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:
N/A
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
1-855-752-2356 or [email protected]
Study Complete

Study Details

Medical Condition
  • Thrombotic Microangiopathy
  • Unmapped
Study Drug
    Date
    Jan 2025 - Mar 2026
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 28+ years
    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
    28+ years

    Study Details

    Medical Condition
    • Thrombotic Microangiopathy
    • Unmapped
    Study Drug
      Date
      Jan 2025 - Mar 2026
      Phase 1
      Phase 2
      Phase 3
      Phase 4
      N/A
      Patient Requirements
      Sex: Female & Male
      Age: 28+ years years
      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
      Brussels, Brussel, Belgium, 1020
      Status
      Completed
      Location
      Research Facility
      Liege, Liege, Belgium, 1020
      Status
      Completed
      Location
      Research Facility
      São Paulo, São Paulo, Brazil, 05403-000
      Status
      Completed
      Location
      Research Facility
      São Paulo, São Paulo, Brazil, 15090-000
      Status
      Completed
      Location
      Research Facility
      Curitiba, Parana, Brazil, 80060-900
      Status
      Completed
      Location
      Research Facility
      Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
      Status
      Completed
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