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Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02179359
Recruitment Status : Recruiting
First Posted : July 1, 2014
Last Update Posted : April 18, 2023
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Description
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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 Not Applicable

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : September 2, 2014
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : August 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. incidence of graft failure [ Time Frame: 42 days ]

Secondary Outcome Measures :
  1. overall survival [ Time Frame: 6 months, 1 and 2 years ]
  2. disease free survival [ Time Frame: 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
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Ages Eligible for Study:   up to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02179359


Contacts
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Contact: Lisa Burke 612-273-8482 lburke3@Fairview.org

Locations
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United States, Minnesota
University of Minnesota Medical Center, Fairview Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Lisa Burke    612-273-8482    lburke3@Fairview.org   
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
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Principal Investigator: Ashish Gupta, MBBS, MPH Masonic Cancer Center, University of Minnesota
More Information
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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT02179359    
Other Study ID Numbers: 2014OC034
MT2014-10C ( Other Identifier: Masonic Cancer Center, University of Minnesota )
First Posted: July 1, 2014    Key Record Dates
Last Update Posted: April 18, 2023
Last Verified: April 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Masonic Cancer Center, University of Minnesota:
Stem Cell Transplant
Additional relevant MeSH terms:
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Shwachman-Diamond Syndrome
Hemoglobinuria
Anemia, Sickle Cell
Thalassemia
Neutropenia
beta-Thalassemia
Hemoglobinuria, Paroxysmal
Hemoglobinopathies
Hematologic Diseases
Anemia, Diamond-Blackfan
Thrombasthenia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Genetic Diseases, Inborn
 Agranulocytosis
Leukopenia
Leukocyte Disorders
Proteinuria
Urination Disorders
Urologic Diseases
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Male Urogenital Diseases
Urological Manifestations
Myelodysplastic Syndromes
Bone Marrow Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic