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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2024 |
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Main ID: |
NCT02179359 |
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Date of registration:
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27/06/2014 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
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Scientific title:
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MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders |
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Date of first enrolment:
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September 2, 2014 |
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Target sample size:
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25 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT02179359 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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N/A
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Countries of recruitment
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United States
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Contacts
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Name:
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Ashish Gupta, MBBS, MPH |
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Address:
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Telephone:
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Email:
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Affiliation:
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Masonic Cancer Center, University of Minnesota |
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Name:
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Lisa Burke |
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Address:
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Telephone:
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612-273-8482 |
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Email:
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lburke3@Fairview.org |
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Affiliation:
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Name:
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Lisa Burke |
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Address:
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Telephone:
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612-273-8482 |
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Email:
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lburke3@Fairview.org |
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Affiliation:
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Key inclusion & exclusion criteria
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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
Age minimum:
N/A
Age maximum:
55 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Diamond Blackfan Anemia
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Paroxysmal Nocturnal Hemoglobinuria
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Severe Congenital Neutropenia
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Sickle Cell Disease
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Glanzmann Thrombasthenia
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Transfusion Dependent Alpha- or Beta- Thalassemia
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Non-Malignant Hematologic Disorders
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Shwachman-Diamond Syndrome
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Intervention(s)
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Drug: Reduced Intensity Preparative Regimen
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Drug: Reduced Toxicity Ablative Regimen
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Drug: Myeloablative Preparative Regimen
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Primary Outcome(s)
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incidence of graft failure
[Time Frame: 42 days]
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Secondary Outcome(s)
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disease free survival
[Time Frame: 6 months, 1 and 2 years]
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overall survival
[Time Frame: 6 months, 1 and 2 years]
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Secondary ID(s)
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2014OC034
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MT2014-10C
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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