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

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ClinicalTrials.gov Identifier: NCT00598481
Recruitment Status : Active, not recruiting
First Posted : January 22, 2008
Last Update Posted : June 2, 2017
Sponsor:
Information provided by (Responsible Party):

January 10, 2008
January 22, 2008
June 2, 2017
October 2, 2002
July 10, 2011   (Final data collection date for primary outcome measure)
survival [ Time Frame: minimum of 1 year ]
Same as current
Complete list of historical versions of study NCT00598481 on ClinicalTrials.gov Archive Site
  • Change in the rate of severe infection [ Time Frame: During follow up ]
  • T-lymphocyte counts [ Time Frame: one year ]
  • Modification of the systemic metabolic defect [ Time Frame: one year ]
  • presence of genetically modified cells in the BM and PB [ Time Frame: one year ]
Same as current
Not Provided
Not Provided
 
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
This is a phase I/II protocol to evaluate the safety and efficacy of ADA gene transfer into hematopoietic stem/progenitor cells for the treatment of adenosine deaminase (ADA)-deficiency. This condition is an autosomal recessive form of Severe Combined Immunodeficiency (SCID) characterized by impaired immune responses, recurrent infections, failure to thrive and systemic toxicity due to accumulation of purine metabolites. Transplants from an HLA-identical sibling donor is the treatment of choice, but available for a minority of patients. The use of alternative bone marrow donors or enzyme replacement therapy is associated with important drawbacks. The drug product studied in this protocol consists of autologous CD34+ hematopoietic stem/progenitor cells engineered ex vivo with a retroviral vector encoding the therapeutic gene ADA. The engineered CD34+ cells are infused following a nonmyeloablative conditioning with busulfan to make space in the bone marrow. The study objectives are: a) to evaluate the safety and the clinical efficacy of gene therapy, in the absence of enzyme replacement therapy; b) to evaluate the biological activity (engraftment, ADA expression) of ADA transduced CD34+ cells and their hematopoietic progeny. c) to evaluate the immunological reconstitution and purine metabolism after gene therapy.

The safety of the study will be evaluated by description of all adverse events and adverse drug reactions.

The study is aimed at reaching the minimum sample size of ten patients.

Long term follow up all patients enrolled into the study will have a long term follow period from 4 to 8 years after gene therapy

Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Immunologic Deficiency Syndromes
Genetic: Gene transduced CD34+ cells
infusion of autologous CD34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with Busulphan
Other Name: Gene therapy
Experimental: CD34+ cells
infusion of autologous CD34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with Busulphan
Intervention: Genetic: Gene transduced CD34+ cells

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
18
June 30, 2019
July 10, 2011   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • ADA-SCID with no HLA-identical sibling donor
  • 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 Long term follow-up
  • Patients who have received treatment with the Medicinal Product, either as part of the main clinical study, or previous pilot studies or compassionate use program

Exclusion Criteria:

  • HIV infection
  • history or current malignancy
  • Patients who received a previous gene therapy treatment in the 12 months prior to receiving GSK2696273
  • any other conditions dangerous for the patients according to the investigator
Sexes Eligible for Study: All
up to 17 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Israel,   Italy
 
 
NCT00598481
115611
15386-PRE21 ( Other Identifier: IRCCS San Raffaele )
No
Not Provided
Not Provided
GlaxoSmithKline
GlaxoSmithKline
Not Provided
Study Director: GSK Clinical Trials GlaxoSmithKline
GlaxoSmithKline
May 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP