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NCT04557735 · ClinicalTrials.gov registry record · Phase 3
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
A Phase 3 study, sponsored by Alexion Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 41
- Enrollment target
NCT04557735: Completed Phase 3 study, sponsored by Alexion Pharmaceuticals.
NCT04557735 is a Phase 3 study that has completed, run by Alexion Pharmaceuticals. The registered enrollment target is 41 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04557735, a Phase 3 study, has completed, sponsored by Alexion Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 41 participants
- Enrollment target
Study 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.
Primary Outcome
The criteria for TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal \[ULN\]) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, w
Interventions
- DRUG Ravulizumab
- OTHER Best Supportive Care
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 41 participants |
| Start Date | 2020-12-07 |
| Est. Completion | 2025-05-27 |
| Phase | Phase 3 |
What the finished NCT04557735 record still lists
NCT04557735 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 41 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower).
The record links to 0 conditions, and to 2 interventions - of which Ravulizumab is the first listed.
NCT04557735 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04557735 about?
NCT04557735 is a clinical study titled "Study of Ravulizumab in Pediatric Participants With HSCT-TMA". 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 peri...
What is the current status of trial NCT04557735?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 41 participants. The study started on 2020-12-07. Estimated completion is 2025-05-27.
What interventions are being tested in trial NCT04557735?
The interventions under investigation include: Ravulizumab (DRUG), Best Supportive Care (OTHER).
Who is sponsoring clinical trial NCT04557735?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04557735's enrollment target sits among peer trials
41 1877th of 2000 higher than 123 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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