Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT ID: NCT02179359

Last Updated: 2025-03-03

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: NA
• Total Enrollment: 38 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-09-02
• Study Completion Date: 2024-11-21

Brief Summary

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

Conditions

Sickle Cell Disease; Transfusion Dependent Alpha- or Beta- Thalassemia; Diamond Blackfan Anemia; Paroxysmal Nocturnal Hemoglobinuria; Glanzmann Thrombasthenia; Severe Congenital Neutropenia; Shwachman-Diamond Syndrome; Non-Malignant Hematologic Disorders

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Reduced Toxicity Ablative Regimen

Intervention Type: DRUG

• Anti-thymocyte Globulin (ATG)
• Fludarabine
• Busulfan
• Stem Cell Infusion Day 0

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.

Group Type: EXPERIMENTAL

Reduced Intensity Preparative Regimen

Intervention Type: DRUG

• Alemtuzumab
• Cyclophosphamide
• Fludarabine
• Total Body Irradiation (TBI)
• Stem Cell Infusion Day 0

Myeloablative Preparative Regimen

For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.

Group Type: EXPERIMENTAL

Myeloablative Preparative Regimen

Intervention Type: DRUG

• Alemtuzumab
• Cyclophosphamide
• Busulfan
• Stem Cell Infusion Day 0

Interventions

Reduced Toxicity Ablative Regimen

• Anti-thymocyte Globulin (ATG)
• Fludarabine
• Busulfan
• Stem Cell Infusion Day 0

Intervention Type: DRUG

Reduced Intensity Preparative Regimen

• Alemtuzumab
• Cyclophosphamide
• Fludarabine
• Total Body Irradiation (TBI)
• Stem Cell Infusion Day 0

Intervention Type: DRUG

Myeloablative Preparative Regimen

• Alemtuzumab
• Cyclophosphamide
• Busulfan
• Stem Cell Infusion Day 0

Intervention Type: DRUG

Eligibility Criteria

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

• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ashish Gupta, MBBS, MPH

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

University of Minnesota Medical Center, Fairview

Minneapolis, Minnesota, United States

Countries

United States

Other Identifiers

MT2014-10C

Identifier Type: OTHER

Identifier Source: secondary_id

2014OC034

Identifier Type: -

Identifier Source: org_study_id