WithdrawnNot Applicable

Study in Adult and Pediatric Patients With HSCT-TMA

Stopped: Sponsor decision to not proceed.

United States0Started 2021-07-12

Plain-language 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.

Who can participate

Age range1 Month

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Proportion of patients achieving TMA Response

Timeframe: During the 26-week period after HSCT-TMA diagnosis

Trial details

NCT IDNCT04970004

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2022-04-01

View on ClinicalTrials.gov More Stem Cell Transplant Complications trials