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BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

NCT05116774 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-03-07

Study results available
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Summary

The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH participants with residual anemia despite treatment with a C5 inhibitor.

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

DRUG

Eculizumab

Administered by intravenous infusion per current dose regimen

DRUG

Ravulizumab

Administered by intravenous infusion per current dose regimen

DRUG

BCX9930

Administered orally twice daily

Sponsors & Collaborators

  • BioCryst Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Austin G Kulasekararaj, MBBS, MD · King's College Hospital NHS Trust

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-06
Primary Completion
2023-09-14
Completion
2023-09-14
FDA Drug
Yes

Countries

  • France
  • Hungary
  • Italy
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05116774 on ClinicalTrials.gov