Study in Adult and Pediatric Patients With HSCT-TMA

NCT ID: NCT04970004

Last Updated: 2022-05-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2021-07-12

Study Completion Date

2022-04-01

Brief Summary

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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

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Conditions

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Stem Cell Transplant Complications Thrombotic Microangiopathies

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

RETROSPECTIVE

Study Groups

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Patients Diagnosed with HSCT-TMA

No intervention

Intervention Type OTHER

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

Interventions

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No intervention

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

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

1 Month

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinical Trial Site

Salt Lake City, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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ALX-TMA-501

Identifier Type: -

Identifier Source: org_study_id

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