Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With ADA Deficienc
Brief Summary: 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 stem/progenitor cells transduced ex vivo with EFS LV encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an HLA-matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product will be infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who have successfully received the OTL-101 product, PEG-ADA ERT will be discontinued at Day+30 (+/-3) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.