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

NCT04702568 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: BCX9930-2012020-000501-93
• Study Type: interventional
• Target: 19
• Locations: 3 countries
• Sites: 5

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria; PNH

Keywords

BCX9930; Factor D inhibitor; PNH; paroxysmal Nocturnal Hemoglobinuria; oral treatment

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs)

  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.

  From first dose of study drug up to 3 weeks after last dose (Week 147)

Secondary Outcomes (16)

• Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue

  Baseline, Weeks 24, 48, 72, 96, and 120

• Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea

  Baseline, Weeks 24, 48, 72, 96, and 120

• Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort

  Baseline, Weeks 24, 48, 72, 96, and 120

• Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing

  Baseline, Weeks 24, 48, 72, 96, and 120

• Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain

  Baseline, Weeks 24, 48, 72, 96, and 120

Study Arms (2)

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

EXPERIMENTAL

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.

Drug: BCX9930

C5 INH Inadequate Response Group

EXPERIMENTAL

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.

Drug: BCX9930; Drug: Eculizumab; Drug: Ravulizumab

Interventions

BCX9930 DRUG

BCX9930 for oral administration

C5 INH Inadequate Response Group; Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group

Eculizumab DRUG

Administered at stable dose at the time of study entry

C5 INH Inadequate Response Group

Ravulizumab DRUG

Administered at stable dose at the time of study entry

C5 INH Inadequate Response Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (5)

Study Center

Vienna, Austria

Location

Study Center

Bloemfontein, South Africa

Location

Study Center

Cape Town, South Africa

Location

Study Center

Pretoria, South Africa

Location

Study Center

London, United Kingdom

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab; ravulizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Limitations and Caveats

The sponsor decided to prematurely terminate the study due to business reasons. Per change in planned analysis, data were analyzed and reported for safety and selected efficacy parameters (assessment of various clinical PNH symptoms severity, incidence of thromboembolic events, change from baseline in clinical measurements of PNH \[LDH, hemoglobin, haptoglobin, reticulocytes, transfusion requirements\], and change from baseline in FACIT\]).

Results Point of Contact

• Title: Study Director
• Organization: BioCryst Pharmaceuticals Inc

clinicaltrials@biocryst.com

+1 919-859-1302

Study Officials

• Morag Griffin, MBChB

  Leeds Teaching Hospitals NHS Trust, Leeds, UK

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2021
• First Posted: January 11, 2021
• Study Start: December 18, 2020
• Primary Completion: October 4, 2023
• Study Completion: October 4, 2023
• Last Updated: April 13, 2025
• Results First Posted: December 13, 2024

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); ZA (3); GB (1)