Study in Adult and Pediatric Patients With HSCT-TMA

Stem Cell Transplant Complications Clinical Trial

Official title:

A Retrospective Observational Study of Adult and Pediatric Patients With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04970004
• Other study ID #: ALX-TMA-501
• Status: Withdrawn
• 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
• 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• Status: Withdrawn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

Related Conditions & MeSH terms

• Stem Cell Transplant Complications
• Thrombotic Microangiopathies
• Vascular Diseases

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