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Gene Therapy ADA Deficiency

NCT01279720 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2015-09-14

No results posted yet for this study

Summary

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.

Conditions

  • Adenosine Deaminase Deficiency

Interventions

BIOLOGICAL

Intravenous infusion of transduced cells

Intravenous infusion of transduced cells

Sponsors & Collaborators

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-10-31
Primary Completion
2013-11-30
Completion
2013-11-30

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01279720 on ClinicalTrials.gov