Study in Adult and Pediatric Patients With HSCT-TMA

Withdrawn

• Conditions: Stem Cell Transplant Complications; Thrombotic Microangiopathies
• Interventions: Other: No intervention

• Registration Number: NCT04970004
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-07-12
• Study First Submitted: 2021-07-16
• Study First Submitted QC: 2021-07-16
• Study First Posted: 2021-07-21
• Primary Completion: 2022-04-01
• Study Completion: 2022-04-01
• Status Verified: 2022-05
• Last Update Submitted: 2022-05-09
• Last Update Posted: 2022-05-12

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients Diagnosed with HSCT-TMA	No intervention	-

Primary Outcome Measures

Name	Time	Method
Proportion of patients achieving TMA Response	During the 26-week period after HSCT-TMA diagnosis	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)

Secondary Outcome Measures

Name	Time	Method
Nonrelapse mortality	At 26 weeks and 52 weeks after HSCT-TMA diagnosis	Death due to any cause during the study, with the exception of death due to underlying disease progression or relapse
Proportion of patients achieving TMA response	During the 52-week period after HSCT-TMA diagnosis
Changes in individual components of TMA response	From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis	Platelets, lactate dehydrogenase, eGFR, chronic kidney disease stage, dialysis status
Overall survival	At 26 weeks and 52 weeks after HSCT-TMA diagnosis

Trial Locations

Locations (1)

Clinical Trial Site; 🇺🇸 Salt Lake City, Utah, United States