Not Yet RecruitingPhase 3

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

39 participantsStarted 2026-11-01

Plain-language summary

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

Who can participate

Age range

6 Years – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Male and female participants between the ages of 6 and 18 years of age. Any deviations from this age range must be approved by the Medical Monitor and Sponsor prior to entry into study.
. Has a diagnosis of "classic" CLN3 disease as determined by age of symptom onset (i.e., 4 to 7 years) and genetic analysis for a defect in the CLN3 (battenin) transmembrane gene at study entry. If no genotype information is available, blood will be collected for the CLN3 gene analysis at the Screening visit.
. Participant must have mild-to-moderate CLN3 disease documented by a total in the 3-domain score of 5 to 7 for the aggregate of the motor, language, and vision domains of the Hamburg Scale and a score of at least 2 in 2 of these 3 domains.
. Participant must be able to independently walk for a distance of at least 20 feet (6 meters).
. Participant must be able to tolerate swallowing oral medication.
. Participants who are of childbearing potential (i.e., have begun menstruation) must have a negative serum pregnancy test at Baseline before receiving PLX-200. Nursing mothers are excluded from participation in this study.
. Participants' parents/guardians must agree to comply in good faith with the conditions of the study, including attending all required baseline and follow-up assessments.
. Participant parents and legal guardians must sign the informed consent form, and participants will provide assent, depending on local regulations and developmental status.

Exclusion criteria

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: 60 weeks

Number of patients with treatment-related adverse events, as assessed by CTCAE v5.0, abnormal laboratory results, and abnormal cardiovascular and/or abdominal findings.

Timeframe: 96 weeks

Trial details

NCT IDNCT04637282

SponsorPolaryx Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-12-31

Contact for this trial

Lisa Bollinger, MD

(301) 922-5108 lisabollinger@polaryx.com

View on ClinicalTrials.gov More Juvenile Neuronal Ceroid Lipofuscinosis trials