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Completed

NCT Number: NCT01279720

Gene Therapy ADA Deficiency

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.

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Key information

Conditions

Adenosine Deaminase Deficiency Severe combined immunodeficiency due to adenosine deaminase deficiency

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Great Ormond Street Hospital for Children NHS Trust

London, WC1N 1EH, United Kingdom

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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)
  • 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
  • Parental/guardian/patient signed informed consent

Treatment and study plan

Intravenous infusion of transduced cells

Biological

Intravenous infusion of transduced cells

Primary outcomes

  1. 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 outcomes

  1. Incidence of adverse reactions

    Time frame: 5 years

    Incidence of adverse reactions. 5 year follow up of the last patient enrolled into study

  2. 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

  3. 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

Sponsors and collaborators

Lead sponsor

Great Ormond Street Hospital for Children NHS Foundation Trust

Other

Registry information

Official study title

Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency

Important dates

Study start
2003
Primary completion
2013
Study completion
2013
First posted
Jan 19, 2011
Registry last updated
Sep 14, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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