Status: Not yet recruiting
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
Participants needed: 24
Trial details
Phase: Phase 2Age: 2-15Biological sex: AllType: InterventionalSponsor: Polaryx Therapeutics, Inc.Updated: Jul 31, 2026
Eligibility criteria
Male or female participants aged 2 to 15 years at the time of informed consent.... [+5]
The participant has a known inherited neurologic disease other than the targeted... [+21]