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SOTERIA: PLX-200 in Pediatric Patients With Lysosomal Storage Disorders
NCT07740512Phase 2Not yet recruiting
- Sponsor
- Polaryx Therapeutics
- Start
- Dec 2026
- Primary completion
- Dec 2028
01
Overview
Phase 2, open-label trial of PLX-200 in lysosomal storage disorder (LSD), neuronal ceroid lipofuscinosis (NCL), Sandhoff disease and 1 more (4 cohorts), sponsored by Polaryx Therapeutics. Primary endpoints: Safety and Tolerability of PLX-200, Change from baseline in clinical laboratory parameters at Week 102 and 3 more. Planned start Dec 2026; status: not yet recruiting.
02
Drugs
PLX-200
03
Conditions
lysosomal storage disorder (LSD)neuronal ceroid lipofuscinosis (NCL)Sandhoff diseaseKrabbe disease
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