Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

Plain-language summary

This study examined a gene therapy (human glucocerebrosidase cDNA) delivered through viruses and blood-forming cells for Gaucher's disease and Fabry disease. Both healthy volunteers and patients with these conditions could take part. The study was carried out in Phase 1 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Completed

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Intervention

human glucocerebrosidase cDNA

Start date

1/1/1988

Estimated completion

4/1/2002

Countries

United States

Related genes

Locations

• Bethesda, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.