (eGFR)
•The proportion of subjects with
stabilization or improvement of
serum creatinine concentration
Enrollment:
12
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
February 23, 2021
Last Update Posted:
April 13, 2022
•Keck School of Medicine, University
of Southern California, Los Angeles,
California, United States
•Children's Hospital Colorado, Aurora,
Colorado, United States
•Washington University, St.Louis, Saint
Louis, Missouri, United States
•NYU Langone Health Transplant
Insitute, New York, New York, United
States
•CUIMC, New York, New York, United
States
•Hospital de Alta Complejidad en Red
El Cruce Dr. Nestor Carlos Kirchner,
Florencio Varela, Provincia De Buenos
Aires, Argentina
•Hospital Universitario Fundacion
Favaloro, Buenos Aires, Argentina
•Monash Medical Centre, Clayton,
Australia
•Medical University of Vienna, Vienna,
Austria
•Irmandade da Santa Casa de
Misericordia de Porto Alegre, Porto
Alegre, RS, Brazil
•and 12 more
- Page 12 of 33 -

13. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
14 Study of Ravulizumab in Proliferative
Lupus Nephritis (LN) or Immunoglobulin
A Nephropathy (IgAN)
Recruiting No Results
Available
•Lupus Nephritis
•Immunoglobulin A
Nephropathy
•Drug: Ravulizumab
•Drug: Placebo
•Other: Background
Therapy
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Triple (Participant,
Investigator, Outcomes
Assessor)
•Primary Purpose: Treatment
Outcome Measures:
•Both Cohorts: Percentage
Change In Proteinuria From
Baseline To Week 26 Assessed
Using 24-hour Urine Collections
•Both Cohorts: Percentage
Change In Proteinuria From
Baseline To Week 50 Assessed
Using 24-hour Urine Collections
•Both Cohorts: Change In
Estimated Glomerular Filtration
Rate (eGFR) From Baseline At
Week 26 And Week 50
•LN Cohort: Percentage Of
Participants Meeting The Criteria
For Complete Renal Response
•LN Cohort: Percentage Of
Participants Meeting The Criteria
For Partial Renal Response
•LN Cohort: Time To Urine
Protein To Creatinine Ratio < 0.5
g/g
•LN Cohort: Percentage
Of Participants Achieving
Corticosteroid Taper To 7.5 mg/
day
•LN Cohort: Percentage Of
Participants With Renal Flare
•LN Cohort: Percentage Of
Participants With Extrarenal
Systemic Lupus Erythematosus
Flare
•IgAN Cohort: Percentage Of
Participants Meeting The Criteria
For Partial Remission
Enrollment:
120
Age:
18 Years to 75 Years
(Adult, Older Adult)
Study Start:
December 11, 2020
Last Update Posted:
March 23, 2023
•Clinical Study Site, Los Angeles,
California, United States
•Clinical Study Site, S. Gate, California,
United States
•Clinical Study Site, San Dimas,
California, United States
•Clinical Study Site, Stanford, California,
United States
•Clinical Study Site, Hollywood, Florida,
United States
•Clinical Study Site, Orlando, Florida,
United States
•Clinical Study Site, Lawrenceville,
Georgia, United States
•Clinical Study Site, Lexington, Kentucky,
United States
•Clinical Study Site, Louisville, Kentucky,
United States
•Clinical Study Site, Boston,
Massachusetts, United States
•and 71 more
- Page 13 of 33 -

14. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
15 A Rollover Extension Program (REP)
to Evaluate the Long-term Safety and
Tolerability of Open Label Iptacopan/
LNP023 in Participants With Primary IgA
Nephropathy
Recruiting No Results
Available
•Primary IgA Nephropathy •Drug: LNP023 Phase:
Phase 3
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Number and percentage of
participants with serious adverse
event
•Number and percentage of
participants with adverse event
•Number and percentage of
participants with adverse events
of special interest
•Number and percentage of
participants with abnormalities in
vital signs
•Number and percentage of
participants with abnormalities in
ECG
•Number and percentage of
participants with abnormalities in
clinical laboratory evaluations
•Annualized total eGFR slope
•Change from baseline in eGFR
•Log transformed ratio to baseline
in UPCR, UACR
Enrollment:
410
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
September 20, 2021
Last Update Posted:
May 16, 2023
•Novartis Investigative Site, Glendale,
Arizona, United States
•Novartis Investigative Site, Kansas City,
Missouri, United States
•Novartis Investigative Site, Parkville,
Victoria, Australia
•Novartis Investigative Site, Edegem,
Antwerpen, Belgium
•Novartis Investigative Site, Leuven,
Belgium
•Novartis Investigative Site, Roeselare,
Belgium
•Novartis Investigative Site, Curitiba, PR,
Brazil
•Novartis Investigative Site, Porto Alegre,
RS, Brazil
•Novartis Investigative Site, Guangzhou,
Guangdong, China
•Novartis Investigative Site, Beijing,
China
•and 31 more
- Page 14 of 33 -

15. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
16 A Study to Evaluate the Effectiveness
and Safety of IONIS-FB-LRx, an
Antisense Inhibitor of Complement Factor
B, in Adult Participants With Primary IgA
Nephropathy
Recruiting No Results
Available
•Primary IgA Nephropathy •Drug: IONIS-FB-LRx Phase:
Phase 2
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Percent Reduction in 24-hour
Urine Protein Excretion
•Absolute Reduction in 24-hour
Urine Protein Excretion
•Absolute Reduction in
Albuminuria (UACr Ratio)
•Absolute Reduction in
Proteinuria (UPCr Ratio)
•Percent Change from Baseline in
Plasma Factor B (FB)
•Percent Change from Baseline in
Plasma AH50
Enrollment:
25
Age:
18 Years to 75 Years
(Adult, Older Adult)
Study Start:
December 4, 2019
Last Update Posted:
August 12, 2022
•IONIS Investigative Site, Liverpool, New
South Wales, Australia
•IONIS Investigative Site, St Leonards,
New South Wales, Australia
•IONIS Investigative Site, Parkville,
Victoria, Australia
•IONIS Investigative Site, Vancouver,
British Columbia, Canada
•IONIS Investigative Site, Toronto,
Ontario, Canada
•IONIS Investigative Site, Christchurch,
New Zealand
•IONIS Investigative Site, Singapore,
Singapore
- Page 15 of 33 -

16. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
17 OL Extension Study of LNP023 in C3G Recruiting No Results
Available
•C3 Glomerulopathy •Drug: LNP023 Phase:
Phase 2
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Number of participants with
composite renal response
•Number of participants with
Adverse Events
•Change in urine protein/
creatinine ratio (UPCR)
•Change in Urine albumin/
creatinine ratio (UACR )
•Change in serum creatinine
•Change in estimated glomerular
filtration rate(eGFR)
•Status of C3G disease
progression
•Levels of complement
component C3
•Levels of complement
component Bb
•Levels of complement
component sC5b-9
•and 5 more
Enrollment:
95
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
October 3, 2019
Last Update Posted:
January 23, 2023
•Novartis Investigative Site, Iowa City,
Iowa, United States
•Novartis Investigative Site, Montpellier,
France
•Novartis Investigative Site, Paris, France
•Novartis Investigative Site, Essen,
Germany
•Novartis Investigative Site, Ranica, BG,
Italy
•Novartis Investigative Site, Barcelona,
Catalunya, Spain
•Novartis Investigative Site, Madrid,
Spain
•Novartis Investigative Site, London,
United Kingdom
•Novartis Investigative Site, Newcastle
Upon Tyne, United Kingdom
- Page 16 of 33 -

17. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
18 A Study of Cemdisiran in Adults With
Immunoglobulin A Nephropathy (IgAN)
Active, not
recruiting
No Results
Available
•IgA Nephropathy (IgAN)
•Berger Disease
•Glomerulonephritis, IgA
•Drug: Placebo
•Drug: Cemdisiran
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Triple (Participant,
Care Provider, Investigator)
•Primary Purpose: Treatment
Outcome Measures:
•Percentage Change From
Baseline in Urine Protein/
Creatinine Ratio (UPCR) as
Measured in 24-hour Urine at
Week 32
•Percent Change from Baseline in
24-Hour Proteinuria at Week 32
•Percentage of Participants with
Partial Clinical Remission at
Week 32
•Percentage of Participants with
>50% Reduction in 24-hour
Proteinuria at Week 32
•Change From Baseline in Urine
Protein/Creatinine Ratio (UPCR)
as Measured in a Spot Urine at
Week 32
•Change From Baseline in
Hematuria at Week 32
•Frequency of Adverse Events
(AEs)
Enrollment:
31
Age:
18 Years to 65 Years
(Adult, Older Adult)
Study Start:
April 24, 2019
Last Update Posted:
March 21, 2023
•Clinical Trial Site, Vancouver, British
Columbia, Canada
•Clinical Trial Site, Brampton, Ontario,
Canada
•Clinical Trial Site, Toronto, Ontario,
Canada
•Clinical Trial Site, Annonay, France
•Clinical Trial Site, Caen, France
•Clinical Trial Site, La Tronche, France
•Clinical Trial Site, Mulhouse, France
•Clinical Trial Site, Paris, France
•Clinical Trial Site, Kuala Lumpur,
Malaysia
•Clinical Trial Site, Kuantan, Malaysia
•and 10 more
- Page 17 of 33 -

18. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
19 Study on Efficacy and Safety of
LNP023 in C3 Glomerulopathy Patients
Transplanted and Not Transplanted
Completed Has Results•Glomerulonephritis •Drug: LNP023 Phase:
Phase 2
Study Design:
•Allocation: Non-Randomized
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Cohort A: Change From Baseline
in Urine Protein to Creatinine
Concentration Ratio (UPCR)
•Cohort B: Change From Baseline
in C3 Deposit
•Change From Baseline in Urine
Protein Creatinine Concentration
Ratio (UPCR)
•Change From Baseline in Urine
Protein (UP) Excretion
•Change From Baseline in
Urine Albumin Creatinine
Concentration Ratio (UACR)
Excretion
•Change From Baseline Change
in Urinary Albumin (UA)
Excretion
•Change From Baseline in
Estimated Glomerular Filtration
Rate (eGFR)
•Change From Baseline in Serum
Creatinine
•Change From Baseline in
Creatinine Clearance
•Number of Patients With
Hematuria
•and 9 more
Enrollment:
27
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
February 20, 2019
Last Update Posted:
August 10, 2022
•Novartis Investigative Site, Iowa City,
Iowa, United States
•Novartis Investigative Site, Montpellier,
France
•Novartis Investigative Site, Paris, France
•Novartis Investigative Site, Essen,
Germany
•Novartis Investigative Site, Ranica, BG,
Italy
•Novartis Investigative Site, Barcelona,
Catalunya, Spain
•Novartis Investigative Site, Madrid,
Spain
•Novartis Investigative Site, London,
United Kingdom
•Novartis Investigative Site, Newcastle
Upon Tyne, United Kingdom
- Page 18 of 33 -

19. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
20 A Proof of Concept Study for a 12 Month
Treatment in Patients With C3G or IC-
MPGN Treated With ACH-0144471
Terminated Has Results•C3 Glomerulonephritis
•C3 Glomerulopathy
•Immune Complex
Membranoproliferative
Glomerulonephritis
•IC-MPGN
•Dense Deposit Disease
•Drug: Danicopan Phase:
Phase 2
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Change From Baseline In
Composite Biopsy Score At End
Of Initial 12-Month Treatment
Period
•Participants With Reduction In
Proteinuria At End Of Initial 12-
Month Treatment Period
•Change From Baseline In
Proteinuria At End Of Initial 12-
Month Treatment Period
•Percent Change From Baseline
In Proteinuria At End Of Initial
12-Month Treatment Period
•Slope Of Estimated Glomerular
Filtration Rate (eGFR) From
Baseline To End Of Initial 12-
Month Treatment Period
•Change From Baseline In eGFR
At End Of Initial 12-Month
Treatment Period
•Participants With Significant
Improvement In eGFR Relative
To Baseline At End Of Initial 12-
Month Treatment Period
•Change From Baseline in eGFR
Over 12 Months of Treatment
For Participants Meeting eGFR
Inclusion Criteria
•Change From Baseline In
Measured GFR At The End Of
The Initial 12-Month Treatment
Period
Enrollment:
22
Age:
12 Years and older (Child,
Adult, Older Adult)
Study Start:
June 20, 2018
Last Update Posted:
August 11, 2022
•Clinical Study Site, Birmingham,
Alabama, United States
•Clinical Study Site, Stanford, California,
United States
•Clinical Study Site, New Haven,
Connecticut, United States
•Clinical Study Site, Cincinnati, Ohio,
United States
•Clinical Study Site, Columbus, Ohio,
United States
•Clinical Study Site, Philadelphia,
Pennsylvania, United States
•Clinical Study Site, Sydney, New South
Wales, Australia
•Clinical Study Site, Brisbane,
Queensland, Australia
•Clinical Study Site, Melbourne, Victoria,
Australia
•Clinical Study Site, Antwerpen, Belgium
•and 3 more
- Page 19 of 33 -

20. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
21 Phase II Study Assessing Safety and
Efficacy of APL-2 in Glomerulopathies
Active, not
recruiting
No Results
Available
•IgA Nephropathy
•Lupus Nephritis
•Membranous Nephropathy
•C3 Glomerulonephritis
•Dense Deposit Disease
•Drug: APL-2 Phase:
Phase 2
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Proteinuria
•Changes of Disease Specific
Biomarkers (serum C3 levels,
AH50 and C3a concentrations,
serum albumin levels)
•Complete clinical remission
defined as normalization of
proteinuria as defined by <200
mg/g uPCR at Week 48
•Stabilization or improvement in
estimated Glomerular Filtration
Rate (eGFR) from baseline to
Week 48
Enrollment:
21
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
January 22, 2018
Last Update Posted:
April 6, 2023
•Stanford University, Stanford, California,
United States
•Children's Hospital Colorado, Aurora,
Colorado, United States
•HealthONE Physician Care, Rocky
Mountain Hospital for Children, Denver,
Colorado, United States
•Washington Nephrology Associates,
Washington, District of Columbia, United
States
•Horizon Research Group, Coral Gables,
Florida, United States
•Emory University, Atlanta, Georgia,
United States
•American Research LLC, Jeffersonville,
Indiana, United States
•Northwest Louisiana Nephrology LLC,
Shreveport, Louisiana, United States
•Washington Nephrology Associates,
Takoma Park, Maryland, United States
•Clinical Research Consultants, Kansas
City, Missouri, United States
•and 7 more
- Page 20 of 33 -

21. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
22 Study of Safety and Efficacy of LNP023
in Patients With Kidney Disease Caused
by Inflammation
Completed Has Results•IgA Nephropathy •Drug: LNP023
•Other: Placebo
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Double (Participant,
Investigator)
•Primary Purpose: Treatment
Outcome Measures:
•MCP-Mod Estimates of the Ratio
to Baseline of Urine Protein to
Creatinine Ratio (UPCR) (g/Mol)
- Parts 1 and 2 at Day 90
•Mixed Model of Repeated
Measures (MMRM) of the
Change From Baseline for
Estimated Glomerular Filtration
Rate (eGFR) - Parts 1 and 2 at
Day 90
•Mixed Model of Repeated
Measures (MMRM) of the
Change From Baseline for
Serum Creatinine - Parts 1 and 2
at Day 90
•Shift Table From Baseline for
Hematuria Levels - Parts 1 and 2
at Day 90
•Mixed Model of Repeated
Measures (MMRM) of the Ratio
to Baseline in 24hour Urine
Protein (UP) - Parts 1 and 2 to
Day 90
•Mixed Model of Repeated
Measures (MMRM) of the Ratio
to Baseline of 24 Hour Urine
Albumin (UA) - Parts 1 and 2 to
Day 90
•Mixed Model of Repeated
Measures (MMRM) of the Ratio
to Baseline in 24 Hour Urine
Albumin to Creatinine (UACR) -
Parts 1 and 2 to Day 90
•Mixed Model of Repeated
Measures (MMRM) of the Ratio
to Baseline in Urine Protein to
Creatinine (UPCR) From 1st
Morning Void - Parts 1 and 2 at
Day 90
•Plasma Pharmacokinetics (PK)
of Area Under the Curve at
Steady State (AUCtau,ss and
AUClast,ss) at Day 30
•Plasma Pharmacokinetics (PK)
of Pre-dose Trough at Steady
State (Ctrough,ss) and Maximum
Concentrations (Cmax,ss) at Day
30
•and 11 more
Enrollment:
112
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
February 7, 2018
Last Update Posted:
January 30, 2023
•Novartis Investigative Site, Caba,
Buenos Aires, Argentina
•Novartis Investigative Site, Ciudad
Autonoma de Bs As, Buenos Aires,
Argentina
•Novartis Investigative Site, Westmead,
New South Wales, Australia
•Novartis Investigative Site, Parkville,
Victoria, Australia
•Novartis Investigative Site, Edegem,
Antwerpen, Belgium
•Novartis Investigative Site, Leuven,
Belgium
•Novartis Investigative Site, Roeselare,
Belgium
•Novartis Investigative Site, Curitiba, PR,
Brazil
•Novartis Investigative Site, Porto Alegre,
RS, Brazil
•Novartis Investigative Site, Guangzhou,
Guangdong, China
•and 46 more
- Page 21 of 33 -

22. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
23 A Proof-of-Concept Study of Danicopan
for 6 Months of Treatment in Participants
With C3 Glomerulopathy (C3G)
Completed Has Results•C3 Glomerulopathy
•C3 Glomerulonephritis
•Dense Deposit Disease
•Drug: Danicopan
•Drug: Placebo
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Double (Participant,
Investigator)
•Primary Purpose: Treatment
Outcome Measures:
•Change From Baseline In
Composite Biopsy Score At
Week 28
•Participants With Reduction In
Proteinuria At Week 28
•Change From Baseline In
Proteinuria At Week 28
•Percent Change From Baseline
In Proteinuria At Week 28
•Slope Of Estimated Glomerular
Filtration Rate (eGFR) From
Baseline To 6 Months
•Slope Of Estimated Glomerular
Filtration Rate (eGFR) After
Open-label Danicopan
Treatment
•Change From Baseline In eGFR
At Week 28
•Participants With Significant
Improvement In eGFR Relative
To Baseline At Week 28
•Participants With Significant
Improvement In eGFR Relative
To Baseline At Week 52
Enrollment:
13
Age:
17 Years to 65 Years
(Child, Adult, Older Adult)
Study Start:
June 12, 2018
Last Update Posted:
October 14, 2022
•Clinical Study Site, Aurora, Colorado,
United States
•Clinical Study Site, Lawrenceville,
Georgia, United States
•Clinical Study Site, Iowa City, Iowa,
United States
•Clinical Study Site, Baltimore, Maryland,
United States
•Clinical Study Site, New York, New
York, United States
•Clinical Study Site, New York, New
York, United States
•Clinical Study Site, London, United
Kingdom
- Page 22 of 33 -

23. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
24 First-In-Human Clinical Study of the
C3 Complement Inhibitor AMY- 101 in
Healthy Male Volunteers
Completed No Results
Available
•Complement Mediated
Diseases
•Drug: AMY-101 Phase:
Phase 1
Study Design:
•Allocation: Randomized
•Intervention Model: Sequential
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Number of participants with
treatment-related adverse events
as assessed by CTCAE v4.0
•Area under the plasma
concentration time curve (AUCt)
after a single dose
•Area under the plasma
concentration time curve from
zero to infinity (AUC 0->#) after a
single dose
•Peak Plasma Concentration
(Cmax) after single and multiple
doses
•Time to Cmax (Tmax) after
single and multiple doses
•Terminal elimination rate
constant (lambdaz) after single
and multiple doses
•Terminal half-life (T1/2) after
single and multiple doses
•Total apparent clearance of drug
from plasma/serum (CL/F) after
single and multiple doses
•Volume of distribution (Vz) /
fraction of drug absorbed (F)
after single and multiple doses
•Area under the curve at steady
state (AUCss) after multiple
doses
•and 7 more
Enrollment:
50
Age:
18 Years to 60 Years
(Adult)
Study Start:
April 24, 2017
Last Update Posted:
January 16, 2018
•HighPoint Clinical Trials Center, High
Point, North Carolina, United States
- Page 23 of 33 -

24. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
25 Controlled Trial Evaluating Avacopan in
C3 Glomerulopathy
Completed Has Results•C3 Glomerulopathy (C3G) •Drug: Avacopan
•Drug: Avacopan Matching
Placebo
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Quadruple (Participant,
Care Provider, Investigator,
Outcomes Assessor)
•Primary Purpose: Treatment
Outcome Measures:
•Change From Baseline to Week
26 in the C3G Histologic Index
for Disease Activity - Subjects
With Elevated C5b-9
•Percent Change From Baseline
to Week 26 in the C3G Histologic
Index for Disease Activity -
Combined C5b-9 Strata
•Proportion of Subjects Who
Have a Histologic Response
Defined as a Decrease
(Improvement) in the Biopsy-
based C3G Histologic Index for
Activity of at Least 35% From
Baseline to 26 Weeks - Subjects
With Elevated C5b-9
•Proportion of Subjects Who
Have a Histologic Response
Defined as a Decrease
(Improvement) in the Biopsy-
based C3G Histologic Index
for Activity of at Least 35%
From Baseline to 26 Weeks -
Combined C5b-9 Strata
•Change From Baseline to Week
26 in the C3G Histologic Index
for Disease Chronicity - Subjects
With Elevated C5b-9
•Change From Baseline to
Week 26 in the C3G Histologic
Index for Disease Chronicity -
Combined C5b-9 Strata
•Renal Function as Assessed by
Percent Change From Baseline
Over 26 Weeks in eGFR -
Subjects With Elevated C5b-9
•Renal Function as Assessed by
Percent Change From Baseline
Over 26 Weeks in eGFR -
Combined C5b-9 Strata
•Renal Function as Assessed by
Change From Baseline Over 26
Weeks in eGFR - Subjects With
Elevated C5b-9
•Renal Function as Assessed
by Change From Baseline Over
26 Weeks in eGFR - Combined
C5b-9 Strata
•and 10 more
Enrollment:
57
Age:
12 Years and older (Child,
Adult, Older Adult)
Study Start:
September 29, 2017
Last Update Posted:
October 14, 2022
•Clinical Site, Palo Alto, California, United
States
•Clinical Site, Chicago, Illinois, United
States
•Clinical Site, Iowa City, Iowa, United
States
•Clinical Site, Boston, Massachusetts,
United States
•Clinical Site, New York, New York,
United States
•Clinical Site, Rochester, New York,
United States
•Clinical Site, Columbus, Ohio, United
States
•Clinical Site, Philadelphia, Pennsylvania,
United States
•Clinical Trial Site, East Providence,
Rhode Island, United States
•Clinical Site, Antwerp, Belgium
•and 31 more
- Page 24 of 33 -

25. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
26 A Proof-of-Mechanism Study to
Determine the Effect of Danicopan on C3
Levels in Participants With C3G or IC-
MPGN
Completed Has Results•C3 Glomerulonephritis
•Dense Deposit Disease
•C3 Glomerulopathy
•Immune Complex Mediated
Membranoproliferative
Glomerulonephritis
•Membranoproliferative
Glomerulonephritis Types I,
II, and III
•Drug: Danicopan Phase:
Phase 2
Study Design:
•Allocation: Non-Randomized
•Intervention Model: Sequential
Assignment
•Masking: None (Open Label)
•Primary Purpose: Basic Science
Outcome Measures:
•Change From Baseline In Serum
C3 Complement Protein (C3)
Levels On Day 15
•Change From Baseline In
Plasma Intact C3 Level On Day
15
•Change From Baseline In Total
Complement Classical Pathway
(CP) Activity On Day 14
•Change From Baseline In
Total Complement Alternative
Pathway (AP) Functional Activity
(AP Wieslab) On Day 15
•Time To Achieving Peak Serum
C3 Levels
•Number Of Participants With
Serious Adverse Events
(SAEs), Grade 3 And Grade 4
Treatment-emergent Adverse
Events (TEAEs), And Adverse
Events (AEs) Leading To
Discontinuation
•Pharmacokinetics (PK):
Area Under The Plasma
Concentration-time Curve From
Time Of Administration To 8
Hours Postdose (AUC0-8)
•PK: Maximum Plasma
Concentration (Cmax)
•PK: Time To Maximum
Concentration (Tmax)
•Change From Baseline In Bb
Fragment Of Complement Factor
B (Bb) At Day 15
•Change From Baseline In
Soluble Terminal Complement
Complex (sC5b-9) At Day 15
Enrollment:
6
Age:
16 Years to 65 Years
(Child, Adult, Older Adult)
Study Start:
August 9, 2017
Last Update Posted:
November 4, 2021
•Clinical Trial Site, Melbourne, Australia
•Clinical Trial Site, Antwerpen, Belgium
•Clinical Trial Site, Leiden, Netherlands
- Page 25 of 33 -

26. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
27 A Phase 3 Clinical Trial of CCX168
(Avacopan) in Patients With ANCA-
Associated Vasculitis
Completed Has Results•ANCA-Associated Vasculitis•Drug: Avacopan
•Drug: Prednisone
•Drug: Cyclophosphamide
•Biological: Rituximab
•Drug: Azathioprine
Phase:
Phase 3
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Quadruple (Participant,
Care Provider, Investigator,
Outcomes Assessor)
•Primary Purpose: Treatment
Outcome Measures:
•Percentage of Subjects
Achieving Disease Remission at
Week 26
•Percentage of Subjects
Achieving Sustained Disease
Remission at Week 52
•Subject Incidence of Treatment-
emergent SAEs, AEs, and
Withdrawals Due to AEs
•Glucocorticoid-induced Toxicity
as Measured by Change From
Baseline Over the First 26
Weeks in the GTI
•Percentage of Participants
With BVAS of 0 at Week
4, Regardless of Whether
the Subjects Received
Glucocorticoids During This
Period of Time and Based on
Assessment by the Blinded AC
•Change From Baseline Over 52
Weeks in Health-related Quality
of Life as Measured by the
Domains and Component Scores
of the SF-36v2 and EQ-5D-5L
VAS and Index
•Percentage of Subjects and
Time to Experiencing a Relapse
After Previously Achieving
Remission at Week 26 in the
Study
•Percentage of Subjects and
Time to Experiencing a Relapse
After Previously Achieving
BVAS=0 at Any Time During the
Treatment Period
•In Subjects With Renal Disease
at Baseline (Based in the BVAS
Renal Component), the Change
in eGFR From Baseline Over 52
Weeks
•In Subjects With Renal Disease
and Albuminuria at Baseline
(Based in the BVAS Renal
Component), the Percent
Change in UACR From Baseline
Over 52 Weeks
•and 18 more
Enrollment:
331
Age:
12 Years and older (Child,
Adult, Older Adult)
Study Start:
March 15, 2017
Last Update Posted:
October 13, 2022
•Clinical Trial Site, Huntsville, Alabama,
United States
•Clinical Trial Site, Phoenix, Arizona,
United States
•Clinical Trial Site, Phoenix, Arizona,
United States
•Clinical Trial Site, Los Angeles,
California, United States
•Clinical Trial Site, Santa Monica,
California, United States
•Clinical Trial Site, Aurora, Colorado,
United States
•Clinical Trial Site, Washington, District of
Columbia, United States
•Clinical Trial Site, Daytona Beach,
Florida, United States
•Clinical Trial Site, Tampa, Florida,
United States
•Clinical Trial Site, Atlanta, Georgia,
United States
•and 188 more
- Page 26 of 33 -

27. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
28 Safety Study of IgAN, LN, MN, & C3
Glomerulopathy Including Dense Deposit
Disease Treated With OMS721
Unknown
status
No Results
Available
•IgAN
•Lupus Nephritis
•MN
•C3 Glomerulopathy
•Biological: OMS721
(narsoplimab)
Phase:
Phase 2
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Cohort 1-3: Proportion of IgAN,
LN, MN, C3 Glomerulopathy
subjects with treatment related
adverse events (AE).
•Cohort 4: Proportion of IgAN
patients of Asian descent with
treatment related AEs.
•Cohort 4: Change from baseline
in serum and urine complement
component levels.
•Cohort 1-3: Change from
baseline in serum narsoplimab
concentrations.
•Cohort 4: Change from
baseline in serum narsoplimab
concentrations.
•Cohort1-3: Change from
baseline in proteinuria.
•Cohort1-3: Change from
baseline in urine albumin/
creatinine ratio.
Enrollment:
54
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
February 2016
Last Update Posted:
April 10, 2020
•Omeros Investigational Site, Denver,
Colorado, United States
•Omeros Investigational Site, Augusta,
Georgia, United States
•Omeros Investigational Site,
Lawrenceville, Georgia, United States
•Omeros Investigational Site, Evergreen
Park, Illinois, United States
•Omeros Investigational Site, Voorhees,
New Jersey, United States
•Omeros Investigational Site, Flushing,
New York, United States
•Omeros Investigational Site, San
Antonio, Texas, United States
•Omeros Investigational Site, Milwaukee,
Wisconsin, United States
•Omeros Investigational Site, Chai Wan,
Hong Kong
•Omeros Investigational Site, Hong Kong,
Hong Kong
•Omeros Investigational Site, Kowloon,
Hong Kong
•Omeros Investigational Site, Sha Tin,
Hong Kong
29 Open-Label Study to Evaluate Safety
and Efficacy of CCX168 in Subjects With
Immunoglobulin A Nephropathy on Stable
RAAS Blockade
Completed No Results
Available
•Immunoglobulin A
Nephropathy
•Drug: CCX168 Phase:
Phase 2
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Safety of CCX168 in subjects
with Immunoglobulin A
nephropathy measured by
number of patients with adverse
events
•Evaluate the efficacy of CCX168
based on numbers of patients
with improvement in proteinuria
Enrollment:
5
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
February 2015
Last Update Posted:
December 20, 2016
•Palo Alto, California, United States
•San Francisco, California, United States
•Reno, Nevada, United States
•Chapel Hill, North Carolina, United
States
•Columbus, Ohio, United States
•Stockholm, Sweden
- Page 27 of 33 -

28. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
30 OMS721 Compassionate Use in Patients
With Thrombotic Microangiopathy
Available No Results
Available
•Thrombotic Microangiopathy•Biological: OMS721 Enrollment:
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
Last Update Posted:
April 15, 2015
- Page 28 of 33 -

29. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
31 Safety and Efficacy Study of
OMS721 in Patients With Thrombotic
Microangiopathies
Completed No Results
Available
•Thrombotic
Microangiopathies
•Biological: OMS721 Phase:
Phase 2
Study Design:
•Allocation: Non-Randomized
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Assess the safety and tolerability
of multiple-dose administration of
OMS721 in subjects with TMA
•Evaluate the response rate to
OMS721 in patients with HSCT-
TMA
•Evaluate the following in patients
with HSCT-TMA treated with
OMS721: 100-day survival
•Evaluate the following in patients
with HSCT-TMA treated with
OMS721: Overall survival
•Evaluate the following in patients
with HSCT-TMA treated with
OMS721: Duration of response
•Evaluate the following in patients
with HSCT-TMA treated with
OMS721: Freedom from platelet
transfusion
•Evaluate the following in patients
with HSCT-TMA treated with
OMS721: Freedom from red
blood cell (RBC) transfusion
•Evaluate the following in
patients with HSCT-TMA treated
with OMS721: Change from
baseline in platelet count, LDH,
haptoglobin, hemoglobin (Hgb),
creatinine
•Evaluate the following in patients
with HSCT-TMA, aHUS, and
TTP: Pharmacokinetics (PK) of
multiple-dose administration of
OMS721
•Evaluate the following in patients
with HSCT-TMA, aHUS, and
TTP: Pharmacodynamics (PD) of
multiple-dose administration of
OMS721 in subjects with TMA
•Evaluate the following in patients
with HSCT-TMA, aHUS, and
TTP: Immunogenicity of multiple-
dose administration of OMS721
in subjects with TMA
Enrollment:
58
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
November 2, 2014
Last Update Posted:
July 6, 2021
•Omeros Investigational Site, Duarte,
California, United States
•Omeros Investigational Site, Rochester,
Minnesota, United States
•Omeros Investigational Site, New York,
New York, United States
•Omeros Investigational Site, Durham,
North Carolina, United States
•Omeros Investigational Site, Madison,
Wisconsin, United States
•Omeros Investigational Site, Brussels,
Belgium
•Omeros Investigational Site, Leuven,
Belgium
•Omeros Investigational Site, Liege,
Belgium
•Omeros Investigational Site, Sofia,
Bulgaria
•Omeros Investigational Site, PokFuLam,
Hong Kong
•and 15 more
- Page 29 of 33 -

30. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
32 Clinical Trial to Evaluate Safety and
Efficacy of CCX168 in ANCA-Associated
Vasculitis
Completed No Results
Available
•ANCA-associated Vasculitis•Drug: CCX168 low dose
plus standard of care
•Drug: CCX168 high dose
plus standard of care
•Other: Placebo BID plus
standard of care
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Quadruple (Participant,
Care Provider, Investigator,
Outcomes Assessor)
•Primary Purpose: Treatment
Outcome Measures:
•BVAS
•eGFR
•Hematuria
•Albuminuria
•Urinary monocyte
chemoattractant protein-1
(MCP-1)
Enrollment:
42
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
September 2014
Last Update Posted:
November 16, 2016
•Huntsville, Alabama, United States
•Phoenix, Arizona, United States
•Tucson, Arizona, United States
•Long Beach, California, United States
•Los Angeles, California, United States
•San Francisco, California, United States
•Aurora, Colorado, United States
•Washington, District of Columbia, United
States
•Miami Springs, Florida, United States
•Tampa, Florida, United States
•and 33 more
33 Eculizumab in Primary MPGN Completed No Results
Available
•Membranoproliferative
Glomerulonephritis
•Drug: Eculizumab Phase:
Phase 2
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•24hours proteinuria
•Terminal complement complex
(sC5b-9) levels
•Glomerular filtration rate (GFR)
measured by iohexol plasma
clearance and estimated.
•Time to disease progression.
Enrollment:
10
Age:
up to 75 Years (Child,
Adult, Older Adult)
Study Start:
March 2014
Last Update Posted:
January 17, 2018
•Ospedale Pediatrico "Giovanni XXIII" -
U:O Nefrologia, Bari, BA, Italy
•A.O. Papa Giovanni XXIII - U.O.
Nefrologia e Dialisi/IRCCS IRFMN
- Centro di Ricerche Cliniche per le
Malattie Rare Aldo e Cele Daccò,
Bergamo, BG, Italy
•Policlinico Sant'Orsola -Malpighi -
U.O.S. Nefrologia e dialisi pediatrica,
Bologna, BO, Italy
•Policlinico "G.Martino" - U.O. Nefrologia
e Dialisi, Messina, ME, Italy
•Policlinico Universitario di Padova - U.O.
Nefrologia Pediatrica, Padova, PD, Italy
•Ospedale Centrale, Bolzano, Italy
•Policlinico "Federico II" - U.O.
Nefrologia, Napoli, Italy
•Ospedale degli Infermi - U.O. Nefrologia
e Dialisi, Rimini, Italy
•C.I. Columbus-Università Cattolica del
S.Cuore - UOC Nefrologia e Dialisi,
Roma, Italy
•Ospedale Pediatrico "Bambin Gesù" -
U.O. Nefrologia, Roma, Italy
•and 3 more
- Page 30 of 33 -

31. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
34 Clinical Trial of CDX-1135 in Pediatric
and Adult Patients With Dense Deposit
Disease
Terminated No Results
Available
•Dense Deposit Disease
•Membranoproliferative
Glomerulonephritis Type II
•C3 Glomerulonephritis
•Drug: CDX-1135 Phase:
Phase 1
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Safety
•C3 and AP Normalization
•Duration of and time to normalize
C3 and AP
•Renal Function
•Renal biopsy
•Immunogenicity
Enrollment:
1
Age:
4 Years and older (Child,
Adult, Older Adult)
Study Start:
January 2013
Last Update Posted:
March 7, 2014
•University of Iowa Hospitals & Clinics,
Iowa City, Iowa, United States
35 A Study to Evaluate the Safety and
Efficacy of CCX168 in Subjects With
ANCA-Associated Vasculitis
Completed Has Results•Vasculitis •Drug: Placebo
•Drug: CCX168
Phase:
Phase 2
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Triple (Participant,
Care Provider, Investigator)
•Primary Purpose: Treatment
Outcome Measures:
Proportion of Subjects Achieving
at Least 50% Reduction in
Birmingham Vasculitis Activity
Score [BVAS] by Week 12 and No
Worsening in Any Body System
Enrollment:
67
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
August 2011
Last Update Posted:
July 27, 2020
•Feldkirch, Austria
•Innsbruck, Austria
•Linz, Austria
•Brussels, Belgium
•Edegem, Belgium
•Gent, Belgium
•Leuven, Belgium
•Liege, Belgium
•Roeselare, Belgium
•Prague, Czechia
•and 34 more
36 Eculizumab Therapy for Dense Deposit
Disease and C3 Nephropathy
Completed Has Results•Dense Deposit Disease
•Membranoproliferative
Glomerulonephritis
•Drug: Eculizumab Phase:
Phase 1
Study Design:
•Allocation: N/A
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
Number of Patients With Change
in Proteinuria or Serum Creatinine
Over Treatment Period
Enrollment:
6
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
July 2010
Last Update Posted:
February 19, 2019
•Columbia University Medical Center,
Glomerular Center, New York, New
York, United States
•Columbia University Medical Center,
Nephrology Clinical Research Center,
New York, New York, United States
- Page 31 of 33 -

32. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
37 Open Label Pilot Trial of Rituximab in
Lupus Membranous Nephritis
Withdrawn No Results
Available
•Lupus Nephritis •Drug: Rituximab Phase:
Not Applicable
Study Design:
•Allocation: Non-Randomized
•Intervention Model: Single Group
Assignment
•Masking: None (Open Label)
•Primary Purpose: Treatment
Outcome Measures:
•Efficacy at 6 months post
treatment and durability of
response at 12 months post
treatment will be determined
by: reduction in 24 hour urinary
protein excretion, reciprocal
creatinine, urinary sediment
•Change from baseline at
6 and 12 months in non-
renal SLE disease activity
(BILAG), lupus serologies
(anti-dsDNA antibodies) and
complement consumption (C3,
C4), serum albumin, cholesterol,
triglycerides
Enrollment:
0
Age:
18 Years and older (Adult,
Older Adult)
Study Start:
December 2008
Last Update Posted:
May 14, 2015
- Page 32 of 33 -

33. Title Status Study
Results
Conditions Interventions Characteristics Population Dates Locations
38 A Study to Evaluate the Efficacy and
Safety of Rituximab in Subjects With
International Society of Nephrology/Renal
Pathology Society (ISN/RPS) 2003 Class
III or IV Lupus Nephritis
Completed Has Results•Lupus Nephritis •Drug: Rituximab
•Drug: Placebo
•Drug: Mycophenolate
mofetil
•Drug: Methylprednisolone
•Drug: Diphenhydramine
•Drug: Acetaminophen
•Drug: Prednisone
Phase:
Phase 3
Study Design:
•Allocation: Randomized
•Intervention Model: Parallel
Assignment
•Masking: Double (Participant,
Investigator)
•Primary Purpose: Treatment
Outcome Measures:
•Percentage of Participants Who
Achieved a Complete Renal
Response (CRR), a Partial Renal
Response (PRR), or no Renal
Response (NRR) at Week 52
•Percentage of Participants Who
Achieved a Complete Renal
Response at Week 24 and
Maintained it to Week 52
•Percentage of Participants Who
Achieved a Complete Renal
Response at Week 52
•Percentage of Participants With
a Baseline Urine Protein to
Creatinine Ratio of > 3.0 Who
Achieved a Urine Protein to
Creatinine Ratio of < 1.0 at Week
52
•British Isles Lupus Assessment
Group (BILAG) Index Score Over
52 Weeks
•Time to Achieve a Complete
Renal Response
•Change From Baseline in the
Systemic Lupus Erythematosus
Expanded Health Survey
Physical Function Score at Week
52
•Change From Baseline in Anti-
double-stranded DNA at Week
52
•Change From Baseline in C3
and C4 Complement Levels at
Week 52
Enrollment:
144
Age:
16 Years to 75 Years
(Child, Adult, Older Adult)
Study Start:
January 2006
Last Update Posted:
January 15, 2015
U.S. National Library of Medicine | U.S. National Institutes of Health | U.S. Department of Health & Human Services
- Page 33 of 33 -