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

NCT04637282 · Status: NOT_YET_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 39

Last updated 2026-05-08

No results posted yet for this study

Summary

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

Conditions

  • Juvenile Neuronal Ceroid Lipofuscinosis

Interventions

DRUG

PLX-200

15 mg/mL oral solution of experimental drug

DRUG

Placebo

Taste and color-matched drug-free solution

Sponsors & Collaborators

  • Polaryx Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Lisa Bollinger, MD · Chief Medical Officer

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-11-01
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

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Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04637282 on ClinicalTrials.gov