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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00023010
Recruitment Status : Completed
First Posted : August 20, 2001
Last Update Posted : July 2, 2017
Information provided by:
National Institutes of Health Clinical Center (CC)

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 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.

Condition or disease
Leukocyte Adhesion Deficiency Syndrome

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.

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Study Type : Observational
Actual Enrollment : 2 participants
Official Title: Follow-Up of a Phase-I Gene Therapy Trial of Patients With Leukocyte Adherence Deficiency
Study Start Date : August 15, 2001
Study Completion Date : December 13, 2010

Resource links provided by the National Library of Medicine

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Two patients treated under FDA IND BB-IND-7949.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00023010

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United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Cancer Institute (NCI)

Layout table for additonal information Identifier: NCT00023010     History of Changes
Obsolete Identifiers: NCT00004470
Other Study ID Numbers: 010236
First Posted: August 20, 2001    Key Record Dates
Last Update Posted: July 2, 2017
Last Verified: December 13, 2010

Keywords provided by National Institutes of Health Clinical Center (CC):
Stem Cell
Gene Transfer
Leukocyte Adhesion Deficiency

Additional relevant MeSH terms:
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Tissue Adhesions
Leukocyte-Adhesion Deficiency Syndrome
Pathologic Processes
Immunologic Deficiency Syndromes
Immune System Diseases