Ignite Creation Date:
2024-05-05 @ 11:24 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00023010
Status:
COMPLETED
Last Update Posted:
2017-07-02
First Post:
2001-08-18
Brief Title:
Gene Therapy for Patients With Leukocyte Adherence Deficiency Follow-Up of Phase 1 Trial
Sponsor:
National Cancer Institute NCI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Follow-Up of a Phase-I Gene Therapy Trial of Patients With Leukocyte Adherence Deficiency
Status:
COMPLETED
Status Verified Date:
2010-12-13
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 patients 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
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 patients 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
Detailed Description:
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 archiving for potential testing for replication competent retrovirus and for complete blood counts Patients will also undergo a history and physical examination at these visits Results from these tests will be forwarded to the investigators who will report to the NIH and FDA
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 01-C-0236 | None | None | None |