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Gene Therapy for ADA-SCID

NCT00599781 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2008-01-24

No results posted yet for this study

Summary

This study investigated the safety and efficacy of different gene therapy approaches for Severe Combined Immunodeficiency (SCID) caused by the deficiency of adenosine deaminase (ADA) enzyme. This is a severe condition that can be cured by HLA-matched sibling donor bone marrow transplantation. Patients were enrolled if no HLA-identical sibling donor was available and the patient showed evidence of failure of enzyme replacement therapy or this treatment was not a long-term available option. The aim of the study was to evaluate the safety and efficacy of the procedure and to identify the relative role of peripheral blood lymphocytes and hematopoietic stem cells and progenitor cells in the long-term reconstitution of immune functions after retroviral vector mediated ADA gene transfer.

Conditions

  • Severe Combined Immunodeficiency Syndrome

Interventions

GENETIC

gene transduced PBL and/or gene transduced HSC

infusions of autologous PBL and/or HSC transduced with retroviral vectors encoding ADA

Sponsors & Collaborators

  • Fondazione Telethon

    collaborator OTHER

  • IRCCS San Raffaele

    lead OTHER

Principal Investigators

  • Bordignon Claudio, MD · IRCCS San Raffaele

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1992-03-31
Primary Completion
2006-07-31
Completion
2007-01-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00599781 on ClinicalTrials.gov