Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)

Completed · Phase 3

Conditions studied: Atypical Hemolytic Uremic Syndrome (aHUS)

In brief

The purpose of the study is to assess the efficacy of ravulizumab to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor (complement inhibitor treatment-naïve), as well as in complement inhibitor-experienced (eculizumab-experienced) adolescent participants.

Key facts

Study ID

NCT03131219

Run by

Alexion Pharmaceuticals, Inc.

People needed

34

Starts

2017-08-31

Expected to finish

2022-12-20

Last updated by the study team

2024-03-15

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Complement Inhibitor Treatment Naïve:
• Participants from birth up to <18 years of age and weighing ≥5 kilograms (kg) at the time of consent.
• Participants had not been previously treated with complement inhibitors.
• Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
• Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae type b.
• Female participants of childbearing potential and male participants with female partners of childbearing potential must have used highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
• Eculizumab Experienced:
• Participants between 12 and <18 years of age (non-Japanese sites) or <18 years of age (Japanese sites) who had been treated with eculizumab according to the labelled dosing recommendation for aHUS for at least 90 days prior to screening.
• Participants with documented diagnosis of aHUS.
• Participants with clinical evidence of response to eculizumab indicated by stable TMA parameters at screening.
• Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae type b.
• Females of childbearing potential and male participants with female partners of childbearing potential must have used highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.

You may not qualify if…

• Known familial or acquired ADAMTS13 ("a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13") deficiency (activity <5%).
• Known Shiga toxin-related hemolytic uremic syndrome.
• Positive direct Coombs test.
• Females who planned to become pregnant during the study or were currently pregnancy or breastfeeding.
• Identified drug exposure-related hemolytic uremic syndrome.
• Bone marrow transplant/hematopoietic stem cell transplant within the last 6 months prior to the start of screening.
• Hemolytic uremic syndrome related to known genetic defects of cobalamin C metabolism.
• Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
• Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage kidney disease.
• For eculizumab-experienced participants, prior use of complement inhibitors other than eculizumab.
• For eculizumab-experienced participants, any known abnormal TMA parameters within 90 days prior to screening.

Where it is running

• Research Site — Hollywood, Florida, United States
• Research Site — Atlanta, Georgia, United States
• Research Site — Detroit, Michigan, United States
• Research Site — Omaha, Nebraska, United States
• Research Site — Hackensack, New Jersey, United States
• Research Site — Charlotte, North Carolina, United States
• Research Site — Brussels, Belgium
• Research Site — Heidelberg, Germany
• Research Site — Milan, Italy
• Research Site — Jeju-do, South Korea
• Research Site — Seoul, South Korea
• Research Site — Yangsan, South Korea
• Research Site — A Coruña, Spain
• Research Site — Barcelona, Spain
• Research Site — Esplugues de Llobregat, Spain
• Research Site — Valencia, Spain
• Research Site — Glasgow, United Kingdom
• Research Site — London, United Kingdom

Full record on ClinicalTrials.gov

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