A Phase III, Multicenter, Single-Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Pediatric Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
This study aims to evaluate the efficacy and safety of crovalimab in pediatric participants with aHUS.
• Body weight \>= 5 kg at screening.
• Vaccination against Neisseria meningitis serotypes A, C, W, and Y; vaccination against serotype B, according to national vaccination recommendations.
• Vaccination against Haemophilus influenzae type B and Streptococcus pneumoniae, according to national vaccination recommendations.
• For patients continuing to receive other therapies concomitantly with crovalimab (e.g., immunosuppressants, corticosteroids, mammalian target of rapamycin inhibitor (mTORi), or calcineurin inhibitors): stable dose for \>=28 days prior to screening and up to the first crovalimab administration.
• For female participants of childbearing potential: an agreement to remain abstinent or use contraception.
• Participants with a prior kidney transplant are eligible if they have a known history of complement-mediated aHUS prior to the kidney transplant.
• Onset of initial TMA presentation within 28 days prior to the first dose of crovalimab (for Naive Cohort only).
• Documented treatment with either eculizumab or ravulizumab (for Switch Cohort only).
• Clinical evidence of response to a C5 inhibitor (for Switch Cohort only).
• Poorly controlled TMA following treatment with another C5 inhibitor (for C5 SNP participants in the Pretreated Cohort only).
• Known C5 polymorphism (for C5 SNP participants in the Pretreated Cohort only).