Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

Completed · Phase 1

Conditions studied: Fanconi Anemia

In brief

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 experience fatigue, bleeding, and increased infections. The purpose of this study is to evaluate the safety and effectiveness of a gene transfer procedure in generating new, healthy cells in individuals with FA.

Key facts

Study ID

NCT00272857

Run by

Boston Children's Hospital

People needed

3

Starts

2004-08-01

Expected to finish

2007-10-01

Last updated by the study team

2017-06-23

Who can join

Age: 1 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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)

Where it is running

• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.