The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
Male or female participants aged 2 to 15 years at the time of informed consent. Any deviations must be approved in advance by the Medical Monitor and Sponsor.
Age of symptom onset consistent with the targeted subtype,
Relevant clinical manifestations, and
Documented genotype at Screening or prior to enrollment. If no genotype is available at Screening, blood samples will be collected for genetic analysis as part of study procedures.
The participant has a known inherited neurologic disease other than the targeted lysosomal storage disorder subtype.
The participant has a neurological illness unrelated to the study indication that may independently cause cognitive or motor decline.
The participant requires ventilatory support, except for noninvasive support during sleep (e.g., Continuous Positive Airway Pressure [CPAP], Bilevel Positive Airway Pressure [BiPAP]).
The participant has moderate or severe hepatic dysfunction, defined as alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin greater than 3 times the upper limit of normal (ULN), except in cases of Gilbert syndrome. The participant has a diagnosis of primary biliary cirrhosis.
Single group
PLX-200
DrugPLX-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 96-week Maintenance Period) based on each participant's weight. Dosing will begin with a 5-week TP based on the participant's weight group to achieve a target maintenance dose (TMD).
To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.
Incidence and severity of treatment-emergent adverse events (TEAEs) until 30 days after the last administration of the study drug as well as withdrawals due to the TEAEs.
To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.
Incidence and severity of serious adverse events (SAEs) until 30 days after the last administration of the study drug.
To evaluate the change from baseline in clinical laboratory parameters in study participants 2 to 15 years old with LSDs during the treatment period.
To evaluate the change from baseline in physical examination results in study participants 2 to 15 years old with LSDs during the treatment period.
To evaluate the clinical activity of PLX-200 in study participants 2 to 15 years old with LSDs as measured by each respective instrument as specified in the Intervention Specific Assessment (ISA).
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (VABS-3) Score
To evaluate the clinical activity of PLX-200 in pediatric patients with late-infantile Krabbe/Sandhoff/CLN2/CLN3 disease using the Vineland Adaptive Behavior Scales, Third Edition (VABS-3), over 101 weeks of treatment.
Caregiver Global Impression of Severity
To evaluate clinical activity of PLX-200 as reported by the caregiver. Change from baseline to Week 102 in Caregiver Global Impression of Severity (CaGI-S).
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