Gene Therapy for ADA-SCID

Severe Combined Immunodeficiency Syndrome Clinical Trial

Official title:

Treatment of ADA-SCID by Gene Therapy on Somatic Cells

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00599781
• Other study ID #: 150291
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: January 8, 2008
• Last updated: January 23, 2008
• Start date: March 1992
• Est. completion date: January 2007

Study information

• Verified date: December 2007
• Source: IRCCS San Raffaele
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: National Bioethics Committee
• Study type: Interventional

Clinical Trial 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.

Description:

This is mono-centric, non-randomized, non-controlled, open label, phase I-II trial that evaluated the safety and efficacy of ADA gene transfer into somatic cells for the treatment of ADA-SCID

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: January 2007
• Est. primary completion date: July 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Severe Combined Immunodeficiency
• Severe Combined Immunodeficiency Syndrome

Intervention

Genetic:
• gene transduced PBL and/or gene transduced HSC
infusions of autologous PBL and/or HSC transduced with retroviral vectors encoding ADA

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
IRCCS San Raffaele	Fondazione Telethon

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene			Yes
Secondary	Evaluation of extent, kinetic and duration of the engraftment of transduced cells and the potential selective advantage of ADA positive cells			No
Secondary	Evaluation of efficacy of the administration of autologous PBL/HSC(Clinical, immunological, hematological, microbiological, ADA activity and purine metabolism)			No
Secondary	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 gene therapy