Study in Adult and Pediatric Patients With HSCT-TMA

May 9, 2022 updated by: Alexion Pharmaceuticals

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

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Observational

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 month and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Adult and pediatric patients who were diagnosed with TMA within 6 months after a HSCT procedure

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Retrospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients Diagnosed with HSCT-TMA
Other: No intervention
No study intervention will be administered as part of this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of patients achieving TMA Response
Time Frame: 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)
During the 26-week period after HSCT-TMA diagnosis

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of patients achieving TMA response
Time Frame: During the 52-week period after HSCT-TMA diagnosis
During the 52-week period after HSCT-TMA diagnosis
Changes in individual components of TMA response
Time Frame: From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis
Platelets, lactate dehydrogenase, eGFR, chronic kidney disease stage, dialysis status
From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis
Overall survival
Time Frame: At 26 weeks and 52 weeks after HSCT-TMA diagnosis
At 26 weeks and 52 weeks after HSCT-TMA diagnosis
Nonrelapse mortality
Time Frame: 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
At 26 weeks and 52 weeks after HSCT-TMA diagnosis

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 12, 2021

Primary Completion (Actual)

April 1, 2022

Study Completion (Actual)

April 1, 2022

Study Registration Dates

First Submitted

July 16, 2021

First Submitted That Met QC Criteria

July 16, 2021

First Posted (Actual)

July 21, 2021

Study Record Updates

Last Update Posted (Actual)

May 12, 2022

Last Update Submitted That Met QC Criteria

May 9, 2022

Last Verified

May 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALX-TMA-501

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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