Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Process Validation of OTL-101 in ADA-SCID
NCT04140539Phase 3Withdrawn
- Sponsor
- University of California, Los Angeles
- Start
- Oct 2019
- Primary completion
- Aug 2021
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of simoladagene autotemcel in severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), sponsored by University of California, Los Angeles. Primary endpoints: Adenosine Deaminase (ADA) enzyme activity at 6 months, T cell (CD3+) count at 6 months and 1 more. Planned start Oct 2019; status: withdrawn.
02
Drugs
simoladagene autotemcel
03
Conditions
severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)
Beyond the preview
Go deeper on NCT04140539.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT04140539.