A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT).

Completed

Conditions studied: Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant

In brief

The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and who were either complement inhibitor treatment naïve or had been treated with eculizumab.

Key facts

Study ID: NCT07732361
Run by: Alexion Pharmaceuticals, Inc.
People needed: 307
Starts: 2025-01-22
Expected to finish: 2026-03-31
Last updated by the study team: 2026-07-28

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Participant must be ≥ 28 days of age at the time of TMA diagnosis.
Body weight ≥ 5 kg at the time of TMA diagnosis.
Documented TMA on/after 01 Jan 2010 to 30 Jun 2024 with concurrent renal dysfunction with no documented alternative explanation ≤ 52 weeks from the HSCT.
Documentation of participant's vital status through 52 weeks after the date of TMA diagnosis.
Documentation of date and type of HSCT, date of TMA, absence of acute GVHD, and infection status at time of TMA diagnosis.
Documentation of at least 2 of the following: indication for most recent HSCT, serum creatinine at time of TMA diagnosis, presence of multiorgan dysfunction at time of TMA diagnosis.
Informed consent obtained if required by local regulations.

You may not qualify if…

Medical Conditions (Ongoing at the Time of TMA Diagnosis): TTP, ST-HUS, Immune-mediated hemolysis not due to TMA, DIC, Bone marrow/graft failure of HSCT, VOD (regardless of severity), HIV infection, Sepsis that required vasopressor support.
Prior/Concomitant Therapy: Received a complement inhibitor other than eculizumab from time of suspicion of HSCT-TMA diagnosis through 52 weeks post-HSCT-TMA diagnosis..
Other Exclusions: Participation in an investigational drug or device study for the treatment of TMA within 30 days prior to TMA diagnosis or during the 52 weeks following TMA diagnosis.

Where it is running

Research Facility — Boston, Massachusetts, United States
Research Facility — Akron, Ohio, United States
Research Facility — Salt Lake City, Utah, United States
Research Facility — Brussels, Brussels Capital, Belgium
Research Facility — Liège, Belgium
Research Facility — Fortaleza, Ceará, Brazil
Research Facility — Brasília, Federal District, Brazil
Research Facility — Curitiba, Paraná, Brazil
Research Facility — Recife, Pernambuco, Brazil
Research Facility — Porto Alegre, Rio Grande do Sul, Brazil
Research Facility — Jaú, São Paulo, Brazil
Research Facility — Rio de Janeiro, Brazil
Research Facility — São Paulo, Brazil
Research Facility — São Paulo, Brazil
Research Facility — São Paulo, Brazil
Research Facility — São Paulo, Brazil
Research Facility — Pessac, Gironde, France
Research Facility — Toulouse, Haute Garonne, France
Research Facility — Grenoble, Isere, France
Research Facility — Paris, France
Research Facility — Thessaloniki, Central Macedonia, Greece
Research Facility — Pátrai, West Greece, Greece
Research Facility — Rome, Lazio, Italy
Research Facility — Ancona, Italy
Research Facility — Brescia, Italy

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.