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Gene Therapy Trial Report

Summary

Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia


NCTID NCT00272857 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Fanconi Anemia Complementation Group A
Disease Ontology Term DOID:0111095
Compound Name MSCV-FANCA
Sponsor Boston Children's Hospital
Funder Type Other
Recruitment Status
Completed
Enrollment Count 3 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant FANCA
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type MSCV
Dose 1 3.5-4.5E5 CD34+ cells/kg

Study Record Dates


Current Stage Phase1
Submit Date 2006-01-04
Completion Date 2007-10
Last Update 2017-06-23

Participation Criteria


Eligible Age 1 Year - 35 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * 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 Criteria: * 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: 1. Refractory anemia with ringed sideroblasts (RARS) 2. Refractory anemia with excess blasts (RAEB) 3. RAEB in transformation (RAEB-T) 4. 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: 1. Detection of Fanconi A proviral sequences by polymerase chain reaction (PCR) analysis 2. 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)
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Clinical study did not demonstrate long-term engraftment or selective advantage

Resources/Links