Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Not Applicable

Terminated

• Conditions: Paroxysmal Nocturnal Hemoglobinuria; Glanzmann Thrombasthenia; Non-Malignant Hematologic Disorders; Shwachman-Diamond Syndrome; Diamond Blackfan Anemia; Sickle Cell Disease; Transfusion Dependent Alpha- or Beta- Thalassemia; Severe Congenital Neutropenia
• Interventions: Drug: Myeloablative Preparative Regimen; Drug: Reduced Toxicity Ablative Regimen; Drug: Reduced Intensity Preparative Regimen

• Registration Number: NCT02179359
• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Brief Summary

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-06-27
• Study First Submitted QC: 2014-06-27
• Study First Posted: 2014-07-01
• Study Start: 2014-09-02
• Primary Completion: 2024-11-21
• Study Completion: 2024-11-21
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-27
• Last Update Posted: 2025-03-03

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 38

Inclusion Criteria

• Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
• Acceptable stem cell source identified
• Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
• Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
• Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
• Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%

Exclusion Criteria

• active, uncontrolled infection
• pregnant or breastfeeding
• HIV positive

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Myeloablative Preparative Regimen	Myeloablative Preparative Regimen	For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.
Reduced Toxicity Ablative Regimen	Reduced Toxicity Ablative Regimen	For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are \<12 years and/or have mild/moderate iron exposure.
Reduced Intensity Preparative Regimen	Reduced Intensity Preparative Regimen	For use in patients with unrelated donor bone marrow and for DBA patients who are \>12 years and/or have significant iron exposure.

Primary Outcome Measures

Name	Time	Method
incidence of graft failure	42 days

Secondary Outcome Measures

Name	Time	Method
overall survival	6 months, 1 and 2 years
disease free survival	6 months, 1 and 2 years	patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)

Trial Locations

Locations (1)

University of Minnesota Medical Center, Fairview; 🇺🇸 Minneapolis, Minnesota, United States