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

Sponsor

BioCryst Pharmaceuticals (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05741346

Collaborator

(none)

Enrollment

Locations

Arm

36.5

Anticipated Duration (Months)

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is designed to provide continued access to BCX9930 for subjects 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.

Condition or Disease	Intervention/Treatment	Phase
Paroxysmal Nocturnal Hemoglobinuria	Drug: BCX9930	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst-sponsored Study

Actual Study Start Date :

Jan 18, 2023

Anticipated Primary Completion Date :

Feb 1, 2026

Anticipated Study Completion Date :

Feb 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: BCX9930 All subjects receive BCX9930 for 96 weeks	Drug: BCX9930 Taken orally at 400 mg twice daily (BID)

Arm

Intervention/Treatment

Experimental: BCX9930

All subjects receive BCX9930 for 96 weeks

Drug: BCX9930

Taken orally at 400 mg twice daily (BID)

Outcome Measures

Primary Outcome Measures

Proportion of subjects with treatment emergent adverse events (TEAEs) [Week 96]
Proportion of subjects with TEAEs. Safety assessments include graded TEAEs / serious TEAEs, lab abnormalities, changes to vital signs, and physical examination findings.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male or non-pregnant, non-lactating female subjects
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 subject's ability to participate in the study or increase the risk of participation for that subject
An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the subject to continue study-specific procedures or it is considered not to be in the subject's best interest to continue or benefit-risk assessment is no longer in favor of the subject's continued treatment