Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Condition: Atypical Hemolytic Uremic Syndrome (aHUS)
MeSH terms: Azotemia; Hemolytic-Uremic Syndrome; Atypical Hemolytic Uremic Syndrome; Syndrome; Hemolysis
Intervention: Ravulizumab
Intervention description: Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight: ≥ 40 to < 60 kilograms (kg), 2400 milligrams (mg) loading, then 3000 mg every 8 weeks; ≥ 60 to < 100 kg, 2700 mg loading, then 3300 mg every 8 weeks; ≥ 100 kg, 3000 mg loading, then 3600 mg every 8 weeks.
Intervention MeSH terms: Ravulizumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 58.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No

Summary:

      The purpose of the study is to assess the safety and efficacy of ravulizumab to control
      disease activity in adolescent and adult participants with aHUS who had not previously used a
      complement inhibitor.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Male or female ≥ 12 years of age and weighing ≥ 40 kg at the time of consent.

          2. Evidence of thrombotic microangiopathy, including low platelet count, hemolysis
             (breaking of red blood cells inside of blood vessels), and decreased kidney function.

          3. Documented meningococcal vaccination not more than 3 years prior to, or at the time
             of, initiating study drug. Participants who received a meningococcal vaccine less than
             2 weeks before initiating ravulizumab treatment must have received treatment with
             appropriate prophylactic antibiotics until 2 weeks after vaccination. Participants who
             had not been vaccinated prior to initiating ravulizumab treatment should have received
             prophylactic antibiotics prior to and for at least 2 weeks after meningococcal
             vaccination. Participants < 18 years of age must have been vaccinated against
             haemophilus influenzae type b and streptococcus pneumoniae according to national and
             local vaccination schedule guidelines.

          4. Female participants of childbearing potential and male participants with female
             partners of childbearing potential had to use highly effective contraception starting
             at screening and continuing until at least 8 months after the last dose of
             ravulizumab.

        Exclusion Criteria:

          1. A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13
             deficiency (activity < 5%).

          2. Shiga toxin-related hemolytic uremic syndrome.

          3. Positive direct Coombs test.

          4. Pregnancy or breastfeeding.

          5. Identified drug exposure-related hemolytic uremic syndrome (HUS).

          6. Bone marrow transplant/hematopoietic stem cell transplant within last 6 months prior
             to start of Screening.

          7. HUS related to known genetic defects of cobalamin C metabolism.

          8. Systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid
             antibody positivity or syndrome.

          9. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy
             for end-stage kidney disease).