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

Phase 2

Active, not recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: BCX9930

• Registration Number: NCT05741346
• Lead Sponsor: BioCryst Pharmaceuticals

Brief Summary

This study is designed to provide continued access to BCX9930 for participants currently receiving treatment with BCX9930 in a BioCryst-sponsored clinical study for paroxysmal nocturnal hemoglobinuria (PNH) who, in the opinion of the investigator, would benefit from continued treatment with BCX9930 and who do not have access to other treatment options.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-01-18
• Study First Submitted: 2023-02-14
• Study First Submitted QC: 2023-02-14
• Study First Posted: 2023-02-23
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-26
• Last Update Posted: 2024-07-29
• Primary Completion: 2026-02
• Study Completion: 2026-02

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 28

Inclusion Criteria

• Male or non-pregnant, non-lactating female participants
• Are receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, have benefited from treatment with BCX9930 and would benefit from continued treatment with BCX9930, and who do not have access to other treatment options

Exclusion Criteria

• Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant
• An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the participant to continue study-specific procedures or it is considered not to be in the participant's best interest to continue or benefit-risk assessment is no longer in favor of the participant's continued treatment

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BCX9930	BCX9930	All subjects receive BCX9930 for 96 weeks

Primary Outcome Measures

Name	Time	Method
Proportion of Participants with Treatment Emergent Adverse Events (TEAEs)	Week 96	Proportion of participants with TEAEs. Safety assessments include graded TEAEs / serious TEAEs, lab abnormalities, changes to vital signs, and physical examination findings.

Trial Locations

Locations (1)

Investigative Site; 🇬🇧 London, United Kingdom