List All Bone Marrow Transplant Trials

Bone Marrow Transplant

Gene Therapy for Fanconi Anemia (2097)
Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)
Status Conditions Phase Study ID
Recruiting Fanconi Anemia Complementation Group A Phase I 2097

This pilot trial will assess the toxicity and efficacy of infusion of gene modified cells, as well as the feasibility of mobilization of peripheral blood stem cells with filgrastim and plerixafor for patients with Fanconi anemia (FA). Infusion of autologous patient blood stem cells that have been corrected in the laboratory by introduction of the normal gene may improve blood counts in patients with FA.

Pamela Becker, MD, PhD
Seattle Cancer Care Alliance 800-804-8824  
Eligibility Criteria (must meet the following to participate in this study)
  • FA demonstrated by a positive test for increased sensitivity to chromosomal breakage with mitomycin C or diepoxybutane performed by a Clinical Laboratory Improvement Amendments (CLIA) or College of American Pathologists (CAP) approved laboratory
  • FA complementation group A as determined by somatic cell hybrids, molecular characterization, Western blot analysis, or acquisition of mitomycin C resistance after in vitro lentiviral transduction with a vector bearing the cDNA for Fanconi complementation group A
  • Bone marrow analysis demonstrating normal cytogenetics, and no more than 5% of cells with a single clonal abnormality by fluorescence in situ hybridization (FISH) for myelodysplastic syndrome (MDS) panel within 3 months of stem cell collection
  • Signed informed consent by the patient or legally authorized representative
  • Platelet count > 20,000 and able to achieve a platelet count of > 50,000 with transfusion support
  • For subjects < 17 years of age, Modified Lansky Play-Performance Score of >= 70%; for subjects 17 and older, Karnofsky score of >= 70%
Exclusions (conditions that would prevent participation in this study)
  • Non-hematopoietic malignancy where the expected survival is less than 2 years
  • Myelodysplastic syndrome as defined by World Health Organization (WHO) criteria
  • Pregnancy or lactation; women with childbearing potential who are admitted to the study will be advised that the study procedures and study drugs may be teratogenic; women of child-bearing potential will be required to take adequate measures to prevent conception for the duration of the study
  • Concurrent enrollment in any other study using an investigational drug
  • Physical or emotional status that would prevent informed consent, protocol compliance, or adequate follow-up
  • Significant associated diseases including documented human immunodeficiency virus (HIV) infection, uncontrolled hypertension (diastolic blood pressures > 95%ile for age), unstable angina, congestive heart failure (> New York Heart Association [NY] II), poorly controlled diabetes, coronary angioplasty within 6 months, myocardial infarction within the last 6 months, or uncontrolled atrial or ventricular cardiac arrhythmia
  • Active ongoing viral, bacterial, or fungal infection
Last Updated
January 04, 2012
See this trial at
Access protocol and consent forms at Fred Hutchinson Cancer Research Center
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  • Whether you are eligible for a research study depends on many things. There are specific requirements to be in research studies. These requirements are different for each study.