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Follow-Up of a Phase-I Gene Therapy Trial of Patients With Leukocyte Adherence Deficiency

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Leukocyte Adhesion Deficiency Syndrome

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Trial Information

Follow-Up of a Phase-I Gene Therapy Trial of Patients With Leukocyte Adherence Deficiency

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.

Inclusion Criteria

- Two patients treated under FDA IND BB-IND-7949.

Type of Study:


Study Design:



United States: Federal Government

Study ID:




Start Date:

August 2001

Completion Date:

December 2010

Related Keywords:

  • Leukocyte Adhesion Deficiency Syndrome
  • CD18
  • Retrovirus
  • RCR
  • Stem Cell
  • Gene Transfer
  • Leukocyte Adhesion Deficiency
  • LAD
  • Tissue Adhesions
  • Leukocyte-Adhesion Deficiency Syndrome



National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland  20892