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Gene Therapy ADA Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01279720
Recruitment Status : Completed
First Posted : January 19, 2011
Last Update Posted : September 14, 2015
Sponsor:
Information provided by (Responsible Party):
Great Ormond Street Hospital for Children NHS Foundation Trust

Tracking Information
First Submitted Date  ICMJE January 18, 2011
First Posted Date  ICMJE January 19, 2011
Last Update Posted Date September 14, 2015
Study Start Date  ICMJE October 2003
Actual Primary Completion Date November 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 18, 2011)
Immunological reconstitution [ Time Frame: 5 years ]
Measurement of Immunological reconstitution and Metabolic Correction. 5 year follow up of the last patient enrolled into study
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01279720 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 18, 2011)
  • Incidence of adverse reactions [ Time Frame: 5 years ]
    Incidence of adverse reactions. 5 year follow up of the last patient enrolled into study
  • Molecular characterisation of gene transfer [ Time Frame: 5 years ]
    Molecular characterisation of gene transfer. 5 year follow up of the last patient enrolled into study
  • Normalisation of nutritional status, growth, and development [ Time Frame: 5 years ]
    Normalisation of nutritional status, growth, and development. 5 year follow up of the last patient enrolled into study
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Gene Therapy ADA Deficiency
Official Title  ICMJE Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency
Brief Summary Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection. This trial aims to treat adenosine deaminase deficiency patients using gene therapy.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Adenosine Deaminase Deficiency
Intervention  ICMJE Biological: Intravenous infusion of transduced cells
Intravenous infusion of transduced cells
Study Arms  ICMJE Experimental: Intravenous infusion of transduced cells
Intravenous infusion of transduced cells
Intervention: Biological: Intravenous infusion of transduced cells
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 11, 2015)
8
Original Estimated Enrollment  ICMJE
 (submitted: January 18, 2011)
10
Actual Study Completion Date  ICMJE November 2013
Actual Primary Completion Date November 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Patients who lack a human leukocyte antigen (HLA)-genotypically identical bone marrow donor OR phenotypically matched family or unrelated donor AND who show incomplete immune reconstitution on Polyethylene glycol-modified adenosine deaminase (PEG-ADA) enzyme replacement therapy (defined by absolute CD4+ count <300 cell/mm3 and who remain on immunoglobulin replacement therapy)
  2. Diagnosis of ADA-SCID (Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA)confirmed by DNA sequencing OR by confirmed absence of <3% of ADA enzymatic activity in peripheral blood or (for neonates) in umbilical cord blood erythrocytes and/or leukocytes or in cultured fetal cells derived from either chorionic villus biopsy or amniocentesis, prior to institution of PEG-ADA replacement therapy
  3. Parental/guardian/patient signed informed consent
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01279720
Other Study ID Numbers  ICMJE 03MI14
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Great Ormond Street Hospital for Children NHS Foundation Trust
Study Sponsor  ICMJE Great Ormond Street Hospital for Children NHS Foundation Trust
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Great Ormond Street Hospital for Children NHS Foundation Trust
Verification Date September 2015

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