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Gene Therapy for the Treatment of Fanconi's Anemia Type C Clinical Trials References presented on Clinical Trials Search is not intended to be a substitute for proven healthcare advice, trips or professional assistance by using a real medical. We aren't mDs. Always confer with your physician about Gene Therapy for the Treatment of Fanconi's Anemia Type C conditions. Clinical Trials Search.org is a website devoted to listing clinical research studies in human subjects. Gene Therapy for the Treatment of Fanconi's Anemia Type C Clinical research trials and Gene Therapy for the Treatment of Fanconi's Anemia Type C medical trials take place in hundreds of localities across the U.S.. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials usually evaluate the effectualness of new does drugs. The purpose of the studies / projects is to solve specific human health questions. Clinical trials are a popular way for physicians, government agencies, and private sector companies to discover treatments for all sorts of conditions, such as Gene Therapy for the Treatment of Fanconi's Anemia Type C. Gene Therapy for the Treatment of Fanconi's Anemia Type C Clinical Trials and other clinical trials permit volunteers to access healthcare treatment choices before they are available to the general public. Some times the subjects recieve professional assistance for without cost, and every now and again they are compensated for their time. Sometimes there is a cost for a Gene Therapy for the Treatment of Fanconi's Anemia Type C clinical trial. Subjects often receive the most expert healthcare possible for their Gene Therapy for the Treatment of Fanconi's Anemia Type C condition. Risks are a reality, nevertheless, and could include additional or frequent dr. calls, healthcare dangers (perhaps life-jeopardising), and/or the treatment being ineffective. Trials are federally governed with stern guidelines to protect clinical trials subjects.
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Home > "G" Clinical Trials Conditions > Gene Therapy for the Treatment of Fanconi's Anemia Type C  Gene Therapy for the Treatment of Fanconi's Anemia Type C
Gene Therapy for the Treatment of Fanconi's Anemia Type C
For Condition: Fanconi's Anemia,Pancytopenia
Status: No longer recruiting
Sponsor(s): National Heart, Lung, and Blood Institute (NHLBI) ,
Synopsis: 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.
Details: Fanconi anemia (FA) is a genetic syndrome manifested by bone marrow failure, physical anomalies, and cancer susceptibility. The disorder almost invariably leads to the development of pancytopenia in affected individuals, and most patients die from complications of the disease in young adulthood. Bone marrow transplantation (BMT) is the usual treatment for patients who have a suitable histocompatible donor. Patients who do not have a matched sibling donor are sometimes referred for matched unrelated BMT, following identification of a suitable donor through international volunteer registries. However, patients who undergo matched unrelated BMT for Fanconi anemia have a very high rate of graft failure (approximately 35%). For this reason, patients may undergo harvesting of autologous bone marrow cells: these cells are then reinfused if the patient rejects the allogeneic marrow graft. For complementation group C FA patients, we propose to genetically modify the autologous bone marrow sample by introduction of the normal FAC gene and to reinfuse these transduced cells back to the patient should he or she reject the allogeneic marrow graft.
Eligibility:
Study Type: Interventional, Treatment, Safety
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA: Patients must meet the following criteria within 30 days prior to study entry (Day 0) unless otherwise noted. Males or females, age greater than or equal to 5 years of age. Diagnosis of Fanconi anemia, complementation group C, as confirmed by 1) Diepoxybutane or mitomycin C testing and 2) DNA analysis indicating FACC mutations. Adequate baseline organ function as assessed by the following laboratory values within 30 days prior to study entry (day -30 to 0). Adequate renal function with estimated creatinine clearance greater than 50 ml/min. (This will be determined by serum creatinine and 24-hour urine creatinine ordered concurrently). Adequate liver function with SGOT, SGPT and alkaline phosphatase less than or equal to 5 times the ULN (if transaminases greater than the upper limit of normal (ULN), patients should have a hepatitis B surface antigen (HBsAG) test prior to study entry. Patients may not enter the study if HBsAG is positive). PT and PTT not more than 1.5 times the ULN. Serum Amylase less than or equal to 1.5 times the ULN. Bilirubin less than or equal to 3.0 mg/dL. Triglyceride less than 400 mg/dl. Ability to give informed consent. Normal cardiac function by history and exam. Resting transcutaneous oxygen saturation greater than 90 percent on room air. Karnofsky Performance Status greater than or equal to 40. Although there are no blood count criteria for inclusion in this study, preference will be given to patients with significant marrow failure as reflected by anemia, neutropenia, and/or thrombocytopenia. Furthermore, we intend to first enroll adults and older children, to the extent possible, before enrolling younger children. EXCLUSION CRITERIA: 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.
Total Enrollment: 9
Location and Contact Information:
National Heart, Lung and Blood Institute (NHLBI)
Bethesda, Maryland, 20892
United States
Additional Information:
Study ID Numbers: 940033; 94-H-0033
Study Start Date: December 3, 1993
Record last reviewed: December 5, 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00001399
Other Pancytopenia Studies:
1. Gene Therapy for the Treatment of Fanconi's Anemia Type C
2. Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Fanconi's Anemia
3. Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia
4. Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
5. Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
Related Studies:
Other Pancytopenia Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
Gene Therapy for the Treatment of Fanconi's Anemia Type C
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