Status:

COMPLETED

A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)

Lead Sponsor:

Alexion Pharmaceuticals, Inc.

Conditions:

C3 Glomerulopathy

C3 Glomerulonephritis

Eligibility:

All Genders

17-65 years

Phase:

PHASE2

Brief 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 ...

Eligibility Criteria

Inclusion

  • Key
  • 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

  • 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 \<30 milliliters/minute/1.73 meters squared at the time of screening or at any time over the preceding 4 weeks
  • Was a renal transplant recipient or receiving renal replacement therapy
  • Had a history of a major organ transplant or hematopoietic stem cell/marrow transplant
  • Had evidence of monoclonal gammopathy of unclear significance, infections, malignancy, autoimmune diseases, or other conditions to which C3G is secondary
  • Had other renal diseases that would interfere with interpretation of the study
  • Had been diagnosed with or showed evidence of hepatobiliary cholestasis
  • Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration
  • Had a 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
  • 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

Key Trial Info

Start Date :

June 12 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 18 2020

Estimated Enrollment :

13 Patients enrolled

Trial Details

Trial ID

NCT03369236

Start Date

June 12 2018

End Date

December 18 2020

Last Update

October 14 2022

Active Locations (7)

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Page 1 of 2 (7 locations)

1

Clinical Study Site

Aurora, Colorado, United States, 80045

2

Clinical Study Site

Lawrenceville, Georgia, United States, 30046

3

Clinical Study Site

Iowa City, Iowa, United States, 52242

4

Clinical Study Site

Baltimore, Maryland, United States, 21205