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Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial) Clinical Trials Information presented on Clinical Trials Search isn't intended to be a substitute for proven healthcare advice, trips or treatment using a real physician. We are not docs. Always confer with your mD on Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial) conditions. Clinical Trials Search.org is a site dedicated to listing clinical research studies in human subjects. Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial) Clinical research trials and Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial) 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 measure the effectiveness of new drugs. The intention of the studies / projects is to resolve certain human health questions. Clinical trials are a popular means for physicians, government agencies, and private sector corporations to detect remedies for all forms of circumstances, like Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial). Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial) Clinical Trials and other clinical trials allow for volunteers to undergo healthcare treatment options before they are available to the masses. Most times the participants receive treatment for free, and every now and again they are paid for their time. Occasionally there is a cost for a Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial) clinical trial. Subjects typically recieve the finest healthcare available for their Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial) condition. Hazards are a reality, nonetheless, and might include more or frequent mD trips, health risks (potentially life-endangering), and/or the treatment being ineffective. Trials are federally regulated with stern guidelines to protect clinical trials subjects.

Home > "G" Clinical Trials Conditions > Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)

Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)



Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)

For Condition: Leukocyte Adhesion Deficiency Syndrome
Status: No longer recruiting
Sponsor(s): National Cancer Institute (NCI) ,
Synopsis: This study will provide long-term monitoring of two patients who received gene therapy for leukocyte adherence deficiency (LAD) under the Food and Drug Administration investigational new drug study BB-IND-7949. The IND protocol has been closed. No other patients are eligible for this study. Patients previously enrolled in BB-IND-7949 (Retrovirus-Mediated Transfer of the cDNA for Human CD18 into Peripheral Blood Repopulating cells of Patients with Leukocyte Adherence Deficiency) will be followed at least yearly for an indefinite period of time to evaluate their medical status and look for treatment side effects. The follow-up visits at the NIH Clinical Center will involve the following: - Interview regarding health status during the past year - Blood draw of approximately 15 milliliters for 3 years, then 5 ml annually thereafter for studies related to LAD and to make sure no unexpected effects of gene therapy have occurred The blood samples collected at the follow-up visits will be frozen and stored. If a serious medical problem arises, the sample may be checked for replication competent virus. If the gene therapy is suspected to be related to a medical problem, investigation may include a review of the patient's medical records or collection of additional blood or tissues for testing. If the patient should die, the family will be asked permission to perform an autopsy, regardless of the cause of death. Tissues taken at autopsy will be tested for any long-term effects from the gene therapy.
Details: Patients with leukocyte adhesion deficiency or LAD have a deficiency of the leukocyte integrin CD18 on their leukocyte cell surface and suffer from severe bacterial infections. Two patients with LAD were enrolled in a Phase I clinical trial conducted at the University of Washington School of Medicine in 1999 to determine the safety and potential efficacy of using retroviral vectors containing CD18 to transduce and correct their CD34+ peripheral blood stem cells. This protocol aims to continue the long-term follow-up of these patients at the National Cancer Institute where Dr. Hickstein is now a Senior Investigator. Blood samples will be collected at the follow-up visits on the yearly anniversary of the treatment date (either locally or off-site by the patients' personal physicians) for testing for replication competent retrovirus, CD18 expression, and complete blood counts. Patients will also undergo a physical examination at these visits. Results from these tests will be forwarded to the investigators who will report to the NIH and FDA.
Eligibility:
Study Type:  Observational, Natural History
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: Two patients treated under FDA IND BB-IND-7949.
Total Enrollment: 2

Location and Contact Information:

National Cancer Institute (NCI)
Bethesda,  Maryland,  20892
United States
 


Additional Information:
Study ID Numbers:  010236;  01-C-0236
Study Start Date: August 15, 2001
Record last reviewed: August 1, 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00023010

Other Leukocyte Adhesion Deficiency Syndrome Studies:
1. Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I

2. Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders

3. Phase I Study of Retrovirus-Mediated Transfer of cDNA for Human CD18 into Peripheral Blood Repopulating Cells of Patients With Leukocyte Adherence Deficiency

4. Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)

5. Use of G-CSF to Obtain Blood Cell Precursors

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