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 range6 Years – 18 Years

SexALL

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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

✕. Participant has asymptomatic CLN3 disease, defined as no evidence of neurological signs or symptoms attributed to CLN3 disease such as seizures, ataxia, language delay, or other developmental delays. Similarly, outliers who progress much more slowly or quickly compared to the rest of the study population will be excluded from study at the discretion of the PI in consultation with the Medical Monitor (e.g., c.1A \> C start codon mutation).
✕. Participant has clinically documented generalized motor status epilepticus within 4 weeks of the Baseline visit (treatment may be postponed after discussion with the Medical Monitor until seizures are adequately controlled).
✕. Participant has another inherited neurologic disease in addition to CLN3 disease.
✕. Participant has another neurological illness that may cause cognitive or motor decline.
✕. Participants with enteral feeding with NG tubing and any difficulty in oral administration and/or absorption of study drug will be excluded.
✕. Participant requires ventilation support, except for noninvasive support at night (e.g., Continuous Positive Airway Pressure \[CPAP\], Bilevel Positive Airway Pressure \[BiPAP\]).
✕. Participant has moderate or severe hepatic dysfunction defined as alanine aminotransferase, aspartate aminotransferase, or total bilirubin \>3x upper limit of normal (ULN) except for participants with Gilbert syndrome. Participant has primary biliary cirrhosis.
✕. Participant has anemia (defined as hemoglobin \<10 g/dL or hematocrit \<30%).

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

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