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

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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.

Study Overview

• Status: Terminated
• Conditions: Paroxysmal Nocturnal Hemoglobinuria; PNH
• Intervention / Treatment: Drug: BCX9930; Drug: Eculizumab; Drug: Ravulizumab

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 2

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria
    • Study Center
• South Africa
  • Bloemfontein, South Africa
    • Study Center
  • Cape Town, South Africa
    • Study Center
  • Pretoria, South Africa
    • Study Center
• United Kingdom
  • London, United Kingdom
    • Study Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: BCX9930; BCX9930 for oral administration
Experimental: 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.	Drug: BCX9930; BCX9930 for oral administration; Drug: Eculizumab; Administered at stable dose at the time of study entry; Drug: Ravulizumab; Administered at stable dose at the time of study entry

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Headache	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	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.	Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	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.	Baseline, Weeks 24, 48, 72, 96, and 120
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 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	Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events.	From first dose of study drug up to 3 weeks after last dose (Week 147)
Number of Blood Transfusions	Number of blood transfusions were reported.	From first dose of study drug up to 3 weeks after last dose (Week 147)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	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.	Baseline, Weeks 24, 48, 72, and 96

Collaborators and Investigators

• Sponsor: BioCryst Pharmaceuticals
• Investigators: Principal Investigator: Morag Griffin, MBChB, Leeds Teaching Hospitals NHS Trust, Leeds, UK

Study record dates

Study Major Dates

• Study Start (Actual): December 18, 2020
• Primary Completion (Actual): October 4, 2023
• Study Completion (Actual): October 4, 2023

Study Registration Dates

• First Submitted: January 7, 2021
• First Submitted That Met QC Criteria: January 8, 2021
• First Posted (Actual): January 11, 2021

Study Record Updates

• Last Update Posted (Actual): April 13, 2025
• Last Update Submitted That Met QC Criteria: April 3, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: PNH; Factor D inhibitor; BCX9930; paroxysmal Nocturnal Hemoglobinuria; oral treatment
• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urination Disorders; Urological Manifestations; Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Proteinuria; Hemoglobinuria; Hemoglobinuria, Paroxysmal; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Complement Inactivating Agents; Ravulizumab; Eculizumab
• Other Study ID Numbers: BCX9930-201; 2020-000501-93 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes