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Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
NCT04543591 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04543591
The ClinicalTrials.gov registry entry for NCT04543591 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 148 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Alexion Pharmaceuticals, which has 109 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04543591 reports 20 study locations spanning 7 distinct geographic areas — top geographies include Other, Florida, Michigan. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04543591?
The interventions under investigation include: Placebo (OTHER), Ravulizumab (BIOLOGICAL), Best supportive care (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04543591?
This trial is sponsored by Alexion Pharmaceuticals, which has 109 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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