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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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12 December 2020 |
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Main ID: |
NCT01071239 |
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Date of registration:
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20/05/2009 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Hematopoietic Stem Cell Transplant for Fanconi Anemia
FA |
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Scientific title:
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A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine |
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Date of first enrolment:
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April 2009 |
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Target sample size:
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1 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01071239 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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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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David A Margolis, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Medical College of Wisconsin |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one
of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic
Syndrome, Acute Myelogenous Leukemia
- Karnofsky or Lansy performance scale > or = to 70%.
- Must have adequate cardiac, hepatic, renal and pulmonary function.
- Must have 7/8 or 8/8 available unrelated donor.
Exclusion Criteria:
- Pregnant or breastfeeding.
- Active CNS leukemic involvement
- Active uncontrolled viral, bacterial or fungal infection
- Positive for HIV.
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Fanconi Anemia
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Intervention(s)
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Device: CliniMACs device
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Drug: ATG
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Drug: Busulfan
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Drug: Fludarabine
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Drug: Cyclophosphamide
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Primary Outcome(s)
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To measure the incidence and quality of engraftment and hematopoietic reconstitution.
[Time Frame: 1, 3, 6 and 12 months post transplant date]
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Secondary Outcome(s)
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The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
[Time Frame: weekly for the first 30 days and then 3, 6, and 12 months post transplant date]
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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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