Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

University of Minnesota (UMN)

Status: Terminated

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

Treatments: Drug: Reduced Toxicity Ablative Regimen; Drug: Reduced Intensity Preparative Regimen; Drug: Myeloablative Preparative Regimen

Study type: Interventional

Funder types: Other

Identifiers

NCT02179359

MT2014-10C (Other Identifier)

2014OC034

Details and patient eligibility

About

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

Enrollment: 38 patients

Sex: All

Ages: Under 55 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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38 participants in 3 patient groups

Reduced Toxicity Ablative Regimen

Experimental group

Description:

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.

Treatment:

Drug: Reduced Toxicity Ablative Regimen

Reduced Intensity Preparative Regimen

Experimental group

Description:

For use in patients with unrelated donor bone marrow and for DBA patients who are \>12 years and/or have significant iron exposure.

Treatment:

Drug: Reduced Intensity Preparative Regimen

Myeloablative Preparative Regimen

Experimental group

Description:

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

Treatment:

Drug: Myeloablative Preparative Regimen