ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
MeSH terms: Hemoglobinuria; Hemoglobinuria, Paroxysmal
Intervention: Ravulizumab; Eculizumab
Intervention description: All treatments were given as intravenous (IV) infusions. For participants weighing ≥40 to <60 kilograms (kg): 2400 mg was given as a single loading dose, followed by 3000 mg as maintenance dose. For participants weighing ≥60 to <100 kg: 2700 mg was given as a loading dose, followed by 3300 mg as maintenance dose. For participants weighing ≥100 kg: 3000 mg was given as a loading dose, followed by 3600 mg as maintenance dose.; All treatments were given as IV infusions. Participants received 900 mg of eculizumab q2w.
Intervention MeSH terms: Eculizumab; Ravulizumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 195.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      The primary purpose of this study was to assess the noninferiority of ravulizumab compared to
      eculizumab in adult participants with PNH who were clinically stable after having been
      treated with eculizumab for at least 6 months.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Male or female ≥18 years of age.

          2. Treated with eculizumab for PNH for at least 6 months prior to Day 1.

          3. Lactate dehydrogenase level ≤1.5 times the upper limit of normal (ULN) at screening.

          4. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry.

          5. Documented meningococcal vaccination not more than 3 years prior to, or at the time
             of, initiating study treatment.

          6. Female participants of childbearing potential must use highly effective contraception
             starting at screening and continuing until at least 8 months after the last dose of
             ravulizumab.

          7. Willing and able to give written informed consent and comply with study visit
             schedule.

        Exclusion Criteria:

          1. History of bone marrow transplantation.

          2. Body weight <40 kilograms at screening.

          3. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease
             that, in the opinion of the investigator or sponsor, would preclude participation.

          4. Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal
             bleeding, severe congestive heart failure, anticipated need for major surgery within 6
             months of randomization, or coexisting chronic anemia unrelated to PNH).

          5. Female participants who are pregnant, breastfeeding, or who have a positive pregnancy
             test at screening or Day 1.

          6. Participation in another interventional clinical study or use of any experimental
             therapy within 30 days before initiation of study treatment on Day 1 in this study or
             within 5 half-lives of that investigational product, whichever is greater.