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

• Register: ClinicalTrials.gov
• Last refreshed on: 9 June 2025
• Main ID: NCT04637282
• Date of registration: 04/11/2020
• Prospective Registration: Yes
• Primary sponsor: Polaryx Therapeutics, Inc.
• Public title: Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3
• Scientific title: 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
• Date of first enrolment: March 1, 2026
• Target sample size: 39
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04637282
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Contacts

• Name: Luis Rojas, MSc, PhD.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. 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.

2. 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.

3. 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.

4. Participant must be able to independently walk for a distance of at least 20 feet (6
meters).

5. Participant must be able to tolerate swallowing oral medication.

6. 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.

7. 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.

8. 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:

1. 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).

2. 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).

3. Participant has another inherited neurologic disease in addition to CLN3 disease.

4. Participant has another neurological illness that may cause cognitive or motor
decline.

5. Participants with enteral feeding with NG tubing and any difficulty in oral
administration and/or absorption of study drug will be excluded.

6. Participant requires ventilation support, except for noninvasive support at night
(e.g., Continuous Positive Airway Pressure [CPAP], Bilevel Positive Airway Pressure
[BiPAP]).

7. 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.

8. Participant has anemia (defined as hemoglobin <10 g/dL or hematocrit <30%).

9. Participant has a baseline serum creatinine >2 mg/dL.

10. Participant has gallbladder disease (e.g., cholelithiasis or cholecystitis).

11. Participant has hypersensitivity to gemfibrozil.

12. Participant is using or requires treatment with 1. HMG-CoA reductase inhibitors, 2.
repaglinide (Prandin®), 3. dasabuvir (Exviera®), 4. selexipag (Uptravi®), or 5.
pioglitazone (Actos®).

13. Since the participant may take anticoagulants, increased frequency of INR monitoring
is essential to avoid potential toxic effects with concurrent PLX-200 and
anticoagulants (in particular with warfarin).

14. Participant has a medical condition or personal circumstance that, in the opinion of
the Investigator, might compromise the participant's or parent/guardian's ability to
comply with the protocol requirements, or compromise the participant's wellbeing,
safety, or the interpretability of the study data.

15. Participant has received any investigational product or medical device within 30
days of the Baseline visit that, in the Investigator's judgment, would make the
participant ineligible or confound results. All subjects who have had an
investigational product or products in the form of stem cell or gene therapy are
excluded, regardless of when the therapy had been initiated and/or discontinued.


Age minimum: 6 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Juvenile Neuronal Ceroid Lipofuscinosis

Intervention(s)

Drug: Placebo

Drug: PLX-200

Primary Outcome(s)

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]

Number of patients with treatment-related adverse events, as assessed by CTCAE v5.0, abnormal laboratory results, and abnormal cardiovascular and/or abdominal findings. [Time Frame: 96 weeks]

Secondary Outcome(s)

Efficacy of PLX in each of the domains of the Hamburg Scale [Time Frame: 24, 48, 60, 72 and 96 weeks]

Evaluation of the baseline motor score decline between PLX-200 and placebo [Time Frame: 60 and 96 weeks]

Assessment of changes in the baseline Vineland Behavior Scale between PLX-200 and Placebo groups [Time Frame: 24, 48, 60, 72, and 96 weeks]

Assessment of changes in the baseline Montreal Assessment Cognitive Scale between PLX-200 and Placebo groups [Time Frame: 24, 48, 60, 72, and 96 weeks]

Assessment of the overall decline status in subjects treated with PLX-200 compared with the placebo group [Time Frame: 60 and 96 weeks]

Assessment of changes in the baseline Clinical Impression of symptom severity between PLX-200 and Placebo groups [Time Frame: 24, 48, 60, 72, and 96 weeks]

PLX-200 pharmacokinetics (PK) will be evaluated through periodic blood draws during the trial. PLX-200 levels will be determined to estimate area under the curve, maximal concentration, time to maximal concentration, half-life, and dosing interval [Time Frame: 96 weeks]

Assessment of changes in the baseline Pediatric Balance Scale between PLX-200 and Placebo groups [Time Frame: 24, 48, 60, 72, and 96 weeks]

Assessment of changes in the baseline walking ability of the patient between PLX-200 and Placebo groups [Time Frame: 24, 48, 60, 72, and 96 weeks]

Secondary ID(s)

PLX-200-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors