NCT04637282

Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3

A Randomized. Multicenter, Double-Blind, Placebo-Controlled Safety, Tolerability, and Efficacy Study of PLX-200 in Participants With Mild-to-Moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 39 (estimated)
• Sponsor: Polaryx Therapeutics, Inc. · Industry
• Sex: All
• Age: 6 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and efficacy of multiple doses of PLX-200 in patients with CLN3 disease.

Detailed description

This is a phase 3, double-blind, placebo-controlled, dose-titration study to evaluate escalating weight-based dose levels of PLX-200, provided as a solution that contains 15 mg/mL PLX-200 and administered orally using a syringe, as needed, twice daily (BID), 30 minutes before breakfast and dinner. Participants will enter the Titration Period, during which the starting dose of PLX-200 or placebo will be based on patient weight. Each patient's dose will be titrated upward on a weekly basis during the Titration Period, until he or she reaches a maximally tolerated dose (MTD) or the Week 5 dose for their weight category. The patient will then enter the Maintenance Period at the final Titration Period dose for a maximum of 60 weeks. Safety, efficacy, and pharmacokinetics will be assessed periodically. Thereafter, all patients will have the opportunity to receive active treatment in an Open-Label Extension (OLE) for an additional 36 weeks.

Conditions

• Juvenile Neuronal Ceroid Lipofuscinosis

Interventions

Type	Name	Description
DRUG	PLX-200	15 mg/mL oral solution of experimental drug
DRUG	Placebo	Taste and color-matched drug-free solution

Timeline

• Start date: 2026-03-01
• Primary completion: 2026-03-31
• Completion: 2026-03-31
• First posted: 2020-11-19
• Last updated: 2025-05-30

Regulatory

• FDA-regulated drug study