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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT04970004
Other study ID # ALX-TMA-501
Secondary ID
Status Withdrawn
Phase
First received
Last updated
Start date July 12, 2021
Est. completion date April 1, 2022

Study information

Verified date May 2022
Source Alexion Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date April 1, 2022
Est. primary completion date April 1, 2022
Accepts healthy volunteers No
Gender All
Age group 1 Month and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Other:
No intervention
No study intervention will be administered as part of this study.

Locations

Country Name City State
United States Clinical Trial Site Salt Lake City Utah

Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Proportion of patients achieving TMA Response TMA Response is defined as platelet count = 50,000/mm^3, lactate dehydrogenase < 1.5 upper limit of normal, absence of schistocytes (if present at baseline), and increase in eGFR = 30% from baseline or discontinuation of dialysis (for patients on dialysis at baseline) During the 26-week period after HSCT-TMA diagnosis
Secondary Proportion of patients achieving TMA response During the 52-week period after HSCT-TMA diagnosis
Secondary Changes in individual components of TMA response Platelets, lactate dehydrogenase, eGFR, chronic kidney disease stage, dialysis status From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis
Secondary Overall survival At 26 weeks and 52 weeks after HSCT-TMA diagnosis
Secondary Nonrelapse mortality Death due to any cause during the study, with the exception of death due to underlying disease progression or relapse At 26 weeks and 52 weeks after HSCT-TMA diagnosis
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