A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
Condition: Paroxysmal Nocturnal Hemoglobinuria
MeSH terms: Hemoglobinuria; Hemoglobinuria, Paroxysmal
Intervention: Ravulizumab
Intervention description: Single intravenous (IV) loading dose on Day 1, followed by regular IV maintenance dosing beginning on Day 15, based on weight.
Intervention MeSH terms: Ravulizumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 13.0
Sex: All
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Ravulizumab; ALXN1210; Ultomiris; Pharmacokinetics; Pharmacodynamics

Summary:

      The purpose of this study was to assess the pharmacokinetics (PK), pharmacodynamics (PD),
      safety, and efficacy of ravulizumab in pediatric participants with paroxysmal nocturnal
      hemoglobinuria (PNH).
    

Eligibility criteria:

        Inclusion Criteria:

          1. Participants from birth up to <18 years of age and weighing ≥ 5 kilograms at the time
             of consent.

          2. PNH diagnosis confirmed by documented high-sensitivity flow cytometry.

          3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months
             of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea),
             anemia, history of a major adverse vascular event (including thrombosis), dysphagia,
             or erectile dysfunction; or history of packed red blood cell transfusion due to PNH.

          4. Lactate dehydrogenase (LDH) level ≥ 1.5 × upper limit of normal (ULN) for participants
             not being treated with eculizumab at screening and LDH level ≤ 1.5 × ULN for
             participants taking eculizumab.

          5. Documented meningococcal vaccination not more than 3 years prior to dosing, and
             vaccination against Streptococcus pneumoniae and Haemophilus influenzae.

          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.

        Exclusion Criteria:

          1. History of bone marrow transplantation.

          2. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease
             that, in the opinion of the investigator or sponsor, would preclude participation.

          3. Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal
             bleed, severe congestive heart failure, anticipated need for major surgery within 6
             months of randomization, coexisting chronic anemia unrelated to PNH).

          4. Females who are pregnant or breastfeeding or who have a positive pregnancy test at
             screening or Day 1.

          5. Participation in another interventional clinical study or use of any experimental
             therapy within 30 days before initiation of study drug on Day 1 in this study or
             within 5 half-lives of that investigational product, whichever is greater.