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Gene Therapy ADA Deficiency

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2012 by Great Ormond Street Hospital for Children NHS Foundation Trust
Sponsor:
Information provided by (Responsible Party):
Great Ormond Street Hospital for Children NHS Foundation Trust
ClinicalTrials.gov Identifier:
NCT01279720
First received: January 18, 2011
Last updated: March 1, 2012
Last verified: March 2012
  Purpose

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.


Condition Intervention Phase
Adenosine Deaminase Deficiency
Biological: Intravenous infusion of transduced cells
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency

Resource links provided by NLM:


Further study details as provided by Great Ormond Street Hospital for Children NHS Foundation Trust:

Primary Outcome Measures:
  • Immunological reconstitution [ Time Frame: 5 years ] [ Designated as safety issue: No ]
    Measurement of Immunological reconstitution and Metabolic Correction. 5 year follow up of the last patient enrolled into study


Secondary Outcome Measures:
  • Incidence of adverse reactions [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
    Incidence of adverse reactions. 5 year follow up of the last patient enrolled into study

  • Molecular characterisation of gene transfer [ Time Frame: 5 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Normalisation of nutritional status, growth, and development. 5 year follow up of the last patient enrolled into study


Estimated Enrollment: 10
Study Start Date: October 2003
Estimated Study Completion Date: October 2018
Estimated Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intravenous infusion of transduced cells
Intravenous infusion of transduced cells
Biological: Intravenous infusion of transduced cells
Intravenous infusion of transduced cells

  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01279720

Contacts
Contact: Bobby Gaspar, Professor 020 7905 2319 H.Gaspar@ich.ucl.ac.uk

Locations
United Kingdom
Great Ormond Street Hospital for Children NHS Trust Recruiting
London, United Kingdom, WC1N 1EH
Contact: Bobby Gapar, Professor    020 7905 2319    H.Gaspar@ich.ucl.ac.uk   
Principal Investigator: Bobby Gaspar, Professor         
Sponsors and Collaborators
Great Ormond Street Hospital for Children NHS Foundation Trust
  More Information

Publications:
Responsible Party: Great Ormond Street Hospital for Children NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT01279720     History of Changes
Other Study ID Numbers: 03MI14
Study First Received: January 18, 2011
Last Updated: March 1, 2012
Health Authority: United Kingdom: Research Ethics Committee

Keywords provided by Great Ormond Street Hospital for Children NHS Foundation Trust:
Adenosine deaminase deficiency
Gene therapy

Additional relevant MeSH terms:
Agammaglobulinemia
Severe Combined Immunodeficiency
Blood Protein Disorders
DNA Repair-Deficiency Disorders
Hematologic Diseases
Immune System Diseases
Immunologic Deficiency Syndromes
Infant, Newborn, Diseases
Lymphatic Diseases
Lymphoproliferative Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on November 24, 2014