Gene Therapy ADA Deficiency

NHS Foundation Trust

Status and phase: Completed; Phase 2; Phase 1

Conditions: Adenosine Deaminase Deficiency

Treatments: Biological: Intravenous infusion of transduced cells

Study type: Interventional

Funder types: Other

Identifiers

NCT01279720

03MI14

Details and patient eligibility

About

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.

Enrollment: 8 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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8 participants in 1 patient group

Intravenous infusion of transduced cells

Experimental group

Description:

Intravenous infusion of transduced cells

Treatment:

Biological: Intravenous infusion of transduced cells