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Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria

NCT05741346 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 28

Last updated 2025-10-24

Study results available
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Summary

This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the opinion of the investigator, would benefit from continued treatment with BCX9930; who did not have access to other effective treatment options; and to monitor the safety of BCX9930 in participants continuing to receive BCX9930 for the treatment of PNH.

Conditions

Interventions

DRUG

BCX9930

Taken orally at 400 mg BID

Sponsors & Collaborators

  • BioCryst Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Phillip Scheinberg, MD, PhD · Beneficência Portuguesa de São Paulo

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-01-18
Primary Completion
2025-01-31
Completion
2025-01-31
FDA Drug
Yes

Countries

  • France
  • Hungary
  • Malaysia
  • South Africa
  • South Korea
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

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 NCT05741346 on ClinicalTrials.gov