CompletedPHASE1, PHASE2NCT01380990

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

Studying Adenosine Deaminase Deficiency

Last synced from ClinicalTrials.gov May 3, 2026

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Key facts

Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
Principal Investigator: Claire Booth, Dr
Great Ormond Street Hospital NHS Foundation Trust
Intervention: Infusion of autologous EFS-ADA LV CD34+ cells(genetic)
Enrollment: 36 enrolled
Eligibility: 15 years · All sexes
Timeline: 2012 – 2019

Study locations (1)

Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom

Collaborators

Orchard Therapeutics

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT01380990 on ClinicalTrials.gov

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