We updated the design of this site on September 25th. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

Gene Therapy ADA Deficiency

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01279720
First Posted: January 19, 2011
Last Update Posted: September 14, 2015
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.
Information provided by (Responsible Party):
Great Ormond Street Hospital for Children NHS Foundation Trust
  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: None (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 ]
    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 ]
    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


Enrollment: 8
Study Start Date: October 2003
Study Completion Date: November 2013
Primary Completion Date: November 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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01279720


Locations
United Kingdom
Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom, WC1N 1EH
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
First Submitted: January 18, 2011
First Posted: January 19, 2011
Last Update Posted: September 14, 2015
Last Verified: September 2015

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

Additional relevant MeSH terms:
Severe Combined Immunodeficiency
Agammaglobulinemia
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Blood Protein Disorders
Hematologic Diseases
Lymphoproliferative Disorders
Lymphatic Diseases
Adenosine
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Arrhythmia Agents
Vasodilator Agents
Purinergic P1 Receptor Agonists
Purinergic Agonists
Purinergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action