Status:

TERMINATED

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

Lead Sponsor:

BioCryst Pharmaceuticals

Conditions:

Paroxysmal Nocturnal Hemoglobinuria

PNH

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief 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...

Eligibility Criteria

Inclusion

  • Male or non-pregnant, non-lactating female participants
  • Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH

Exclusion

  • Apart from a diagnosis of PNH, any clinically significant medical or psychiatric condition or medical history, other than those associated with PNH disease, that, in the opinion of the Investigator or Sponsor, would interfere with the participant's ability to participate in the study or participation would increase the risk for that participant
  • Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating
  • Note: Other protocol-defined Inclusion/Exclusion criteria may apply

Key Trial Info

Start Date :

December 18 2020

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

October 4 2023

Estimated Enrollment :

19 Patients enrolled

Trial Details

Trial ID

NCT04702568

Start Date

December 18 2020

End Date

October 4 2023

Last Update

April 13 2025

Active Locations (5)

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Page 1 of 2 (5 locations)

1

Study Center

Vienna, Austria

2

Study Center

Bloemfontein, South Africa

3

Study Center

Cape Town, South Africa

4

Study Center

Pretoria, South Africa