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OpenTrials
Completed

NCT Number: NCT07732361

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).

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.

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Key information

Age range

4 week and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Research Facility, Brussels, Brussels Capital, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Primary outcomes

  1. Overall Survival (OS)

    Time frame: 52 weeks after TMA diagnosis

    To assess OS through 52 weeks after TMA diagnosis.

Secondary outcomes

  1. Overall Survival (OS)

    Time frame: 100 days and 26 weeks after TMA diagnosis

    OS through 100 days and 26 weeks after TMA diagnosis.

  2. Non-relapse mortality

    Time frame: Through 100 days, 26 weeks, and 52 weeks after TMA diagnosis

    Non-relapse mortality, defined as death due to any cause other than primary disease progression (i.e. indication fror transplant) or relapse.

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

A Secondary Real-world Data Study of Pediatric and Adult Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)

Acronym: ALX-TMA-502

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Jul 28, 2026
Registry last updated
Jul 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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