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Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

StatusNot Yet Recruiting
PhasePhase 2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study First Submitted
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
Trial Details
NCT Number NCT07740512
Lead Sponsor Polaryx Therapeutics, Inc.
Conditions
(from sponsor)		 Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease, CLN2, CLN3
Related terms Leukodystrophy, Globoid Cell, Lysosomal Storage Diseases, Sandhoff Disease
Enrollment 24 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov