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Completed
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).
Conditions: Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant
Sex: All
Ages: 4 Weeks – N/A
Healthy volunteers: No
Enrollment: 307
Sponsor: Alexion Pharmaceuticals, Inc.
Location: Research Facility Boston Massachusetts
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.
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.
Source: ClinicalTrials.gov (NCT07732361). StuddyBuddy aggregates publicly available trial information.