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

Sponsor
Polaryx Therapeutics, Inc.
Study ID
NCT04637282
Phase
PHASE3
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

Eligibility Criteria

Sex
ALL
Age
6 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • PLX-200 — DRUG
    15 mg/mL oral solution of experimental drug
  • Placebo — DRUG
    Taste and color-matched drug-free solution

Study Details

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

Key Dates

Start date
Nov 1, 2026
Status verified
May 2025
Primary completion
Dec 31, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
39 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: PLX-200
    This is randomized, placebo-controlled comparator study of PLX-200 in patients with CLN3 disease.
  • Placebo Comparator: Placebo
    This is randomized comparator study of PLX-200 vs. placebo in a 2:1 ratio in patients with CLN3 disease.

Primary Outcome Measure

Efficacy of PLX-200 in CLN3 as assessed by the change in the motor score of the Hamburg Rating Scale compared with that of the Placebo group [ Time Frame: 60 weeks ]

Central Contacts