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).

Study Identifier:
D9284C00003
ClinicalTrials.gov Identifier:
NCT07732361
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Thrombotic Microangiopathy
Study Drug
    Date
    Jan 2025 - Mar 2026
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 4+ weeks
    Requirements Information
    Inclusion and Exclusion 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.
    Sex
    Female & Male
    Age
    4+ weeks

    Study Details

    Medical Condition
    • Thrombotic Microangiopathy
    Study Drug
      Date
      Jan 2025 - Mar 2026
      Phase 1
      Phase 2
      Phase 3
      Phase 4
      N/A
      Patient Requirements
      Sex: Female & Male
      Age: 4+ weeks
      Requirements Information
      Inclusion and Exclusion 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.

      Protocol 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 Locations

      Location
      Status
      Location
      Research Facility
      Boston, Massachusetts, United States, 02215
      Status
      N/A
      Location
      Research Facility
      Akron, Ohio, United States, 44308
      Status
      N/A
      Location
      Research Facility
      Salt Lake City, Utah, United States, 84108
      Status
      N/A
      Location
      Research Facility
      Brussels, Brussels Capital, Belgium, 1020
      Status
      N/A
      Location
      Research Facility
      Liège, Belgium, 1020
      Status
      N/A
      Location
      Research Facility
      Fortaleza, Ceará, Brazil, 60430-372
      Status
      N/A
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