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BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

NCT05116787 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-01-03

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 adult participants with PNH not currently receiving complement inhibitor therapy.

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

DRUG

Placebo

Administered orally twice daily

DRUG

BCX9930 monotherapy

Administered orally twice daily

Sponsors & Collaborators

  • BioCryst Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • David J Kuter, MD, DPhil · Massachusetts General Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-26
Primary Completion
2023-09-18
Completion
2023-09-18
FDA Drug
Yes

Countries

  • Malaysia
  • South Africa
  • South Korea

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 NCT05116787 on ClinicalTrials.gov