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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

28 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-01-18

Study Completion Date

2025-01-31

Brief Summary

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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.

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Detailed Description

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This was an open-label, non-randomized study to evaluate the long-term safety of BCX9930 in participants with PNH. PNH is an acquired, rare, serious, and potentially life-threatening disorder characterized by destruction of red blood cells (RBCs) resulting from uncontrolled activity of complement.

The participants in this study had previously benefited from BCX9930 in BioCryst studies BCX9930-201, BCX9930-202, and BCX9930-203. As the development program was not being discontinued for safety reasons or due to a lack of efficacy, and the preliminary data available from the ongoing clinical studies in PNH did not change the current safety or efficacy profile for BCX9930, BioCryst remained committed to providing BCX9930 to those participants who had participated in the ongoing studies who were currently receiving benefit from BCX9930 and wished to continue treatment.

This study was used to meet that commitment by allowing for continued access to treatment with BCX9930 for any clinical study participant with PNH currently receiving treatment with BCX9930. As the development of BCX9930 was terminated, treatment was provided for a maximum of 96 weeks, as long as the investigator believed it was in the participant's best interest to continue treatment, or until the participant had access to alternative therapy for PNH, whichever came first.

Conditions

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Paroxysmal Nocturnal Hemoglobinuria

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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BCX9930

Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg twice daily (BID) for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.

Group Type EXPERIMENTAL

BCX9930

Intervention Type DRUG

Taken orally at 400 mg BID

Interventions

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BCX9930

Taken orally at 400 mg BID

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male or non-pregnant, non-lactating female participants
* Were receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, had benefited from treatment with BCX9930 and would have benefited from continued treatment with BCX9930, and who did 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 have interfered with the participant's ability to participate in the study or increased 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, compromised the ability of the participant to continue study-specific procedures or it was considered not to be in the participant's best interest to continue, or benefit-risk assessment was no longer in favor of the participant's continued treatment

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No