Clinical trials: Adenosine

Severe Combined Immunodeficiency Due to ADA Deficiency

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

• Completed
• Age: 30 days to 17 years
• Phase 1/2
• Injection or infusion
• Results posted

This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic…

Trial name

NCT02999984

Testing

Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT

Where

United States

Volunteers

10 people

Sponsor

University of California, Los Angeles