Clinical Trial

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

Name: Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) — clinical trial record (NCT07740512)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Not Yet Recruiting Phase 2

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Summary

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	Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease, CLN2, CLN3
Enrollment	24 participants
Start Date	2026-12-01
Primary Completion	2028-12-31 (estimated)
Study Completion	2029-02-01 (estimated)
Updated on ClinicalTrials.gov	2026-07-31