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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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11 July 2016 |
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Main ID: |
NCT01082133 |
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Date of registration:
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05/03/2010 |
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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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Multicenter Transplant Study for Fanconi Anemia
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Scientific title:
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A Multicenter 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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October 2009 |
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Target sample size:
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45 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01082133 |
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Study type:
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Interventional |
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Study design:
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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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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Stella Davies, MBBS |
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Address:
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Telephone:
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Email:
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Affiliation:
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CCHMC |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Confirmed Diagnosis of Fanconi Anemia
- One of the following:Severe Aplastic Anemia or Severe Single Lineage Cytopenia;
Myelodysplastic Syndrome; Acute Myelogenous Leukemia
- Unrelated and Related Donors
- Adequate Physical Function (Cardiac, Hepatic, Renal, Pulmonary)
- Available for Long-Term Follow Up
- Performance status >= 70%
Exclusion Criteria:
- Co-existing medical problems that increase the risk of transplant
- Active CNS (central nervous system) leukemic involvement
- Pregnant or Breastfeeding (Females)
- Active, Uncontrolled Infection
- HIV/HTLV (Human T-lymphotropic virus)Positive
Age minimum:
N/A
Age maximum:
N/A
Gender:
Both
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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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Drug: Chemotherapy
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Biological: Miltenyi CliniMACS
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Primary Outcome(s)
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The primary objectives are: The incidence and quality of engraftment and hematopoietic reconstitution. The incidence of early transplant related mortality The incidence and severity of acute GvHD (graft versus host disease) and chronic GvHD.
[Time Frame: 1-5 years]
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Secondary Outcome(s)
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The incidence of overall survival and disease free survival over time.
[Time Frame: 5 Years]
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Secondary ID(s)
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Multicenter FA SCT
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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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