Study in Adult and Pediatric Patients With HSCT-TMA

NCT04970004 | Withdrawn

• 1 other identifier: ALX-TMA-501
• Study Type: observational
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Stem Cell Transplant Complications; Thrombotic Microangiopathies

Keywords

Hematopoietic Stem Cell Transplant; Transplant-associated TMA; Thrombotic Microangiopathy (TMA)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Proportion of patients achieving TMA response

  During the 52-week period after HSCT-TMA diagnosis

• Changes in individual components of TMA response

  From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis

• Overall survival

  At 26 weeks and 52 weeks after HSCT-TMA diagnosis

• Nonrelapse mortality

  At 26 weeks and 52 weeks after HSCT-TMA diagnosis

Study Arms (1)

Patients Diagnosed with HSCT-TMA

Other: No intervention

Interventions

No intervention OTHER

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

Patients Diagnosed with HSCT-TMA

Eligibility Criteria

• Age: 1 Month+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Clinical Trial Site

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Conditions

Thrombotic Microangiopathies

Condition Hierarchy (Ancestors)

Thrombocytopenia; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2021
• First Posted: July 21, 2021
• Study Start: July 12, 2021
• Primary Completion: April 1, 2022
• Study Completion: April 1, 2022
• Last Updated: May 12, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)