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

BioCryst

Status and phase: Terminated; Phase 2

Conditions: PNH; Paroxysmal Nocturnal Hemoglobinuria

Treatments: Drug: BCX9930; Drug: Eculizumab; Drug: Ravulizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT04702568

2020-000501-93 (EudraCT Number)

BCX9930-201

Details and patient eligibility

About

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.

Enrollment: 19 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Note: Other protocol-defined Inclusion/Exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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19 participants in 2 patient groups

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

Experimental group

Description:

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.

Treatment:

Drug: BCX9930

C5 INH Inadequate Response Group

Experimental group

Description:

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.

Treatment:

Drug: Ravulizumab

Drug: Eculizumab

Drug: BCX9930