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Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells


Phase 1
N/A
N/A
Not Enrolling
Both
Gaucher's Disease

Thank you

Trial Information

Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells


This protocol was developed in order to obtain bone marrow stem cells for ex vivo
transduction with retroviruses containing the human glucocerebrosidase gene. We continue to
enter a small number of patients to this protocol each year. Studies with the bone marrow
hematopoietic progenitor cells have enabled us to identify the most effective retroviral
construct currently available in order to carry out gene therapy trials in patients with
Gaucher's disease. The data revealed that a comparatively simple retroviral construct
containing human glucocerebrosidase cDNA driven by the MoLV promoter is highly effective.
We have obtained approval and initiated a Phase I safety and gene marking investigation in
patients with Type I Gaucher's Disease.

Inclusion Criteria


Normal and patient volunteers.

Individuals with platelet counts less than 40,000/ul, PT greater than 15 seconds, or PTT
greater than 40 seconds will not undergo bone marrow aspiration.

Individuals with hematologic disorders other than Gaucher Disease, Fabry Disease, or mild
iron deficiency will not undergo bone marrow aspiration.

HIV positive individuals will be excluded from participating.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety Study, Primary Purpose: Treatment

Authority:

United States: Federal Government

Study ID:

880019

NCT ID:

NCT00001234

Start Date:

January 1988

Completion Date:

April 2002

Related Keywords:

  • Gaucher's Disease
  • Bone Marrow
  • Retroviral-Mediated Gene Transfer
  • Hematopoietic Progenitor Cell
  • Gaucher Disease

Name

Location

National Institute of Neurological Disorders and Stroke (NINDS)Bethesda, Maryland  20892