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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).
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).

NCT07732361

CompletedN/A

Sponsor: Alexion Pharmaceuticals, Inc.

Conditions: Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant

Countries: United States, Belgium, Brazil, France, Greece, Italy, Japan, South Korea

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.

Eligibility overview

Sex: ALL

Age: 4 Weeks to —

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria
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.
Locations (43)
  • Boston, Massachusetts, United States
  • Akron, Ohio, United States
  • Salt Lake City, Utah, United States
  • Brussels, Brussels Capital, Belgium
  • Liège, Belgium
  • Fortaleza, Ceará, Brazil
  • Brasília, Federal District, Brazil
  • Curitiba, Paraná, Brazil
  • Recife, Pernambuco, Brazil
  • Porto Alegre, Rio Grande do Sul, Brazil
  • Jaú, São Paulo, Brazil
  • Rio de Janeiro, Brazil
  • São Paulo, Brazil
  • São Paulo, Brazil
  • São Paulo, Brazil
  • São Paulo, Brazil
  • Pessac, Gironde, France
  • Toulouse, Haute Garonne, France
  • Grenoble, Isere, France
  • Paris, France
  • Thessaloniki, Central Macedonia, Greece
  • Pátrai, West Greece, Greece
  • Rome, Lazio, Italy
  • Ancona, Italy
  • Brescia, Italy
  • Florence, Italy
  • Genova, Italy
  • Naples, Italy
  • Pavia, Italy
  • Roma, Italy
  • + 13 more on CT.gov