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

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-12-18

Study Completion Date

2023-10-04

Brief Summary

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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.

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Detailed Description

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Conditions

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Paroxysmal Nocturnal Hemoglobinuria PNH

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group

This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis.

Group Type EXPERIMENTAL

BCX9930

Intervention Type DRUG

BCX9930 for oral administration

C5 INH Inadequate Response Group

This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy.

Group Type EXPERIMENTAL

BCX9930

Intervention Type DRUG

BCX9930 for oral administration

Eculizumab

Intervention Type DRUG

Administered at stable dose at the time of study entry

Ravulizumab

Intervention Type DRUG

Administered at stable dose at the time of study entry

Interventions

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BCX9930

BCX9930 for oral administration

Intervention Type DRUG

Eculizumab

Administered at stable dose at the time of study entry

Intervention Type DRUG

Ravulizumab

Administered at stable dose at the time of study entry

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* 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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No