A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

20150168

This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.

2019-01-22

2022-07-12

2022-07-12

Completed

Early phase I

42

Inclusion Criteria: * Men and women ≥ 18 years of age. * Historical diagnosis of PNH. * Administration of eculizumab for ≥ 6 months and currently receiving 900 mg of eculizumab. * Hemoglobin ≥ 9.0 g/dL for at least 6 weeks before randomization. * Lactate dehydrogenase \< 1.5 × the upper limit of normal at screening. * Platelet count ≥ 50 × 10\^9/L. * Absolute neutrophil count (ANC) ≥ 0.5 x 10\^9/L (500/μL). * Participants must be vaccinated against Neisseria meningitidis. * Participants must sign an IRB/IEC-approved ICF before participation in any procedures. Exclusion Criteria: * Known or suspected hereditary complement deficiency. * Clinically significant cardiovascular disease (including myocardial infarction, unstable angina, symptomatic congestive heart failure \[New York Heart Association ≥ Class III\], serious uncontrolled cardiac arrhythmia), peripheral vascular disease, cerebrovascular accident, or transient ischemic attack in the previous 6 months. * Evidence of acute thrombosis (liver Doppler ultrasound of hepatic and portal veins). * Known to be positive for human immunodeficiency virus. * Woman who is pregnant or breastfeeding. * Participant is currently enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(s), or participant is receiving other investigational agent(s). * Participant has known sensitivity to any of the products to be administered during the study, including mammalian cell-derived drug products. * History of meningococcal infection. * Presence or suspicion of active bacterial infection, or recurrent bacterial infection. * History of bone marrow transplantation. * Red blood cell transfusion required within 12 weeks before randomization. * Participant experienced ≥ 2 breakthrough events, (ie, signs and symptoms of intravascular hemolysis, that require dose and/or schedule adjustments of eculizumab) in the previous 12 months before screening.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'CROSSOVER', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'maskingDescription': 'double-blind', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 42, 'type': 'ACTUAL'}}

2023-05-23