A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN

• Status: Completed
• Trial end date: 2019-01-09
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study was to determine whether ACH-0144471 (also known as danicopan and ALXN2040) increases blood C3 complement protein (C3) levels in participants with low C3 levels due to either C3G or IC-MPGN.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Achillion Pharmaceuticals; Alexion Pharmaceuticals
• Collaborator: Achillion, a wholly owned subsidiary of Alexion

Criteria

Key Inclusion Criteria:

• Must have had clinical diagnosis of C3G (C3 glomerulonephritis or dense deposit disease, the 2 types of C3G) or idiopathic IC-MPGN by renal biopsy for at least 3 months prior to dosing, with the pathologic diagnosis verified by a review of the renal biopsy by the study central pathologist

• C3 must have been <50% of the lower limit of normal

• C4 complement protein (C4) must have been >90% of the lower limit of normal

• Must have been willing to comply with study-specific vaccination requirements for Haemophilus influenzae, Streptococcus pneumoniae, and Neisseria meningitidis strains A, C, W, and Y

• Negative pregnancy test for females prior to dosing and throughout the study

Key Exclusion Criteria:

• History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant. Individuals receiving renal replacement therapy were also excluded

• Evidence of monoclonal gammopathy of unclear significance, infections, malignancy, autoimmune diseases, or other conditions to which C3G or IC-MPGN may have been secondary

• Estimated glomerular filtration rate (using Modification of Diet in Renal Disease equation) <45 milliliters/minute/1.73 square meters at the time of Screening or at any time over the preceding 4 weeks

• Receipt of eculizumab at any dose or interval within the past 75 days prior to dosing

• Use of tacrolimus or cyclosporine within 2 weeks of the first dose of danicopan

• History of febrile illness, a body temperature >38°Celsius, or other evidence of a clinically significant active infection, within 14 days prior to study drug administration

• History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection

• Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration