Evaluating oral BCX9930 in renal disease
Research type
Research Study
Full title
An Open-Label, Safety, Tolerability, and Proof-of-Concept Study of Oral BCX9930 Therapy in Subjects with Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, or Primary Membranous Nephropathy
IRAS ID
297898
Contact name
Kathryn Hutchinson
Contact email
Sponsor organisation
BioCryst Pharmaceuticals Inc
Eudract number
2020-005855-19
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Summary of research
The study will assess the efficacy and safety of daily oral treatment of BCX9930 in three different renal diseases.
BCX9930 is a Factor D inhibitor. Factor D is the rate limiting enzyme in the alternative pathway of the complement cascade.
The study will assess participants with either C3G (Complement 3 Glomerulopathy), or IgA Nephropathy or PMN (Primary Membranous Nephropathy). All three diseases have been associated with alterations in the complement pathway, have limited treatment options and will eventually lead to ESRD.
The study is open label, and will treat approximately 14 participants in each of the three cohorts. Participants will be treated for 24 weeks, as chronic renal indications typically require long treatment periods to see an improvement in renal function.Summary of Results
In December 2022, BioCryst stopped development of BCX9930 based on changes in the competitive environment. Therefore, this study was prematurely discontinued. A total of 3 subjects were screened and 2 subjects were administered BCX9930.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
21/EM/0182
Date of REC Opinion
13 Aug 2021
REC opinion
Further Information Favourable Opinion