Study in Adult and Pediatric Patients With HSCT-TMA

Alexion Pharmaceuticals

Status: Withdrawn

Conditions: Thrombotic Microangiopathies; Stem Cell Transplant Complications

Treatments: Other: No intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT04970004

ALX-TMA-501

Details and patient eligibility

About

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.

Sex: All

Ages: 1+ month old

Volunteers: No Healthy Volunteers

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

Trial design

0 participants in 1 patient group

Patients Diagnosed with HSCT-TMA

Treatment:

Other: No intervention