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

Key facts

Sponsor

University of California, Los Angeles

Principal Investigator

Donald B. Kohn, MD

University of California, Los Angeles

Intervention

Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)(genetic)

Enrollment

10 enrolled

Eligibility

17 years · All sexes

Timeline

2016 – 2019

Study locations (1)

• University of California, Los Angeles, Los Angeles, California, United States

Collaborators

California Institute for Regenerative Medicine (CIRM) · 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.

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