Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria

NCT05741346 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: BCX9930-2052021-006776-17
• Study Type: interventional
• Target: 28
• Locations: 7 countries
• Sites: 11

Brief Summary

This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the opinion of the investigator, would benefit from continued treatment with BCX9930; who did not have access to other effective treatment options; and to monitor the safety of BCX9930 in participants continuing to receive BCX9930 for the treatment of PNH.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

BCX9930; Factor D inhibitor; Proximal complement inhibitor; Oral therapy; Paroxysmal nocturnal hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-emergent Events (TEAEs)

  An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study intervention or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important event. An AE is considered treatment emergent if its start date was on or after the date of first dose of study treatment in Study 205 or if the AE was ongoing from the prior study. TEAEs included both serious TEAEs and non-serious TEAEs.

  From Day 1 up to 30 days after last dose (up to approximately 100 weeks)

Study Arms (1)

BCX9930

EXPERIMENTAL

Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg twice daily (BID) for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.

Drug: BCX9930

Interventions

BCX9930 DRUG

Taken orally at 400 mg BID

BCX9930

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
• Were receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, had benefited from treatment with BCX9930 and would have benefited from continued treatment with BCX9930, and who did not have access to other treatment options

You may not qualify if:

• Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would have interfered with the participant's ability to participate in the study or increased the risk of participation for that participant
• An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromised the ability of the participant to continue study-specific procedures or it was considered not to be in the participant's best interest to continue, or benefit-risk assessment was no longer in favor of the participant's continued treatment

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (11)

Investigative Site

Paris, France

Location

Investigative Site

Budapest, Hungary

Location

Investigative Site

Ampang, Malaysia

Location

Investigative Site

Bloemfontein, South Africa

Location

Investigative Site

Cape Town, South Africa

Location

Investigative Site

Pretoria, South Africa

Location

Investigative Site

Daejeon, South Korea

Location

Investigative Site

Barcelona, Spain

Location

Investigative Site

Valencia, Spain

Location

Investigative Site

Leeds, United Kingdom

Location

Investigative Site

London, United Kingdom

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

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. The decision to stop the development of BCX9930 was not due to safety reasons or due to lack of efficacy.

Results Point of Contact

• Title: Study Director
• Organization: BioCryst Pharmaceuticals Inc.

clinicaltrials@biocryst.com

+001 919859 1302

Study Officials

• Phillip Scheinberg, MD, PhD

  Beneficência Portuguesa de São Paulo

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 14, 2023
• First Posted: February 23, 2023
• Study Start: January 18, 2023
• Primary Completion: January 31, 2025
• Study Completion: January 31, 2025
• Last Updated: October 24, 2025
• Results First Posted: October 24, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); KR (1); HU (1); MY (1); GB (2); ZA (3); ES (2)