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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.
Register: ClinicalTrials.gov
Last refreshed on: 11 July 2016
Main ID:  NCT01082133
Date of registration: 05/03/2010
Prospective Registration: No
Primary sponsor: Children's Hospital Medical Center, Cincinnati
Public title: Multicenter Transplant Study for Fanconi Anemia
Scientific title: 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
Date of first enrolment: October 2009
Target sample size: 45
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT01082133
Study type:  Interventional
Study design:  Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Stella Davies, MBBS
Address: 
Telephone:
Email:
Affiliation:  CCHMC
Key inclusion & exclusion criteria

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
Health Condition(s) or Problem(s) studied
Fanconi Anemia
Intervention(s)
Drug: Chemotherapy
Biological: Miltenyi CliniMACS
Primary Outcome(s)
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]
Secondary Outcome(s)
The incidence of overall survival and disease free survival over time. [Time Frame: 5 Years]
Secondary ID(s)
Multicenter FA SCT
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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