Clinical Trials

Clinical Trial Detail

Return to search results.

Gene Therapy for Fanconi Anemia

Complete title: Gene Transfer for Patients with Fanconi Anemia Complementation Group A (FANCA)

Research Study Number       2097.00
    
Principal Investigator       Pamela Becker, MD, PhD
    
Phase       I

Look up trial at NIH

Research Study Description

This pilot trial will access the toxicity and efficacy of infusion of gene modified cells 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.

Eligibility Criteria (must meet the following to participate in this study)

Ages Eligible for Study: 4 Years and older

Genders Eligible for Study: Both

- 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 complementary deoxyribonucleic acid (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

- Absolute neutrophil count >= 0.5 X 10^9/L

- Hemoglobin >= 8 g/dL

- Platelet count >= 20 X 10^9/L and able to achieve a platelet count of >= 50 X 10^9/L with transfusion support

- Adequate hepatic function with aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 5 X upper limit of normal (ULN)

- Adequate renal function with creatinine (Cre) =< 1.5; if greater, then glomerular filtration rate (GFR) > 60 mL/min/ 1.73 m^2 as calculated by the Modification of Diet in Renal Disease equation

- Adequate pulmonary function with corrected diffusion capacity of carbon monoxide (DLCO) > 50%

- For subjects < 17 years of age, Modified Lansky Play-Performance Score of >= 70%; for subjects 17 and older, Karnofsky score of >= 70%

Other eligibility criteria may apply.

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

- Acute myeloid leukemia as defined by WHO criteria

- Pregnancy or lactation; females of childbearing potential and males who are admitted to the study will be advised that the study procedures and study drugs may be teratogenic, and they 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

- Patients for whom an human leukocyte antigen (HLA) matched sibling donor bone marrow transplant is being actively pursued will not be eligible for study until it is determined that no sibling donor is available or that a stem cell transplant is not feasible during the time the patient might be on study

- ** No patient will be included in this study as an alternative to a clinically indicated HLA matched sibling donor stem cell transplant

- ** If an HLA matched sibling donor is identified, but stem cell or marrow collection is not feasible (e.g., donor is in utero, is a newborn from whom cord blood was not collected, or is unable to undergo a donation procedure because of ill health), a patient may be included in the study at the discretion of the investigators

- 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 [NYHA] class II), poorly controlled diabetes (hemoglobin A1c [Hgb A1c] > 7%), coronary angioplasty within 6 months, myocardial infarction within the last 6 months, or uncontrolled atrial or ventricular cardiac arrhythmia, abnormal coagulation, persistent abnormal urinalysis reflecting intrinsic renal disease

- Active ongoing viral, bacterial, or fungal infection

Other exclusion criteria may apply.



Research Study Number       2097.00
    
Contact       Seattle Cancer Care Alliance Intake Office
    
Telephone       800-804-8824 / 206-288-1024
    
   

Keywords
Diamond Blackfan Anemia; Fanconi Anemia; Non-malignant Condition

Disclaimer: We update this information regularly. However, what you read today may not be completely up to date.

Please remember:

  • Talk to your health care providers first before making decisions about your health care.
  • 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.

Subscribe to an RSS feed of all trials

Fred Hutchinson Cancer Research Center is a world leader in research to prevent, detect and treat cancer and other life-threatening diseases.