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

NCT04637282 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: PLX-200-002
• Study Type: interventional
• Target: 39
• Locations: 0 countries
• Sites: N/A

Brief 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

Keywords

CLN3; Batten disease; Children

Outcome Measures

Primary Outcomes (2)

• 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

  The change from baseline in the motor score of the Hamburg Rating Scale at Week 60 of maintenance therapy in participants treated with PLX-200 compared with that of Placebo group. Baseline is defined as the motor score of the Hamburg Rating Scale assessment prior to starting drug or Placebo group in the Titration Period. The Hamburg Rating Scale records a rating of 0 - 3 in four domains: motor skills, vision, language, and seizures, all from 0 - 3 which is worse to normal, respectively, with a minimum (worse) score of 0 to a maximum (normal) score of 12.

  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.

  The aggregate of clinical chemistries, hematology, urinalysis, electrocardiogram readings, abdominal ultrasound findings, and tabulation of the number and severity of adverse events will be compared with baseline values throughout the study to evaluate the safety and tolerability of PLX-200 at escalating oral doses in children with CLN3 disease.

  96 weeks

Secondary Outcomes (9)

• Efficacy of PLX in each of the domains of the Hamburg Scale

  24, 48, 60, 72 and 96 weeks

• Assessment of the overall decline status in subjects treated with PLX-200 compared with the placebo group

  60 and 96 weeks

• Evaluation of the baseline motor score decline between PLX-200 and placebo

  60 and 96 weeks

• Assessment of changes in the baseline Clinical Impression of symptom severity between PLX-200 and Placebo groups

  24, 48, 60, 72, and 96 weeks

• Assessment of changes in the baseline Pediatric Balance Scale between PLX-200 and Placebo groups

  24, 48, 60, 72, and 96 weeks

Study Arms (2)

PLX-200

EXPERIMENTAL

This is randomized, placebo-controlled comparator study of PLX-200 in patients with CLN3 disease.

Drug: PLX-200

Placebo

PLACEBO COMPARATOR

This is randomized comparator study of PLX-200 vs. placebo in a 2:1 ratio in patients with CLN3 disease.

Drug: Placebo

Interventions

PLX-200 DRUG

15 mg/mL oral solution of experimental drug

Also known as: Gemfibrozil

PLX-200

Placebo DRUG

Taste and color-matched drug-free solution

Placebo

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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%).
• Participant has a baseline serum creatinine \>2 mg/dL.
• Participant has gallbladder disease (e.g., cholelithiasis or cholecystitis).
• Participant has hypersensitivity to gemfibrozil.
• 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®).
• 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).
• 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.
• 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.

Sponsors & Collaborators

• Polaryx Therapeutics, Inc.: lead

MeSH Terms

Conditions

Neuronal Ceroid-Lipofuscinoses

Interventions

Gemfibrozil

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Fibric Acids; Isobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Pentanoic Acids; Valerates; Phenyl Ethers; Ethers; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Fatty Acids, Volatile; Fatty Acids; Lipids

Study Officials

• Lisa Bollinger, MD

  Chief Medical Officer

  STUDY DIRECTOR

Central Study Contacts

Lisa Bollinger, MD

CONTACT

(301) 922-5108; lisabollinger@polaryx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized, double-blind, placebo-controlled, within-subject, dose-titration design

Study Record Dates

• First Submitted: November 4, 2020
• First Posted: November 19, 2020
• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: May 8, 2026

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share