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

Phase 2

Terminated

• Conditions: PNH; Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: BCX9930; Drug: Eculizumab; Drug: Ravulizumab

• Registration Number: NCT04702568
• Lead Sponsor: BioCryst Pharmaceuticals

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-12-18
• Study First Submitted: 2021-01-07
• Study First Submitted QC: 2021-01-08
• Study First Posted: 2021-01-11
• Primary Completion: 2023-10-04
• Study Completion: 2023-10-04
• Results First Submitted: 2024-10-03
• Status Verified: 2024-12
• Results First Submitted QC: 2024-12-11
• Last Update Submitted: 2024-12-11
• Results First Posted: 2024-12-13
• Last Update Posted: 2024-12-13

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 19

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group	BCX9930	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.
C5 INH Inadequate Response Group	BCX9930	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.
C5 INH Inadequate Response Group	Eculizumab	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.
C5 INH Inadequate Response Group	Ravulizumab	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	From first dose of study drug up to 3 weeks after last dose (Week 147)	An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of fatigue. The severity of clinical PNH symptom of fatigue was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of Chest pain/discomfort. The severity of clinical PNH symptom of chest pain/discomfort was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of abdominal pain. The severity of clinical PNH symptom of abdominal pain was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of Dyspnea. The severity of clinical PNH symptom of Dyspnea was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of difficulty swallowing. The severity of clinical PNH symptom of difficulty swallowing was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of headache. The severity of clinical PNH symptom of headache was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of erectile dysfunction. The severity of clinical PNH symptom of erectile dysfunction was graded as none, mild, moderate and severe based solely on investigator's discretion.
Change From Baseline in Haptoglobin	Baseline, Weeks 24, 48, 72, 96, and 120
Change From Baseline in Reticulocytes	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Blood Transfusions or Thromboses	From first dose of study drug up to 3 weeks after last dose (Week 147)	Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events.
Number of Blood Transfusions	From first dose of study drug up to 3 weeks after last dose (Week 147)	Number of blood transfusions were reported.
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of Hemoglobinuria.The severity of clinical PNH symptom of hemoglobinuria was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of jaundice. The severity of clinical PNH symptom of jaundice was graded as none, mild, moderate and severe based solely on investigator's discretion.
Change From Baseline in Lactate Dehydrogenase (LDH)	Baseline, Weeks, 24, 48, 72, 96, 120, and 144
Change From Baseline in Hemoglobin	Baseline, Weeks 24, 48, 72, 96, 120, and 144
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Baseline, Weeks 24, 48, 72, and 96	The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.

Trial Locations

Locations (1)

Study Center; 🇬🇧 London, United Kingdom