Study in Adult and Pediatric Patients With HSCT-TMA

Study Identifier:
ALX-TMA-501
ClinicalTrials.gov Identifier:
NCT04970004
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other

Study Details

Medical Condition
  • Unmapped
Study Drug
    Date
    Jul 2021 - Apr 2022
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 1+ years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Body weight ≥ 5 kg at the time of HSCT-TMA diagnosis
    • Documented TMA diagnosis within 6 months from the HSCT
    • Evidence of renal dysfunction
    • Presence of hypertension
    Exclusion Criteria
    • History or presence of familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity \< 5%)
    • Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
    • Positive direct Coombs test
    • Diagnosis of disseminated intravascular coagulation
    • History or presence of bone marrow/graft failure
    • Diagnosis of veno-occlusive disease
    • Received a complement inhibitor (eg, eculizumab) post-HSCT through 12 months post TMA diagnosis
    Sex
    Female & Male
    Age
    1+ years

    Study Details

    Medical Condition
    • Unmapped
    Study Drug
      Date
      Jul 2021 - Apr 2022
      Phase 1
      Phase 2
      Phase 3
      Phase 4
      N/A
      Patient Requirements
      Sex: Female & Male
      Age: 1+ years years
      Requirements Information
      Inclusion and Exclusion Criteria
      Inclusion Criteria
      • Body weight ≥ 5 kg at the time of HSCT-TMA diagnosis
      • Documented TMA diagnosis within 6 months from the HSCT
      • Evidence of renal dysfunction
      • Presence of hypertension
      Exclusion Criteria
      • History or presence of familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity \< 5%)
      • Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
      • Positive direct Coombs test
      • Diagnosis of disseminated intravascular coagulation
      • History or presence of bone marrow/graft failure
      • Diagnosis of veno-occlusive disease
      • Received a complement inhibitor (eg, eculizumab) post-HSCT through 12 months post TMA diagnosis

      Protocol Summary

      This is an observational, retrospective study designed to assess outcomes in patients diagnosed with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) who were not treated with complement component (C5) inhibitor therapy. Data required to evaluate study outcomes will be abstracted from the medical records of all patients who meet study eligibility criteria.

      Trial Locations

      Location
      Status
      Location
      Clinical Trial Site
      Salt Lake City, Utah, United States, 84112
      Status
      N/A