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Study of Ravulizumab in Pediatric Participants With HSCT-TMA

NCT04557735 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2026-01-12

Study results available
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Summary

This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to \< 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.

Conditions

Interventions

DRUG

Ravulizumab

Weight-based doses of ravulizumab will be administered intravenously as a loading dose regimen followed by maintenance dosing every 4 or 8 weeks, depending upon weight.

OTHER

Best Supportive Care

Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-07
Primary Completion
2024-11-26
Completion
2025-05-27
FDA Drug
Yes

Countries

  • United States
  • Israel
  • Italy
  • Japan
  • South Korea
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04557735 on ClinicalTrials.gov