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Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

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ClinicalTrials.gov Identifier: NCT02179359
Recruitment Status : Recruiting
First Posted : July 1, 2014
Last Update Posted : September 25, 2018
Sponsor:
Information provided by (Responsible Party):
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.

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 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 : August 2019
Estimated Study Completion Date : August 2021


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




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)



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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

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
Contact: Kim Nelson 612-273-2925 knelso62@fairview.org

Locations
United States, Minnesota
University of Minnesota Medical Center, Fairview Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Kim Nelson    612-273-2925    knelso62@fairview.org   
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Angela R Smith, MD Masonic Cancer Center, University of Minnesota

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT02179359     History of Changes
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: September 25, 2018
Last Verified: September 2018

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:
Anemia, Sickle Cell
Anemia, Diamond-Blackfan
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Thalassemia
Neutropenia
beta-Thalassemia
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Hemoglobinopathies
Hematologic Diseases
Thrombasthenia
Bone Marrow Diseases
Lipomatosis
Exocrine Pancreatic Insufficiency
Genetic Diseases, Inborn
Agranulocytosis
Leukopenia
Leukocyte Disorders
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Myelodysplastic Syndromes
Red-Cell Aplasia, Pure
Blood Coagulation Disorders, Inherited