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New drug shows promise for rare post-transplant complication in kids

NCT ID NCT04557735

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called ravulizumab in 41 children who developed a serious blood clotting problem (TMA) after a stem cell transplant. The goal was to see if the drug could help control the condition and improve blood counts and kidney function. The treatment lasted 26 weeks, and children were monitored for another 26 weeks after stopping the drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

41 people

The number who actually took part.

Started

Dec 2020

Finished

May 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. ≥ 28 days of age up to \< 18 years of age at the time of signing the informed consent. 2. Received HSCT within the past 12 months. 3. Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition. 4. A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or ≤14 days prior to the Screening Period. 5. Body weight ≥ 5 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent). 6. Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception. 7. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants \<18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible. 8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent. Exclusion Criteria: 1. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency. 2. Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test. 3. Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA. 4. Clinical diagnosis of disseminated intravascular coagulation (DIC). 5. Known bone marrow/graft failure for the current HSCT. 6. Diagnosis of veno-occlusive disease (VOD) which is unresolved at the time of Screening. 7. Human immunodeficiency virus (HIV) infection. 8. Unresolved meningococcal disease. 9. Presence of sepsis requiring vasopressor support. 10. Pregnancy or breastfeeding. 11. Hypersensitivity to murine proteins or to 1 of the excipients of Ravulizumab. 12. Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study. 13. Respiratory failure requiring mechanical ventilation. 14. Previously or currently treated with a complement inhibitor. 15. Participation in an interventional treatment study of any therapy for TMA.

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Conditions

The condition(s) this trial relates to.

thrombotic microangiopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Thrombotic microangiopathy

Contacts and locations

Locations

  • Research Site

    Tucson, Arizona, 85724, United States

  • Research Site

    Aurora, Colorado, 80045, United States

  • Research Site

    Atlanta, Georgia, 30322, United States

  • Research Site

    Chicago, Illinois, 60611, United States

  • Research Site

    Portland, Oregon, 97239, United States

  • Research Site

    Dallas, Texas, 75235, United States

  • Research Site

    Salt Lake City, Utah, 84108, United States

  • Research Site

    Madison, Wisconsin, 53792, United States

  • Research Site

    Haifa, 91096, Israel

  • Research Site

    Jerusalem, 91120, Israel

  • Research Site

    Petah Tikva, 4920235, Israel

  • Research Site

    Ramat Gan, 5265601, Israel

  • Research Site

    Roma, 00165, Italy

  • Research Site

    Fukushima, 960-1295, Japan

  • Research Site

    Kobe, 650-0047, Japan

  • Research Site

    Nagoya, 466-8560, Japan

  • Research Site

    Osaka, 534-0021, Japan

  • Research Site

    Seoul, 03080, South Korea

  • Research Site

    Seoul, 5505, South Korea

  • Research Site

    Barcelona, 08041, Spain

  • Research Site

    Esplugues de Llobregat, 8950, Spain

  • Research Site

    Madrid, 28046, Spain

  • Research Site

    Salamanca, 37007, Spain

  • Research Site

    Birmingham, B4 6NH, United Kingdom

  • Research Site

    Bristol, BS2 8BJ, United Kingdom

  • Research Site

    Newcastle upon Tyne, NE1 4LP, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.