NCT03459443

A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471

An Open-Label Phase 2 Proof-of-Concept Study in Patients With C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) Treated With ACH-0144471

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study was to evaluate the efficacy of 12 months of oral ACH-0144471 (also known as danicopan and ALXN2040) in participants with C3G or IC-MPGN based on histologic scoring and proteinuria.

Detailed description

This was an open-label study to evaluate the efficacy of treatment with danicopan in participants 12 years of age or older with biopsy-confirmed C3G or IC-MPGN who had not undergone renal transplantation. All participants were to receive active treatment with danicopan for approximately 40 months. The starting dosage was to be 100 mg TID, and after 2 weeks, the dosage was to be increased to 200 mg TID for participants with body weight ≥ 60 kg or 150 mg TID for participants with body weight \< 60 kg. Planned enrollment was approximately 20 participants.

Conditions

• C3 Glomerulonephritis
• C3 Glomerulopathy
• Immune Complex Membranoproliferative Glomerulonephritis
• IC-MPGN
• Dense Deposit Disease

Interventions

Type	Name	Description
DRUG	Danicopan	Danicopan was to be administered as an oral tablet.

Timeline

• Start date: 2018-06-20
• Primary completion: 2021-03-29
• Completion: 2021-03-29
• First posted: 2018-03-09
• Last updated: 2023-08-21
• Results posted: 2022-08-11

Locations

13 sites across 5 countries: United States, Australia, Belgium, Italy, Netherlands

Regulatory

• FDA-regulated drug study