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Clinical Trial Summary

The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process.


Clinical Trial Description

The safety and efficacy of OTL-101 for the treatment of patients with ADA- SCID have been established in previous clinical trials. The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process in order to facilitate collection of data necessary for final manufacturing. Assessments will focus on monitoring safety and engraftment, through the evaluation of parameters describing immunological recovery, ADA enzyme activity and persistence of gene marking (VCN) at 6 months and 12 months. After completion of 12 months of follow-up on the current study protocol, subjects will be enrolled in an observational long-term follow-up study, in order to monitor the long-term safety of treatment with OTL-101. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04140539
Study type Interventional
Source University of California, Los Angeles
Contact
Status Withdrawn
Phase Phase 2/Phase 3
Start date October 15, 2019
Completion date August 30, 2021

See also
  Status Clinical Trial Phase
Completed NCT03765632 - Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID Phase 1/Phase 2
Completed NCT02999984 - Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID Phase 1/Phase 2
Active, not recruiting NCT04959890 - Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy
Completed NCT03232203 - Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)