Gene Therapy ADA Deficiency

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2003-10-31

Study Completion Date

2013-11-30

Brief Summary

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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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Detailed Description

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Conditions

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Adenosine Deaminase Deficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Intravenous infusion of transduced cells

Group Type EXPERIMENTAL

Intravenous infusion of transduced cells

Intervention Type BIOLOGICAL

Intravenous infusion of transduced cells

Interventions

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Intravenous infusion of transduced cells

Intervention Type BIOLOGICAL

Eligibility Criteria

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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

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No