CompletedPHASE1, PHASE2NCT03765632

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Studying Severe combined immunodeficiency

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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 cryopreserved EFS-ADA LV CD34+ cells (OTL-101)(genetic)
Enrollment
13 enrolled
Eligibility
17 years · All sexes
Timeline
20182022

Study locations (1)

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 NCT03765632 on ClinicalTrials.gov

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