BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

NCT05116787 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: BCX9930-2032020-004403-14
• Study Type: interventional
• Target: 12
• Locations: 3 countries
• Sites: 5

Brief Summary

The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult participants with PNH not currently receiving complement inhibitor therapy.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Keywords

BCX9930; factor D inhibitor; proximal complement inhibitor; oral therapy; paroxysmal nocturnal hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Part 1: Change From Baseline in Hemoglobin at Week 12

  Baseline, Week 12

Secondary Outcomes (15)

• Part 1: Number of Participants Who Were Transfusion-free

  From Week 4 to Week 12

• Part 1: Number of Units of pRBCs Transfused

  From Week 4 to Week 12

• Part 1: Percent Change From Baseline in Lactate Dehydrogenase

  Baseline, Week 12

• Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

  Baseline, Week 12

• Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused

  Prestudy (12 months prior to screening) and from Week 4 to Week 12

Study Arms (2)

BCX9930/BCX9930

EXPERIMENTAL

Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. Initially participants were to receive BCX9930 500 mg twice daily (BID) orally. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days.

Drug: BCX9930 monotherapy

Placebo/BCX9930

PLACEBO COMPARATOR

Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days.

Drug: Placebo; Drug: BCX9930 monotherapy

Interventions

Placebo DRUG

Administered orally twice daily

Placebo/BCX9930

BCX9930 monotherapy DRUG

Administered orally twice daily

BCX9930/BCX9930; Placebo/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 female, aged ≥ 18 years old
• Body weight ≥ 40 kg
• Documented diagnosis of PNH
• No complement inhibitor therapy for ≥ 12 months prior to screening
• Contraindication to or no access to approved (C3 or C5) complement inhibitor therapies
• Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series
• At screening: PNH clone of ≥ 10%, hemoglobin ≤ 10.5 g/dL and lactate dehydrogenase ≥ 2 × upper limit of normal

You may not qualify if:

• Known history of or existing diagnosis of hereditary complement deficiency
• History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation
• Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition
• History of malignancy within 5 years prior to the screening visit
• Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening
• Treatment with anti-thymocyte globulin within 180 days prior to screening
• Initiation of treatment with an erythrocyte or platelet growth factor, or danazol within 28 days prior to screening
• Receiving iron supplementation with an unstable dose in the 28 days prior to screening

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (5)

Investigative Site

Ampang, Malaysia

Location

Investigative Site

Bloemfontein, South Africa

Location

Investigative Site

Cape Town, South Africa

Location

Investigative Site

Pretoria, South Africa

Location

InvestigativeSite

Daejeon, South Korea

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 terminate the development program (including this study).

Results Point of Contact

• Title: Study Director
• Organization: BioCryst Pharmaceuticals Inc

clinicaltrials@biocryst.com

+1 919-859-1302

Study Officials

• David J Kuter, MD, DPhil

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2021
• First Posted: November 11, 2021
• Study Start: October 26, 2021
• Primary Completion: September 18, 2023
• Study Completion: September 18, 2023
• Last Updated: January 3, 2025
• Results First Posted: January 3, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1); MY (1); ZA (3)