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Clinical Trial 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 (Elongation Factor 1α Short form) Lentiviral Vector (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 Human Leukocyte Antigen (HLA) matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who successfully receive the OTL-101 product, pegademase bovine (PEG-ADA) Enzyme Replacement Therapy (ERT) is discontinued at Day+30 (-3/+15) 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.

Clinical Trial Description


Study Design

Related Conditions & MeSH terms

NCT number NCT02999984
Study type Interventional
Source Orchard Therapeutics
Status Completed
Phase Phase 1/Phase 2
Start date December 16, 2016
Completion date September 26, 2019

See also
  Status Clinical Trial Phase
Suspended NCT04140539 - A Clinical Study to Enable Process Validation of Commercial Grade OTL-101 Phase 2/Phase 3
Active, not recruiting NCT03765632 - Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID Phase 1/Phase 2
Enrolling by invitation NCT04959890 - Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy
Enrolling by invitation NCT03232203 - Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)