ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID

Plain-language summary

This study evaluates a gene therapy (Strimvelis) in which the ADA gene is inserted into the patient's own blood stem cells, for Severe Combined Immunodeficiency caused by ADA enzyme deficiency (ADA-SCID). It included pediatric patients without an HLA-matched sibling donor for whom enzyme replacement was insufficient. It was a Phase 2 study 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 2

Status

Completed

Sponsor

Fondazione Telethon

Intervention

Gene Therapy, Busulfan

Start date

10/2/2002

Estimated completion

6/19/2019

Countries

Israel, Italy

Related genes

Locations

• Jerusalem, Israel
• Milan, Italy

Eligibility criteria

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

Inclusion Criteria:

* ADA-SCID with no HLA-identical sibling donor available
* pediatric age and at least one of the following criteria:
* inadequate immune response after PEG-ADA for \> 6 months
* patients who discontinued PEG-ADA due to intolerance, allergy or auto-immunity
* patients for whom enzyme replacement therapy is not a life long therapeutic option

Exclusion Criteria:

* HIV infection
* history or current malignancy
* Patients who received a previous gene therapy treatment in the 12 months prior to receiving Strimvelis
* any other conditions dangerous for the patients according to the investigator