Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for stem cell transplant patients with dangerous blood clotting disorder

NCT ID NCT04543591

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 people aged 12 and older who developed a serious blood clotting condition (thrombotic microangiopathy) after a stem cell transplant. The goal was to see if the drug could prevent death or worsening of the condition over 26 weeks. Participants received either ravulizumab or a placebo, along with standard care.

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

148 people

The number who actually took part.

Started

Dec 2020

Finished

Mar 2026

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

12 to 100 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. 12 years of age or older at time of consent/assent. 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 ≥ 30 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 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 one 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. Acute and/or chronic heart failure with an ejection fraction ≤ 40%. 15. Previously or currently treated with a complement inhibitor. 16. Participation in an interventional treatment study of any therapy for TMA.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Thrombotic microangiopathy are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

    Tampa, Florida, 33612, United States

  • Research Site

    Grosse Pointe Farms, Michigan, 48236, United States

  • Research Site

    Durham, North Carolina, 27705, United States

  • Research Site

    Pittsburgh, Pennsylvania, 15232, United States

  • Research Site

    Seattle, Washington, 98109, United States

  • Research Site

    Parkville, 3050, Australia

  • Research Site

    Bruges, 8000, Belgium

  • Research Site

    Brussels, 1200, Belgium

  • Research Site

    Chênée, 4032, Belgium

  • Research Site

    Yvoir, 5530, Belgium

  • Research Site

    Cerqueira César, 05403-000, Brazil

  • Research Site

    Florianópolis, 88034-000, Brazil

  • Research Site

    Jaú, 17210-080, Brazil

  • Research Site

    Porto Alegre, 90035-903, Brazil

  • Research Site

    Porto Alegre, 90110-270, Brazil

  • Research Site

    Rio de Janeiro, 20230-130, Brazil

  • Research Site

    São José do Rio Preto, 15090-000, Brazil

  • Research Site

    São Paulo, 05.403-010, Brazil

  • Research Site

    Calgary, Alberta, T2N 4N1, Canada

  • Research Site

    Suzhou, 215006, China

  • Research Site

    Tianjin, 300020, China

  • Research Site

    Angers, 49033, France

  • Research Site

    La Tronche, 38043, France

  • Research Site

    Nice, 06200, France

  • Research Site

    Hamburg, 20246, Germany

  • Research Site

    Ulm, 89081, Germany

  • Research Site

    Athens, 12462, Greece

  • Research Site

    Pátrai, 26504, Greece

  • Research Site

    Thessaloniki, 57010, Greece

  • Research Site

    Halfa, 31096, Israel

  • Research Site

    Ramat Gan, 52621, Israel

  • Research Site

    Roma, 00168, Italy

  • Research Site

    Udine, 33100, Italy

  • Research Site

    Akita, 010-8543, Japan

  • Research Site

    Anjo, 446-8602, Japan

  • Research Site

    Chiba, 260-8677, Japan

  • Research Site

    Fukushima, 960-1295, Japan

  • Research Site

    Isehara-shi, 259-1193, Japan

  • Research Site

    Kurashiki-shi, 710-8602, Japan

  • Research Site

    Minatoku, 105-8470, Japan

  • Research Site

    Okayama, 700-8558, Japan

  • Research Site

    Osaka, 545-8586, Japan

  • Research Site

    Osakasayama-shi, 589-8511, Japan

  • Research Site

    Sapporo, 060-8638, Japan

  • Research Site

    Suita-shi, 565-0871, Japan

  • Research Site

    Tsukuba, 305-8576, Japan

  • Research Site

    Wakayama, 641-8510, Japan

  • Research Site

    Groningen, 9713 GZ, Netherlands

  • Research Site

    Goyang-si, 10408, South Korea

  • Research Site

    Seoul, 03080, South Korea

  • Research Site

    Seoul, 03722, South Korea

  • Research Site

    Seoul, 06351, South Korea

  • Research Site

    Barcelona, 08036, Spain

  • Research Site

    Granada, 18014, Spain

  • Research Site

    L'Hospitalet de Llobregat, 08908, Spain

  • Research Site

    Madrid, 28007, Spain

  • Research Site

    Madrid, 28034, Spain

  • Research Site

    Madrid, 28040, Spain

  • Research Site

    Madrid, 28046, Spain

  • Research Site

    Málaga, 29010, Spain

  • Research Site

    Pamplona, 31008, Spain

  • Research Site

    Salamanca, 37007, Spain

  • Research Site

    Seville, 41013, Spain

  • Research Site

    Huddinge, 141 57, Sweden

  • Research Site

    London, W12 0HS, United Kingdom

  • Research Site

    Nottingham, NG5 1PB, United Kingdom

More trials for these conditions

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