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A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT04702568 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2025-04-13

Study results available
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Summary

This study was designed to evaluate the long-term safety of daily oral treatment with BCX9930 in participants who had participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allowed continued access to BCX9930 for enrolled participants. The study also evaluated the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the participant's satisfaction with the medication.

Conditions

Interventions

DRUG

BCX9930

BCX9930 for oral administration

DRUG

Eculizumab

Administered at stable dose at the time of study entry

DRUG

Ravulizumab

Administered at stable dose at the time of study entry

Sponsors & Collaborators

  • BioCryst Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Morag Griffin, MBChB · Leeds Teaching Hospitals NHS Trust, Leeds, UK

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-18
Primary Completion
2023-10-04
Completion
2023-10-04
FDA Drug
Yes

Countries

  • Austria
  • South Africa
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
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 NCT04702568 on ClinicalTrials.gov