Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study

Plain-language summary

This study examined a gene transfer therapy and the natural course of disease in patients with severe combined immunodeficiency (SCID) caused by adenosine deaminase (ADA) deficiency. Patients with ADA deficiency could enroll for evaluation of their immune system and pre-treatment testing. It was carried out as a study with no assigned phase 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

N/A

Status

Completed

Sponsor

National Human Genome Research Institute (NHGRI)

Intervention

ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes

Start date

9/1/1990

Estimated completion

7/1/2002

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.

New patients will not be treated under protocol 90-HG-0195 as new and improved vectors and technologies have become available in the recent years.

New patients with ADA deficiency, however, may be enrolled in protocol 90-HG-0195 for clinical evaluation of their immune system and pre-treatment testing of transduction procedures.