Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
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.
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).