Controlled Trial Evaluating Avacopan in C3 Glomerulopathy | NCT03301467

NCT03301467

ID	Type	Description	Link
1R01FD006342-01	U.S. FDA Grant/Contract		View source

Status Module

Sponsor/Collaborators Module

Name	Class
Medpace, Inc.	INDUSTRY

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Avacopan	Experimental	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period	Drug: Avacopan
Avacopan Matching Placebo	Placebo Comparator	Matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period	Drug: Avacopan Matching Placebo

Name	Type	Description	Arm Group Labels	Other Names
Avacopan	Drug	Orally administered	Avacopan	CCX168

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Change From Baseline to Week 26 in the C3G Histologic Index for Disease Activity - Subjects With Elevated C5b-9	Change from baseline to Week 26 in the biopsy-based C3G Histologic Index for disease activity - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population. C3G Histological Index for Disease Activity Scores can range from 0 to 21. A decrease indicates improvement. C3G=C3 glomerulopathy	Week 26
Percent Change From Baseline to Week 26 in the C3G Histologic Index for Disease Activity - Combined C5b-9 Strata	Percent change from baseline to Week 26 in the biopsy-based C3G Histologic Index for disease activity - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population. C3G Histological Index for Disease Activity Scores can range from 0 to 21. A decrease indicates improvement. C3G=C3 glomerulopathy * Multiple Imputation: Missing Week 26 values are imputed using the regression method to create 100 complete datasets.	Week 26

Secondary Outcomes

Measure	Description	Time Frame
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 - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population. C3G Histological Index for Disease Activity Scores can range from 0 to 21. A decrease indicates improvement. C3G=C3 glomerulopathy

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Biopsy-proven C3G, either DDD or C3GN, with or without a renal transplant, and with the following observations upon renal biopsy taken within 12 weeks prior to screening or during screening:
1. ≥2-levels of magnitude greater staining of C3 than any combination of IgG, IgM, IgA, kappa and lambda light chains, and C1q by immunohistochemistry, and
2. evidence of proliferative glomerulonephritis (mesangial hypercellularity of greater than 3 mesangial cells per mesangial area and/or endocapillary hypercellularity defined as an increased number of cells within glomerular capillary lumina, causing luminal narrowing) based on light microscopy, and
3. confirmation of the presence of electron dense deposits in the glomeruli on electron microscopy corresponding with the C3 immunofluorescence positivity;
Male or female subjects, aged at least 18 years; where approved, adolescents (12-17 year old) may be enrolled; female subjects of childbearing potential (i.e., those who have experienced menarche and who is not permanently sterile or postmenopausal, defined as at least 12 consecutive months with no menses without an alternative medical cause) may participate if adequate contraception is used during, and for at least the three months after study completion; Male subjects with partners of childbearing potential may participate in the study if they had a vasectomy at least 6 months prior to randomization or if adequate contraception is used during, and for at least the 3 months after study completion; Adequate contraception is defined as resulting in a failure rate of less than 1% per year (combined estrogen and progestogen [oral, intravaginal, or transdermal], or progestogen-only hormonal contraception (oral, injectable, or implantable), intra-uterine device, intra-uterine hormone releasing system, bilateral tubal occlusion, vasectomized partner, or true sexual abstinence, i.e., in line with the preferred and usual lifestyle of the subject);
Willing and able to give written Informed Consent and to comply with the requirements of the study protocol; written Assent and Informed Consent must be obtained from the legal guardian in accordance with regional laws or regulations for subjects 12 to 17 years of age; and
Judged to be otherwise fit for the study by the Investigator, based on medical history, physical examination, and clinical laboratory assessments. Subjects with clinical laboratory values that are outside of normal limits (other than those specified in the Exclusion Criteria) and/or with other abnormal clinical findings that are judged by the Investigator not to be of clinical significance, may be entered into the study.

Exclusion Criteria:

Pregnant or nursing;
Tubulointerstitial fibrosis appears to be more than 50% based on standard assessment using trichrome staining of the renal biopsy;
Use of eculizumab or another anti-C5 antibody within 26 weeks prior to dosing;
Secondary C3 disease, e.g., infection-associated disease, or associated with another systemic or autoimmune disease; presence of a monoclonal spike on serum or urine protein electrophoresis or immunofixation assay;
Currently on dialysis or likely will require dialysis within 7 days after screening;
History or presence of any form of cancer within the 5 years prior to screening, with the exception of excised basal cell or squamous cell carcinoma of the skin, or carcinoma in situ such as cervical or breast carcinoma in situ that has been excised or resected completely and is without evidence of local recurrence or metastasis;
Positive hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) viral screening test indicative of acute or chronic infection;
Evidence of tuberculosis based on interferon γ release assay (IGRA), tuberculin purified protein derivative (PPD) skin test, or chest radiography done at screening or within 6 weeks prior to screening;
WBC count less than 3500/μL, or neutrophil count less than 1500/μL, or lymphocyte count less than 500/μL before start of dosing;
Evidence of hepatic disease; AST, ALT, alkaline phosphatase, or bilirubin >3 x the upper limit of normal before start of dosing;
Currently using a strong inducer of the CYP3A4 enzyme, such as carbamazepine, phenobarbital, phenytoin, rifampin, or St. John's wort;
Known hypersensitivity to avacopan or inactive ingredients of the avacopan capsules (including gelatin, polyethylene glycol, or Cremophor) or inability to swallow the capsules;
Participated in any clinical study of an investigational product within 30 days prior to screening or within 5 half-lives after taking the last dose; and
History or presence of any medical condition or disease which, in the opinion of the Investigator, may place the subject at unacceptable risk for study participation.

Contacts/Locations Module

Name	Affiliation	Role
MD	Amgen	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Clinical Site		Palo Alto	California	94305	United States
Clinical Site

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
39392695	Derived	Bomback AS, Herlitz LC, Kedia PP, Petersen J, Yue H, Lafayette RA; ACCOLADE Study Group. Safety and Efficacy of Avacopan in Patients with Complement 3 Glomerulopathy: Randomized, Double-Blind Clinical Trial. J Am Soc Nephrol. 2025 Mar 1;36(3):487-499. doi: 10.1681/ASN.0000000526. Epub 2024 Oct 11.

IPD Sharing Statement Module

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
FG001	Avacopan Group

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Mar 20, 2019	May 19, 2022

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

Masking Info

Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period

Avacopan: Orally administered

Type of Units Analyzed

Arm/Group Information

ID	Title	Description
BG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
BG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered
BG002	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	OG000-0.9(-2.2 to 0.4) OG001-1.0(-2.3 to 0.4)

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		ANCOVA		0.9670		Mean Difference (Final Values)	-0.0			2-Sided	95	-1.9	1.8					Superiority

Primary

Percent Change From Baseline to Week 26 in the C3G Histologic Index for Disease Activity - Combined C5b-9 Strata

Percent change from baseline to Week 26 in the biopsy-based C3G Histologic Index for disease activity - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

C3G Histological Index for Disease Activity Scores can range from 0 to 21. A decrease indicates improvement.

C3G=C3 glomerulopathy

* Multiple Imputation: Missing Week 26 values are imputed using the regression method to create 100 complete datasets.

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population

Posted

Mean

Standard Error

Percentage change

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

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 - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

C3G Histological Index for Disease Activity Scores can range from 0 to 21. A decrease indicates improvement.

C3G=C3 glomerulopathy

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with C3G Histological Index of Disease Activity Percent Change at Week 26 is reported.

Posted

Count of Participants

Participants

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

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 (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

C3G Histological Index for Disease Activity Scores can range from 0 to 21. A decrease indicates improvement.

C3G=C3 glomerulopathy

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with C3G Histological Index of Disease Activity Percent Change at Week 26 is reported.

The ITT Population included all randomized subjects who received at least one dose of study medication; subjects with a histologic activity score of 0 at baseline or who were ongoing and had not completed the Week 26 visit were not included in the ITT population.

Posted

Count of Participants

Participants

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

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 biopsy-based C3G Histologic Index for disease chronicity over the placebo-controlled treatment period - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

C3G Histological Index for Disease Chronicity Scores can range from 0 to 10. A decrease indicates improvement.

C3G=C3 glomerulopathy

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

score on a scale

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Change From Baseline to Week 26 in the C3G Histologic Index for Disease Chronicity - Combined C5b-9 Strata

Change from baseline to Week 26 in the biopsy-based C3G Histologic Index for disease chronicity over the placebo-controlled treatment period - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

C3G Histological Index for Disease Chronicity Scores can range from 0 to 10. A decrease indicates improvement.

C3G=C3 glomerulopathy

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

score on a scale

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Renal Function as Assessed by Percent Change From Baseline Over 26 Weeks in eGFR - Subjects With Elevated C5b-9

The percent change from baseline over 26 weeks in estimated Glomerular Filtration Rate (eGFR) - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

Percentage change

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Units

Secondary

Renal Function as Assessed by Percent Change From Baseline Over 26 Weeks in eGFR - Combined C5b-9 Strata

The percent change from baseline over 26 weeks in estimated Glomerular Filtration Rate (eGFR) - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

Percentage change

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Renal Function as Assessed by Change From Baseline Over 26 Weeks in eGFR - Subjects With Elevated C5b-9

The change from baseline over 26 weeks in estimated Glomerular Filtration Rate (eGFR) - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

mL/min/1.73m^2

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Units

Secondary

Renal Function as Assessed by Change From Baseline Over 26 Weeks in eGFR - Combined C5b-9 Strata

The change from baseline over 26 weeks in estimated Glomerular Filtration Rate (eGFR) - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

mL/min/1.73m^2

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Percent Change From Baseline Over 26 Weeks in UPCR in Patients With Abnormal UPCR at Baseline - Subjects With Elevated C5b-9

Percent change from baseline Over 26 Weeks in UPCR in patients with Abnormal UPCR at Baseline (>= 0.15 g/g) - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

LSM=Least Squares Mean; UPCR = Urine Protein : Creatinine Ratio

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

Percentage change

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Percent Change From Baseline Over 26 Weeks in UPCR in Patients With Abnormal UPCR at Baseline - Combined C5b-9 Strata

Percent change from baseline Over 26 Weeks in UPCR in patients with Abnormal UPCR at Baseline (>= 0.15 g/g) - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

LSM=Least Squares Mean; UPCR = Urine Protein : Creatinine Ratio

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

Percentage change

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Percent Change From Baseline Over 26 Weeks in Urinary MCP-1 - Subjects With Elevated C5b-9

Percent change from baseline over 26 weeks in urinary MCP-1: creatinine ratio - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

LSM=Least Squares Mean; MCP-1=Monocyte Chemoattractant Protein-1

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

Percentage change

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Percent Change From Baseline Over 26 Weeks in Urinary MCP-1 - Combined C5b-9 Strata

Percent change from baseline over 26 weeks in urinary MCP-1: creatinine ratio - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

LSM=Least Squares Mean; MCP-1=Monocyte Chemoattractant Protein-1

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

Percentage change

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Change From Baseline Over 26 Weeks in EQ-5D-5L Health Scale VAS and Index Score - Subjects With Elevated C5b-9

Change from baseline over 26 weeks in EQ-5D-5L Health Scale VAS and Index Score - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

EQ-5D-5L: EuroQuality of Life-5 Domains-5 Levels. The EQ-5D-5L consists of 2 pages: the EQ-5D descriptive system and the EQ visual analogue scale (EQ VAS). The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The answers given can be converted into an Index Score ranging from 0 for death to 1 for perfect health. The EQ-5D questionnaire also includes a Visual Analog Scale (VAS), by which respondents can report their perceived health status with a grade ranging from 0 (the worst imaginable health) to 100 (the best imaginable health).

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

score on a scale

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered

Secondary

Change From Baseline Over 26 Weeks in EQ-5D-5L Health Scale VAS and Index Score - Combined C5b-9 Strata

Change from baseline over 26 weeks in EQ-5D-5L Health Scale VAS and Index Score - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

score on a scale

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered

Secondary

Change From Baseline Over 26 Weeks in SF-36 v2 - Subjects With Elevated C5b-9

Change from baseline over 26 weeks in SF-36 v2 - Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat Population.

SF-36v2: Medical Outcomes Survey Short Form-36 version 2.

SF-36v2 measures each of the following eight health domains: Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role-Emotional, and Mental Health. Scores on each item are summed and averaged. The SF-36v2 component domain scores range from 0 (worst health) to 100 (best health).

Subjects with Elevated C5b-9 (> 244 ng/mL) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

score on a scale

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Change From Baseline Over 26 Weeks in SF-36 v2 - Combined C5b-9 Strata

Change from baseline over 26 weeks in SF-36 v2 - Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat Population.

SF-36v2: Medical Outcomes Survey Short Form-36 version 2.

Combined C5b-9 Strata (elevated [> 244 ng/mL] and non-elevated C5b-9) in the Intent-to-Treat (ITT) Population. The number of subjects with data at baseline and the specified visit is reported.

Posted

Least Squares Mean

95% Confidence Interval

score on a scale

Week 26

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Secondary

Number of Subjects With Treatment-emergent SAEs, AEs, Relatedness to Study Medication and Withdrawals Due to AEs

Number of Subjects with Treatment-emergent SAEs, AEs, relatedness to study medication and Withdrawals Due to AEs

AEs=Adverse events SAEs=Serious adverse events TEAE=Treatment-emergent adverse events 'Possibly related' refers to the Investigators' causality assessment

Safety Population: The safety population included all subjects who were randomized and had received at least one dose of study drug.

Posted

Number

participants

From day 1 throughout the study period (day 182/week 26)

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Units	Counts
Participants

Secondary

Number of Treatment-emergent SAEs, AEs, Relatedness to Study Medication and Withdrawals Due to AEs

Number of Treatment-emergent SAEs, AEs, relatedness to study medication and Withdrawals Due to AEs

AEs=Adverse events SAEs=Serious adverse events TEAE=Treatment-emergent adverse events 'Possibly related' refers to the Investigators' causality assessment

Safety Population: The safety population included all subjects who were randomized and had received at least one dose of study drug.

Posted

Number

Adverse events

From day 1 throughout the study period (day 182/week 26)

ID	Title	Description
OG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered
OG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered

Units	Counts
Participants

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Placebo Group	Avacopan-matching placebo capsules x 3 administered twice daily during the 26 week blinded treatment period period Avacopan-matching placebo: Orally administered The safety population included all subjects who were randomized and had received at least one dose of study drug.	0	29	3	29	24	29
EG001	Avacopan Group	Avacopan (formerly CCX168) 10 mg capsules x 3 administered twice daily during the blinded 26 week blinded treatment period Avacopan: Orally administered The safety population included all subjects who were randomized and had received at least one dose of study drug.	0	28	3	28	25	28

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Gastroenteritis salmonella	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected28 at risk
Neutropenic sepsis	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected28 at risk
Pneumonia	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0011 events1 affected28 at risk
Bacterial parotitis	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected28 at risk
Bronchitis	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected28 at risk
Neutrophil count decreased	Investigations	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected28 at risk
Sjogren's syndrome	Musculoskeletal and connective tissue disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected28 at risk
Acute kidney injury	Renal and urinary disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected28 at risk
Alcohol withdrawal syndrome	Psychiatric disorders	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected28 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Oedema peripheral	General disorders	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0016 events5 affected28 at risk
Fatigue	General disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0013 events3 affected28 at risk
Pyrexia	General disorders	MedDRA 20.1	Systematic Assessment	EG0004 events2 affected29 at risk EG0010 events0 affected28 at risk
Influenza	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0012 events2 affected28 at risk
Lower respiratory tract infection	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected28 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0003 events3 affected29 at risk EG0013 events2 affected28 at risk
Nasopharyngitis	Infections and infestations	MedDRA 20.1	Systematic Assessment	EG0004 events3 affected29 at risk EG0011 events1 affected28 at risk
Vomiting	Gastrointestinal disorders	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0014 events4 affected28 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 20.1	Systematic Assessment	EG0004 events3 affected29 at risk EG0012 events2 affected28 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 20.1	Systematic Assessment	EG0003 events3 affected29 at risk EG0011 events1 affected28 at risk
Nausea	Gastrointestinal disorders	MedDRA 20.1	Systematic Assessment	EG0003 events3 affected29 at risk EG0011 events1 affected28 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected28 at risk
Mouth ulceration	Gastrointestinal disorders	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected28 at risk
Blood creatine phosphokinase increased	Investigations	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0014 events3 affected28 at risk
Blood creatinine increased	Investigations	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0013 events3 affected28 at risk
Weight decreased	Investigations	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0013 events3 affected28 at risk
Lipase increased	Investigations	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected28 at risk
Aspartate aminotransferase increased	Investigations	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0011 events1 affected28 at risk
Anaemia	Blood and lymphatic system disorders	MedDRA 20.1	Systematic Assessment	EG0003 events3 affected29 at risk EG0013 events3 affected28 at risk
Lymphopenia	Blood and lymphatic system disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0013 events3 affected28 at risk
Hand fracture	Injury, poisoning and procedural complications	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected28 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected28 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 20.1	Systematic Assessment	EG0003 events3 affected29 at risk EG0011 events1 affected28 at risk
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA 20.1	Systematic Assessment	EG0003 events3 affected29 at risk EG0010 events0 affected28 at risk
Headache	Nervous system disorders	MedDRA 20.1	Systematic Assessment	EG0004 events2 affected29 at risk EG0015 events5 affected28 at risk
Tremor	Nervous system disorders	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected28 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0013 events3 affected28 at risk
Hypokalaemia	Metabolism and nutrition disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected28 at risk
Hyperkalaemia	Metabolism and nutrition disorders	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0012 events1 affected28 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0011 events1 affected28 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0011 events1 affected28 at risk
Acne	Skin and subcutaneous tissue disorders	MedDRA 20.1	Systematic Assessment	EG0001 events1 affected29 at risk EG0012 events2 affected28 at risk
Depression	Psychiatric disorders	MedDRA 20.1	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected28 at risk
Hypertension	Vascular disorders	MedDRA 20.1	Systematic Assessment	EG0003 events3 affected29 at risk EG0014 events3 affected28 at risk
Orthostatic hypotension	Vascular disorders	MedDRA 20.1	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected28 at risk

First MCP Posted Date

ID	Term
D015432	Glomerulonephritis, Membranoproliferative

Ancestor Terms

ID	Term
D005921	Glomerulonephritis
D009393	Nephritis
D007674	Kidney Diseases
D014570	Urologic Diseases
D052776	Female Urogenital Diseases
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D052801	Male Urogenital Diseases
D007154	Immune System Diseases

ID	Term
C000620232	avacopan

Units	Counts
Participants	OG00021 OG00119

Units	Counts
Participants	OG00020 OG00118

Units	Counts
Participants	OG00025 OG00124

Units	Counts
Participants	OG00024 OG00123

(-4.2634 to 11.2958)

(-4.6899 to 11.1821)