Study of Ravulizumab in Pediatric Participants With HSCT-TMA
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.
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
- Enrollment
- 41 participants
Conditions and interventions
Conditions
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 6, 2020
- Primary completion
- Nov 25, 2024
- Completion
- May 26, 2025
- Last update posted
- Jan 11, 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 11, 2026 · Synced Sep 1, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04557735 live on ClinicalTrials.gov.