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).
ALX-TMA-502
A Secondary Real-world Data Study of Pediatric and Adult Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)
2 other identifiers
observational
307
10 countries
43
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jan 2025
43 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 22, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
March 31, 2026
CompletedFirst Submitted
Initial submission to the registry
July 23, 2026
CompletedFirst Posted
Study publicly available on registry
July 28, 2026
CompletedJuly 28, 2026
July 1, 2026
1.2 years
July 23, 2026
July 24, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall Survival (OS)
To assess OS through 52 weeks after TMA diagnosis.
52 weeks after TMA diagnosis
Secondary Outcomes (2)
Overall Survival (OS)
100 days and 26 weeks after TMA diagnosis
Non-relapse mortality
Through 100 days, 26 weeks, and 52 weeks after TMA diagnosis
Study Arms (4)
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
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
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
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
Eligibility Criteria
The study included pediatric and adult participants who were diagnosed with TMA within 52 weeks after an HSCT procedure and who were either complement inhibitor treatment naïve or had been treated with eculizumab. Data was collected from medical records in 41 study centers across 10 countries
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..
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (43)
Research Facility
Boston, Massachusetts, 02215, United States
Research Facility
Akron, Ohio, 44308, United States
Research Facility
Salt Lake City, Utah, 84108, United States
Research Facility
Brussels, Brussels Capital, 1020, Belgium
Research Facility
Liège, 1020, Belgium
Research Facility
Fortaleza, Ceará, 60430-372, Brazil
Research Facility
Brasília, Federal District, 70684-831, Brazil
Research Facility
Curitiba, Paraná, 80060-900, Brazil
Research Facility
Recife, Pernambuco, 52010-010, Brazil
Research Facility
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
Research Facility
Jaú, São Paulo, 17210-120, Brazil
Research Facility
Rio de Janeiro, 20230-130, Brazil
Research Facility
São Paulo, 04023-064, Brazil
Research Facility
São Paulo, 05403-000, Brazil
Research Facility
São Paulo, 05410-030, Brazil
Research Facility
São Paulo, 15090-000, Brazil
Research Facility
Pessac, Gironde, 33604, France
Research Facility
Toulouse, Haute Garonne, 31059, France
Research Facility
Grenoble, Isere, 38043, France
Research Facility
Paris, 75475, France
Research Facility
Thessaloniki, Central Macedonia, 57010, Greece
Research Facility
Pátrai, West Greece, 26500, Greece
Research Facility
Rome, Lazio, 00168, Italy
Research Facility
Ancona, 60126, Italy
Research Facility
Brescia, 25123, Italy
Research Facility
Florence, 50134, Italy
Research Facility
Genova, 16147, Italy
Research Facility
Naples, 80123, Italy
Research Facility
Pavia, 27100, Italy
Research Facility
Roma, 00165, Italy
Research Facility
Torino, 10126, Italy
Research Facility
Fukushima, Fukushima, 960-1295, Japan
Research Facility
Tsukuba, Ibaraki, 305-8576, Japan
Research Facility
Saitama-shi, Saitama, 330-8777, Japan
Research Facility
Wakayama, Wakayama, 641-8510, Japan
Research Facility
Seoul, 03080, South Korea
Research Facility
Seoul, 05505, South Korea
Research Facility
Seoul, 06351, South Korea
Research Facility
Barcelona, 08041, Spain
Research Facility
Barcelona, 08950, Spain
Research Facility
London, Greater London, W12 0HS, United Kingdom
Research Facility
Newcastle, Tyne & Wear, NE1 4LP, United Kingdom
Research Facility
Birmingham, West Midlands, B4 6NH, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 23, 2026
First Posted
July 28, 2026
Study Start
January 22, 2025
Primary Completion
March 31, 2026
Study Completion
March 31, 2026
Last Updated
July 28, 2026
Record last verified: 2026-07
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
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