|
Protocol Number:
01-C-0236
- Title:
Follow-Up of a Phase-I Gene Therapy Trial of Patients with Leukocyte Adherence Deficiency
- Number:
01-C-0236
- Summary:
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.
- Sponsoring Institute:
-
National Cancer Institute (NCI)
- Recruitment Detail
- Type:
No longer recruiting/follow-up only
- Gender:
Male & Female
- Referral Letter Required:
No
- Population Exclusion(s):
None
- Eligibility Criteria:
This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
- Special Instructions:
Currently Not Provided
- Keyword(s):
-
CD18
-
Retrovirus
-
RCR
-
Stem Cell
-
Gene Transfer
- Recruitment Keyword(s):
-
Leukocyte Adhesion Deficiency
-
LAD
- Condition(s):
-
Leukocyte Adhesion Deficiency Syndrome
- Investigational Drug(s):
- None
- Investigational Device(s):
- None
- Interventions:
- None
- Supporting Site:
-
National Cancer Institute
- Contact(s):
-
This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
- Citation(s):
-
The severe and moderate phenotypes of heritable Mac-1, LFA-1 deficiency: their quantitative definition and relation to leukocyte dysfunction and clinical features
-
Leukocyte adhesion deficiency: an inherited defect in the Mac-1, LFA-1, and p150,95 glycoproteins
If you have:
Search The Studies | Help | Questions | Clinical Center Home | NIH Home
National Institutes of Health Clinical Center
Bethesda, Maryland 20892. Last update: 01/30/2009
|
|