Gene Therapy ADA Deficiency

Great Ormond Street Hospital for Children NHS Foundation Trust8 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Adenosine Deaminase Deficiency

Interventions

Intravenous infusion of transduced cellsBIOLOGICAL

Intravenous infusion of transduced cells

Eligibility

Sex: ALLMax age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Great Ormond Street Hospital for Children NHS Trust

London, United Kingdom

Outcomes

Primary Outcomes

Immunological reconstitution

Measurement of Immunological reconstitution and Metabolic Correction. 5 year follow up of the last patient enrolled into study

Time frame: 5 years

Secondary Outcomes

Incidence of adverse reactions

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

Time frame: 5 years

Molecular characterisation of gene transfer

Molecular characterisation of gene transfer. 5 year follow up of the last patient enrolled into study

Time frame: 5 years

Normalisation of nutritional status, growth, and development

Normalisation of nutritional status, growth, and development. 5 year follow up of the last patient enrolled into study

Time frame: 5 years

Data from ClinicalTrials.gov

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