Status:
COMPLETED
Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia
Lead Sponsor:
Boston Children's Hospital
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Children's Hospital Medical Center, Cincinnati
Conditions:
Fanconi Anemia
Eligibility:
All Genders
1-35 years
Phase:
PHASE1
Brief Summary
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the bone marrow cells that produce various types of blood cells. Individuals with FA may ...
Detailed Description
FA is a rare, inherited disease that is caused by a gene defect and that primarily affects an individual's bone marrow, resulting in decreased production of blood cells. The lack of white blood cells ...
Eligibility Criteria
Inclusion
- FA, as determined by a positive test for increased sensitivity to chromosomal breakage with mitomycin C or diepoxybutane
- FA complementation group A, as determined by somatic cell hybrids or molecular characterization; transduction of peripheral blood or bone marrow cells with the complementation group of specific retrovirus used in this study must demonstrate correction of mitomycin C sensitivity or cell cycle arrest
- Weighs at least 7.5 kg
- Normal cytogenetics on bone marrow within 3 months of study entry
- A minimum of 2 x 10(6) CD34+ cells/kg after CD34+ selection of the harvested bone marrow or mobilized peripheral blood product must be available to proceed with thaw (if cryopreserved) and transduction
- Human leukocyte antigen (HLA) typing with initial donor limited search results that indicate a potentially acceptable matched unrelated donor in the National Marrow Donor Program database
Exclusion
- Cancer
- Clonal cytogenetic abnormality on bone marrow or peripheral blood karyotype within 3 months of study entry
- Myelodysplastic syndrome based on the FAB classification including:
- Refractory anemia with ringed sideroblasts (RARS)
- Refractory anemia with excess blasts (RAEB)
- RAEB in transformation (RAEB-T)
- Chronic myelomonocytic leukemia (CMML) (myelodysplastic changes in greater than two cell lines, refractory anemia alone, or aplastic anemia with dysplastic changes are permitted)
- Positive baseline screening result for both of the following:
- Detection of Fanconi A proviral sequences by polymerase chain reaction (PCR) analysis
- Detection of replication competent retrovirus by repeat testing by PCR of gibbon ape leukemia virus (GALV) envelope sequence or a positive S+L- assay
- Pregnant or breastfeeding; women of childbearing potential who are enrolled will be advised that the drug may cause birth defects and will be required to use an acceptable form of contraception
- Concurrent enrollment in any other study using an investigational agent, excluding androgens and thyroxine
- Physical or emotional status that would prevent informed consent, protocol compliance, or adequate follow-up with participant or legal guardian
- Participants for whom an acceptable HLA identical matched sibling donor (HLA A, B, DRB1; 6/6 match) has been identified (HLA typing of normal siblings must be documented)
Key Trial Info
Start Date :
August 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2007
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00272857
Start Date
August 1 2004
End Date
October 1 2007
Last Update
June 23 2017
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229