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Completed Phase 3 Interventional Results available

Study of Ravulizumab in Pediatric Participants With HSCT-TMA

ClinicalTrials.gov ID: NCT04557735

Public ClinicalTrials.gov record NCT04557735. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 17, 2026, 3:43 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplantation (HSCT)

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT04557735
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Enrollment
41 participants

Conditions and interventions

Interventions

Drug · Other

Eligibility (public fields only)

Age range
Up to 17 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Dec 7, 2020 (Actual)
Primary completion
Nov 26, 2024 (Actual)
Completion
May 27, 2025 (Actual)
Last update posted
Jan 12, 2026

2020 – 2025

United States locations

U.S. sites
8
U.S. states
8
U.S. cities
8
Facility City State ZIP Site status
Research Site Tucson Arizona 85724 —
Research Site Aurora Colorado 80045 —
Research Site Atlanta Georgia 30322 —
Research Site Chicago Illinois 60611 —
Research Site Portland Oregon 97239 —
Research Site Dallas Texas 75235 —
Research Site Salt Lake City Utah 84108 —
Research Site Madison Wisconsin 53792 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 18 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT04557735, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Jan 12, 2026 · Synced Oct 17, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT04557735 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →