Myelodysplastic Syndrome Clinical Trial
BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy
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.
This is a randomized, placebo-controlled, double-blind, parallel-group, 2-part study. Parts 1 and 2 will be conducted in the same subjects.
Part 1 of the study is designed to evaluate the efficacy, safety, and tolerability of treatment with oral BCX9930 monotherapy for 12 weeks versus placebo in subjects with PNH who are not currently receiving treatment with complement inhibitor therapy. Subjects will be randomized to receive BCX9930 or placebo under double blind conditions for the 12-week randomized treatment period. The primary efficacy and safety analyses will be based on Part 1.
Part 2 of the study is designed to evaluate the long-term safety, tolerability, and effectiveness of open-label BCX9930 monotherapy when administered through Week 52. All subjects in Part 2 will receive BCX9930. Subjects who are randomized to BCX9930 monotherapy in Part 1 will continue to receive BCX9930 in Part 2. Subjects who are randomized to placebo in Part 1 will discontinue that therapy at the Week 12 visit and receive BCX9930 in Part 2.
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
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
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There are 3 Locations for this study
Boston Massachusetts, 02114, United States
Ampang , , Malaysia
Bloemfontein , 9301, South Africa
Cape Town , , South Africa
Pretoria , 0044, South Africa
Barcelona , , Spain
Taipei , , Taiwan
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