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).

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT07732361
Status
Completed

Conditions

  • Hematopoietic Stem Cell Transplant
  • Thrombotic Microangiopathy

Eligibility Criteria

Sex
ALL
Age
4 Weeks - N/A
Healthy Volunteers
Not accepted

Study Details

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 Dates

First listed
Jul 28, 2026
Start date
Jan 22, 2025
Status verified
Jul 2026
Primary completion
Mar 31, 2026
Completion
Mar 31, 2026

Study Design

Enrollment
307 participants (actual)

Arms

  • Arm: TMA after HSCT pediatric participants, complement inhibitor treatment naïve
    The study included pediatric participants who were diagnosed with TMA within 52 weeks after an HSCT procedure and who were complement inhibitor treatment naïve
  • Arm: TMA after HSCT pediatric participants who had been treated with eculizumab
    The study included pediatric participants who were diagnosed with TMA within 52 weeks after an HSCT procedure and who had been treated with eculizumab
  • Arm: TMA after HSCT adult participants, complement inhibitor treatment naïve
    The study included adult participants who were diagnosed with TMA within 52 weeks after an HSCT procedure and who were complement inhibitor treatment naïve
  • Arm: TMA after HSCT adult participants who had been treated with eculizumab
    The study included adult participants who were diagnosed with TMA within 52 weeks after an HSCT procedure and who had been treated with eculizumab

Primary Outcome Measure

Overall Survival (OS) [ Time Frame: 52 weeks after TMA diagnosis ]

Locations (3)

FacilityCityStateZIPSite coordinators
Research FacilityBostonMassachusetts02215-
Research FacilityAkronOhio44308-
Research FacilitySalt Lake CityUtah84108-

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