Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT06100900 | Completed Phase 1 DMC

• 1 other identifier: BCX10013-105
• Study Type: interventional
• Target: 8
• Locations: 2 countries
• Sites: 4

Brief Summary

This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH. Approximately 8 participants will be enrolled in this study. Participants may receive treatment for up to 52 weeks.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Paroxysmal Nocturnal Hemoglobinuria; Factor D; complement inhibitor; alternative pathway inhibitor; BioCryst

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings.

  up to 52 weeks

Secondary Outcomes (9)

• CFB in Lactate Dehydrogenase

  Baseline, Week 52

• CFB in the Ratio of Total PNH Red Blood Cell Clone Size to PNH White Blood Cell Clone Size

  Baseline, Week 52

• CFB in Hemoglobin

  Baseline, Week 52

• Percentage of Participants who are Transfusion-free

  52 weeks

• Percentage of Participants Achieving a Within-subject Clinically Meaningful CFB in the FACIT-Fatigue scale

  52 weeks

Study Arms (1)

BCX10013

EXPERIMENTAL

Participants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur.

Drug: BCX10013

Interventions

BCX10013 DRUG

Multiple dose levels may be tested in this study.

BCX10013

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 adults ≥ 18 years old.
• Documented diagnosis of PNH confirmed by flow cytometry.
• Body mass index (BMI) ≤ 40 kg/m\^2.
• Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with ravulizumab for at least 12 months prior to the screening visit and have received no treatment with eculizumab or pegcetacoplan for 6 months prior to the screening visit.
• Documentation of current vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae type B \[Hib\] or willingness to start vaccination series at least 14 days prior to Day 1.

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 during the study.
• Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis.
• History of malignancy within 5 years prior to the screening visit.
• Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
• Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit.
• Receiving iron with an unstable dose (ie, increasing or decreasing) in the 28 days prior to the screening visit.

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (4)

BioCryst Investigative Site

Ampang, Malaysia

Location

BioCryst Investigative Site

Bloemfontein, South Africa

Location

BioCryst Investigative Site

Cape Town, South Africa

Location

BioCryst Investigative Site

Pretoria, South Africa

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Multiple dose levels of BCX10013 are planned for escalation.

Study Record Dates

• First Submitted: October 20, 2023
• First Posted: October 25, 2023
• Study Start: October 24, 2023
• Primary Completion: December 11, 2024
• Study Completion: December 11, 2024
• Last Updated: January 8, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

MY (1); ZA (3)