BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy

• Status: Recruiting
• Trial end date: 2023-08-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: BioCryst Pharmaceuticals

Criteria

Inclusion Criteria:

• 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

• 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

Exclusion Criteria:

• 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