Phase 2
N=13
A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)
C3 Glomerulopathy · C3 Glomerulonephritis · Dense Deposit Disease
Bottom Line
View on ClinicalTrials.gov: NCT03369236 ↗Enrolled (actual)
13
Serious AEs
4.0%
Results posted
Nov 2021
Primary outcome: Primary: Change From Baseline In Composite Biopsy Score At Week 28 — 11.7; 9.3; 9.2; 10.7 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Danicopan (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 17+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Dec 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline In Composite Biopsy Score At Week 28 |
11.7; 9.3; 9.2; 10.7; -2.0; 1.3 | — |
| PRIMARY Participants With Reduction In Proteinuria At Week 28 |
0; 1 | — |
| SECONDARY Change From Baseline In Proteinuria At Week 28 |
6137.67; 4274.47; 5301.75; 5186.80; 302.00; 182.20 | — |
| SECONDARY Percent Change From Baseline In Proteinuria At Week 28 |
12.5; -10.4 | — |
| SECONDARY Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To 6 Months |
-2.26840; -1.45421 | — |
| SECONDARY Slope Of Estimated Glomerular Filtration Rate (eGFR) After Open-label Danicopan Treatment |
-1.02556; -1.84935; -1.39169 | — |
| SECONDARY Change From Baseline In eGFR At Week 28 |
79.890; 68.381; 67.543; 50.874; -12.347; -8.700 | — |
| SECONDARY Participants With Significant Improvement In eGFR Relative To Baseline At Week 28 |
0; 0 | — |
| SECONDARY Participants With Significant Improvement In eGFR Relative To Baseline At Week 52 |
0; 0 | — |
Summary
The primary purpose of this proof-of-concept clinical study was to evaluate the efficacy and safety of the study drug, ACH-0144471 (also known as danicopan and ALXN2040), in participants with C3G who also had significant proteinuria attributable to C3G.
Eligibility Criteria
Key Inclusion Criteria
- Had biopsy-confirmed primary C3G
- Had clinical evidence of ongoing disease based on significant proteinuria, attributable to C3G disease in the opinion of the Principal Investigator (PI), and present prior to study entry and confirmed during Screening
- Was willing to comply with vaccination requirements.
Key Exclusion Criteria
- Had a history or presence of any clinically relevant co-morbidities that would make the participant inappropriate for the study
- Had ever received danicopan
- Had more than 50% fibrosis or more than 50% of glomeruli with cellular crescents on the pre-treatment renal biopsy
- Had an estimated glomerular filtration rate 38°Celsius, or other evidence of a clinically significant active infection, within 14 days prior to study drug administration
- Had evidence of human immunodeficiency virus, hepatitis B infection, or active hepatitis C infection at Screening
- Had laboratory abnormalities at screening that, in the opinion of the PI, would make the participant inappropriate for the study
Data sourced from ClinicalTrials.gov (NCT03369236). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.