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A Phase 2a Study of TPN-101 in Patients with Aicardi-Goutières Syndrome (AGS)
2024-511176-32-00Phase 2Completed
- Sponsor
- Transposon Therapeutics, Inc.
- Start
- Sep 2024
- Modality
- Small Molecules
- Countries
- 2
01
Overview
Phase 2, single-arm trial of censavudine in Aicardi-Goutières syndrome (AGS), sponsored by Transposon Therapeutics, Inc. Primary endpoints: Reduction in innate immune signaling (interferon score in whole blood) and Incidence and severity of treatment-emergent adverse events. Started Sep 2024; status: completed.
02
Drugs
censavudine
03
Conditions
Aicardi-Goutières syndrome (AGS)
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