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BCX9930

DRUG5 trials

Sponsors

BioCryst Pharmaceuticals

Conditions

Complement 3 GlomerulopathyImmunoglobulin A NephropathyMembranous NephropathyPNHParoxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria (PNH)

Phase 1

First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH
CompletedNCT04330534
BioCryst PharmaceuticalsParoxysmal Nocturnal Hemoglobinuria
Start: 2020-03-03End: 2021-01-25Updated: 2022-02-17

Phase 2

A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
TerminatedNCT04702568
BioCryst PharmaceuticalsParoxysmal Nocturnal Hemoglobinuria, PNH
Start: 2020-12-18End: 2023-10-04Updated: 2025-04-13
BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy
TerminatedNCT05116774
BioCryst PharmaceuticalsParoxysmal Nocturnal Hemoglobinuria (PNH)
Start: 2021-12-06End: 2023-09-14Updated: 2025-03-07
Study to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN)
TerminatedNCT05162066
BioCryst PharmaceuticalsComplement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Membranous Nephropathy
Start: 2022-02-18End: 2022-09-23Updated: 2024-05-02
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
TerminatedNCT05741346
BioCryst PharmaceuticalsParoxysmal Nocturnal Hemoglobinuria
Start: 2023-01-18End: 2025-01-31Updated: 2025-10-24

Related Papers

Clinical study outcomes in IgA nephropathy: A systematic literature review and narrative synthesis.
2025-06-101 citations
Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial.
2022-08-0249 citations
P826: FACTOR D INHIBITION WITH ORAL BCX9930 LEADS TO SUSTAINED CONTROL OF HEMOLYSIS AND SYMPTOMS OVER 48 WEEKS IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA INADEQUATELY CONTROLLED ON C5 INHIBITORS
HemaSphere2022-06-016 citations
POS-045 Evaluating BCX9930, an Oral Factor D Inhibitor for Treatment of Complement-Mediated Kidney Disease: A Proof-of-Concept Study (RENEW)
Kidney International Reports2022-06-016 citations
P825: FACTOR D INHIBITION WITH ORAL BCX9930 MONOTHERAPY LEADS TO SUSTAINED CONTROL OF HEMOLYSIS AND SYMPTOMS OVER 48 WEEKS IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA NAÏVE TO C5 INHIBITORS
HemaSphere2022-06-014 citations
POS-040 BCX9930, AN ORAL FACTOR D INHIBITOR IN DEVELOPMENT FOR THE TREATMENT OF COMPLEMENT-MEDIATED KIDNEY DISEASES, INHIBITS COMPLEMENT ALTERNATIVE PATHWAY ACTIVITY IN HEALTHY SUBJECTS
Kidney International Reports2022-06-01
Preclinical Characterization of BCX9930, a Potent Oral Complement Factor D Inhibitor, Targeting Alternative Pathway-Mediated Diseases Including Paroxysmal Nocturnal Hemoglobinuria (PNH)
Blood2020-11-053 citations
BCX9930, an Oral Factor D Inhibitor, for the Potential Treatment of Alternative Pathway Mediated Diseases: Interim Results of a Phase 1 Study in Healthy Subjects
Blood2020-11-044 citations
BCX9930, a Potent, Selective, Oral Factor D Inhibitor, Demonstrates Proof-of-Concept As Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
Blood2020-11-044 citations