Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04543591 · ClinicalTrials.gov registry record · Phase 3
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
A Phase 3 study of Thrombotic Microangiopathy, sponsored by Alexion Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 148
- Enrollment target
- 20
- Study locations
NCT04543591: Active Phase 3 study of Thrombotic Microangiopathy, sponsored by Alexion Pharmaceuticals.
NCT04543591 is a Phase 3 study of Thrombotic Microangiopathy that is active but no longer recruiting, run by Alexion Pharmaceuticals. The registered enrollment target is 148 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (81% lower). The trial reports 20 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04543591, a Phase 3 study of Thrombotic Microangiopathy, is active but no longer recruiting, sponsored by Alexion Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 148 participants
- Enrollment target
- 20
- Study locations
Study Summary
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). In Stage 1, an open-label, single-arm period, the dosing regimen will be confirmed. In Stage 2, participants will be randomized to receive either blinded ravulizumab plus best supportive care or matching placebo plus best supportive care. The treatment period is 26 weeks (open-label for Stage 1, and randomized, double-blind, and placebo-controlled for Stage 2) followed by a 26-week follow-up period.
Primary Outcome
Event free survival during the 26 weeks treatment period defined as the time from randomization until the first of the two following events: death and clinical worsening.
Conditions Studied
Interventions
- OTHER Placebo
- BIOLOGICAL Ravulizumab
- OTHER Best supportive care
Study Locations (20)
Other
- Research Site - Parkville
- Research Site - Bruges
- Research Site - Brussels
- Research Site - Chênée
- Research Site - Yvoir
- Research Site - Cerqueira César
- Research Site - Florianópolis
- Research Site - Jaú
- Research Site - Porto Alegre
- Research Site - Porto Alegre
- Research Site - Rio de Janeiro
- Research Site - São José do Rio Preto
- Research Site - São Paulo
- Research Site - Suzhou
Florida
- Research Site - Tampa
Michigan
- Research Site - Grosse Pointe Farms
North Carolina
- Research Site - Durham
Pennsylvania
- Research Site - Pittsburgh
Washington
- Research Site - Seattle
Alberta
- Research Site - Calgary
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 148 participants |
| Start Date | 2020-12-10 |
| Est. Completion | 2026-03-20 |
| Phase | Phase 3 |
What the registry record for NCT04543591 still lists
NCT04543591 is an interventional study that assigns participants to a tested intervention. The registered 148 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (81% lower).
The record links to 1 condition, with Thrombotic Microangiopathy appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.
NCT04543591 names 20 study sites across 7 states, led by Other, Florida, Michigan.
Frequently Asked Questions
What is clinical trial NCT04543591 about?
NCT04543591 is a clinical study titled "Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant". This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). In Stage 1, an open-label, single-arm period, the dosing regimen will be...
What is the current status of trial NCT04543591?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 148 participants. The study started on 2020-12-10. Estimated completion is 2026-03-20.
What conditions does trial NCT04543591 study?
This clinical trial studies the following conditions: Thrombotic Microangiopathy.
What interventions are being tested in trial NCT04543591?
The interventions under investigation include: Placebo (OTHER), Ravulizumab (BIOLOGICAL), Best supportive care (OTHER).
Who is sponsoring clinical trial NCT04543591?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04543591 being conducted?
This trial has 20 study locations across Florida, Michigan, North Carolina, Pennsylvania, Washington. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04543591's enrollment target sits among peer trials
148 1547th of 2000 higher than 451 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00739362 · 148 participants · Phase 2
Effects of Brain Stimulation on Food Intake and Behavioral Weight Loss Treatment
- NCT03161223 · 148 participants · Phase 1
Durvalumab in Different Combinations With Pralatrexate, Romidepsin and Oral 5-Azacitidine for Lymphoma
- NCT03858231 · 148 participants · Phase 4
Opioids Versus Non-Opioids Postoperative After Knee Arthroscopic Surgery
- NCT04486352 · 148 participants · Phase 1
A Study of Targeted Agents for Patients With Recurrent or Persistent Endometrial Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI