U.S., Aug. 1 -- ClinicalTrials.gov registry received information related to the study (NCT07740512) titled 'Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)' on July 17.
Brief 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.
Study Start Date: Dec. 01, 2026
Study Type: INTERVENTIONAL
Condition:
Lysosomal Storage Disorders
Sandhoff Disease
Krabbe Disease
CLN2
CLN3
Intervention:
DRUG: PLX-200
PLX-200 will be BID, with equal doses given approximately 12 hours apart, 30 minutes before the morning and evening meals, for 101 weeks (5-week Titration Period and 9...