A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
Condition: Paroxysmal Nocturnal Hemoglobinuria
MeSH terms: Hemoglobinuria; Hemoglobinuria, Paroxysmal
Intervention: ABP 959; Eculizumab
Intervention description: intravenous infusion; intravenous infusion
Intervention MeSH terms: Eculizumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking description: double-blind
Enrollment: 42.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Marchiafava-Micheli Syndrome; Paroxysmal Cold Hemoglobinuria

Summary:

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

Eligibility criteria:

        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.