Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS) (NCT03131219) | Clinical Trial Compass
CompletedPhase 3
Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
United States34 participantsStarted 2017-08-31
Plain-language summary
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.
Who can participate
SexALL
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Inclusion criteria
✓. 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.
✓. 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.
Exclusion criteria
✕. 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.
What they're measuring
1
Percentage Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26