Gene Therapy for the Treatment of Fanconi's Anemia Type C
Completed · Phase 1
Conditions studied: Fanconi's Anemia, Pancytopenia
In brief
Fanconi's Anemia is an inherited disorder that can produce bone marrow failure. In addition, some patients with Fanconi's anemia have physical defects usually involving the skeleton and kidneys. The major problem for most patients is aplastic anemia, the blood counts for red blood cells, white blood cells, and platelets are low because the bone marrow fails to produce these cells. Some patients with Fanconi's anemia can develop leukemia or cancers of other organs. Many laboratory studies have suggested that Fanconi's anemia is caused by an inherited defect in the ability of cells to repair DNA. Recently, the gene for one of the four types of Fanconi's anemia, type C, has been identified. It is known that this gene is defective in patients with Fanconi's anemia type C. Researchers have conducted laboratory studies that suggest Fanconi's anemia type C may be treatable with gene therapy. Gene therapy works by placing a normal gene into the cells of patients with abnormal genes responsible for Fanconi's anemia type C. After the normal gene is in place, new normal cells can develop and grow. Drugs can be given to these patients kill the remaining abnormal cells. The new cells containing normal genes and will not be harmed by these drugs. The purpose of this study is to test whether researchers can safely place the normal Fanconi's anemia type C gene into cells of patients with the disease. The gene will be placed into special cells in the bone marrow called stem cells. These stem cells are responsible for producing new red blood cells, white blood cells, and platelets.
Key facts
- Study ID
- NCT00001399
- Run by
- National Heart, Lung, and Blood Institute (NHLBI)
- People needed
- 9
- Starts
- 1993-12-03
- Expected to finish
- 2009-02-11
- Last updated by the study team
- 2017-07-02
Who can join
Age: 5 and older. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Patients who meet any one of the following criteria will be excluded from study entry:
- Patients presenting with acute leukemia or bone marrow aspirate revealing greater than 10 percent blasts.
- Pregnant or lactating females (all patients must practice adequate birth control and females of child-bearing potential must have a negative serum beta-HCG pregnancy test (within Day -7 to Day 0).
- Acute infection: any acute viral, bacterial, or fungal infection which requires specific therapy. Acute therapy must have been completed within 14 days prior to study treatment.
- Hepatitis-B surface antigen positive patients.
- HIV-infected patients.
- Acute medical problems such as ischemic heart or lung disease that may be considered an unacceptable anesthetic or operative risk.
- No patients with any underlying conditions which would contraindicate therapy with study treatment (or allergies to reagents used in this study).
- Patients less than 25 kg in weight .
- Patients who elect bone marrow transplantation.
Where it is running
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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