Gene Therapy for ADA-SCID

Plain-language summary

This study looks at a gene therapy in which a healthy gene is delivered into the patient's own cells for ADA-SCID, an inherited disease that severely affects the immune system. People who had no HLA-matched sibling donor and who did not benefit enough from enzyme replacement therapy, or for whom that therapy was not a lifelong option, could take part. The study was carried out in Phase 1/Phase 2 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

PHASE1/PHASE2

Status

Completed

Sponsor

IRCCS San Raffaele

Intervention

gene transduced PBL and/or gene transduced HSC (gene therapy)

Start date

3/1/1992

Estimated completion

1/1/2007

Related genes

Eligibility criteria

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

Inclusion Criteria:

* Lack of HLA-identical sibling donor and
* Evidence of failure of the enzyme replacement treatment after \>6 months or
* PEG-ADA is not available as a life long option

Exclusion Criteria:

* HLA identical bone marrow sibling donor
* HIV infection
* Malignancy