Gene Therapy for ADA-SCID

Related Clinical Trial
cliniMACs HUD for T Cell Depletion Cord Blood Stem Cell Transplantation Study (COBLT) Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency Genetic Basis of Immunodeficiency IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency Sirolimus Prophylaxis for aGVHD in TME SCID Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow Allograft Influences on Female Adolescents’ Decisions Regarding Testing for Carrier Status of XSCID Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant EZN-2279 in Patients With ADA-SCID AMG191 Conditioning/CD34+CD90 Stem Cell Transplant Study for SCID Patients Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Newborn Screening for Severe Combined Immunodeficiency (SCID) in a High-Risk Population Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) Patients Treated for SCID (1968-Present) Clinical Characteristics and Genetic Profiles of Severe Combined Immunodeficiency in China Gene Therapy for ADA-SCID MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim Natural History Study of SCID Disorders Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency SCID Bu/Flu/ATG Study With T Cell Depletion Generalized Neonatal Screening of Severe Combined Immunodeficiencies Neonatal Screening of Severe Combined Immunodeficiencies Gene Therapy for X-linked Severe Combined Immunodeficiency An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) Registry Study of Revcovi Treatment in Patients With ADA-SCID Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID

Brief Title

Gene Therapy for ADA-SCID

Official Title

Treatment of ADA-SCID by Gene Therapy on Somatic Cells

Brief 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.
    

Detailed 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
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene

Secondary Outcome

 Evaluation of extent, kinetic and duration of the engraftment of transduced cells and the potential selective advantage of ADA positive cells

Condition

Severe Combined Immunodeficiency Syndrome

Intervention

gene transduced PBL and/or gene transduced HSC

Study Arms / Comparison Groups

 PBL/HSC
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Genetic

Estimated Enrollment

8

Start Date

March 1992

Completion Date

January 2007

Primary Completion Date

July 2006

Eligibility Criteria

        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
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Bordignon Claudio, MD, , 



Administrative Informations


NCT ID

NCT00599781

Organization ID

150291



Study Sponsor

IRCCS San Raffaele

Collaborators

 Fondazione Telethon

Study Sponsor

Bordignon Claudio, MD, Principal Investigator, IRCCS San Raffaele


Verification Date

December 2007