Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Sponsor

Masonic Cancer Center, University of Minnesota (Other)

Overall Status

Recruiting

CT.gov ID

NCT02179359

Collaborator

(none)

Enrollment

Location

Arms

106.9

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
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	Drug: Reduced Toxicity Ablative Regimen Drug: Reduced Intensity Preparative Regimen Drug: Myeloablative Preparative Regimen	N/A

Study Design

Study Type:

Interventional

Anticipated Enrollment :

25 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

Actual Study Start Date :

Sep 2, 2014

Anticipated Primary Completion Date :

Jan 1, 2023

Anticipated Study Completion Date :

Aug 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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.	Drug: Reduced Toxicity Ablative Regimen Anti-thymocyte Globulin (ATG) Fludarabine Busulfan Stem Cell Infusion Day 0
Experimental: 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.	Drug: Reduced Intensity Preparative Regimen Alemtuzumab Cyclophosphamide Fludarabine Total Body Irradiation (TBI) Stem Cell Infusion Day 0
Experimental: Myeloablative Preparative Regimen For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.	Drug: Myeloablative Preparative Regimen Alemtuzumab Cyclophosphamide Busulfan Stem Cell Infusion Day 0

Arm

Intervention/Treatment

Experimental: 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.

Drug: Reduced Toxicity Ablative Regimen

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

Experimental: 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.

Drug: Reduced Intensity Preparative Regimen

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

Experimental: Myeloablative Preparative Regimen

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

Drug: Myeloablative Preparative Regimen

Alemtuzumab Cyclophosphamide Busulfan Stem Cell Infusion Day 0

Outcome Measures

Primary Outcome Measures

incidence of graft failure [42 days]

Secondary Outcome Measures

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)

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 55 Years

Sexes Eligible for Study:

All

Accepts 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%