A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Conditions

• Atypical Hemolytic Uremic Syndrome

Eligibility Criteria

Sex

ALL

Age

28 Days - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Crovalimab — DRUG
  Crovalimab will be administered at a dose of 1000 mg intravenously (IV) (for participants weighing =\> 40 to \<100 kg) or 1500 mg IV (for participants weighing \>=100 kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, crovalimab will be administered at a dose of 340 mg subcutaneously (SC). On Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants weighing =\> 40 to \<100 kg) or 1020 mg SC (for participants weighing \>=100 kg). Enrollment of participants weighing \<40 kg will be staggered using two weight-based dose confirmation groups (Group 1 participants weighing \>=20 kg to \<40 kg, followed by Group 2 participants weighing \>=5 kg to \<20 kg). All participants will receive an initial IV loading dose, which will be followed by SC dosing at either Q2W or Q4W intervals (depending on body weight), until study completion.

Study Details

This study aims to evaluate the efficacy and safety of crovalimab in pediatric participants with aHUS.

Key Dates

Start date

Nov 17, 2021

Status verified

Mar 2026

Primary completion

Jul 4, 2025

Completion

May 19, 2029

Study Design

Enrollment

41 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Crovalimab
  Participants will be enrolled in three cohorts: \[1\] Naive Cohort - participants who have not been previously treated with complement inhibitor therapy; \[2\] Switch Cohort - participants who switch to crovalimab from another C5 inhibitor and \[3\] Pretreated Cohort (includes C5 SNP (Single Nucleotide Polymorphism) participants) - participants who received treatment with another C5 inhibitor and subsequently discontinued it.

Primary Outcome Measure

Percentage of Participants with complete TMA response (cTMAr) (Naive Cohort only) [ Time Frame: Baseline up to Week 25 (after 24 weeks on treatment) ]

Locations (3)

Related coverage on Hipa.ai

• Crovalimab Phase 3 Trial for Atypical Hemolytic Uremic Syndrome Reaches…
  Crovalimab · Jul 4, 2025 · ClinicalTrials.gov

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