Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

NCT07740512 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: PLX-200-600
• Study Type: interventional
• Target: 24
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.

Conditions

CLN3; Lysosomal Storage Disorders; Sandhoff Disease; Krabbe Disease; CLN2

Keywords

CLN3; CLN2; Krabbe Disease; Pediatric; LSD

Outcome Measures

Primary Outcomes (6)

• To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.

  Incidence and severity of treatment-emergent adverse events (TEAEs) until 30 days after the last administration of the study drug as well as withdrawals due to the TEAEs.

  Until 30 days after the last administration of the study drug.

• To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.

  Incidence and severity of serious adverse events (SAEs) until 30 days after the last administration of the study drug.

  30 days after the last administration of the study drug

• To evaluate the change from baseline in clinical laboratory parameters in study participants 2 to 15 years old with LSDs during the treatment period.

  Week 102

• To evaluate the change from baseline in physical examination results in study participants 2 to 15 years old with LSDs during the treatment period.

  Week 102

• To evaluate the change from baseline vital signs in study participants 2 to 15 years old with LSDs during the treatment period.

  Week 102

• To evaluate the change from baseline in 12-lead electrocardiogram in study participants 2 to 15 years old with LSDs during the treatment period.

  Week 102

Secondary Outcomes (3)

• To evaluate the clinical activity of PLX-200 in study participants 2 to 15 years old with LSDs as measured by each respective instrument as specified in the Intervention Specific Assessment (ISA).

  Week 102

• Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (VABS-3) Score

  Week 102

• Caregiver Global Impression of Severity

  Week 102

Study Arms (4)

PLX-200 (CLN3 Disease Cohort)

EXPERIMENTAL

An Open-Label, Multicenter, Phase 2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients with CLN3 Disease (Juvenile Neuronal Ceroid Lipofuscinosis) (CLN3 Intervention-Specific Assessment \[ISA\] Under the PLX-200-600 Master Protocol)

Drug: PLX-200

PLX-200 (CLN2 Disease Cohort)

EXPERIMENTAL

An Open-Label, Multicenter, Phase 2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients with CLN2 Disease (Late-Infantile Neuronal Ceroid Lipofuscinosis) (CLN2 Intervention-Specific Assessment \[ISA\] Under the PLX-200-600 Master Protocol)

Drug: PLX-200

PLX-200 (Sandhoff Disease Cohort)

EXPERIMENTAL

An Open-Label, Multicenter, Phase 2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients with Sandhoff Disease (GM2 Gangliosidosis Type II) (Sandhoff Disease Intervention-Specific Assessment \[ISA\] Under the PLX-200-600 Master Protocol)

Drug: PLX-200

PLX-200 (Krabbe Disease Cohort)

EXPERIMENTAL

An Open-Label, Multicenter, Phase 2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients with Krabbe Disease (Globoid Cell Leukodystrophy) (Krabbe Disease Intervention-Specific Assessment \[ISA\] Under the PLX-200-600 Master Protocol)

Drug: PLX-200

Interventions

PLX-200 DRUG

PLX-200 will be BID, with equal doses given approximately 12 hours apart, 30 minutes before the morning and evening meals, for 101 weeks (5-week Titration Period and 96-week Maintenance Period) based on each participant's weight. Dosing will begin with a 5-week TP based on the participant's weight group to achieve a target maintenance dose (TMD).

Also known as: Gemfibrozil

PLX-200 (CLN2 Disease Cohort); PLX-200 (CLN3 Disease Cohort); PLX-200 (Krabbe Disease Cohort); PLX-200 (Sandhoff Disease Cohort)

Eligibility Criteria

• Age: 2 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female participants aged 2 to 15 years at the time of informed consent. Any deviations must be approved in advance by the Medical Monitor and Sponsor.
• Genetically confirmed diagnosis of one of the four LSDs included in this study: CLN2, CLN3, Sandhoff disease or Krabbe disease. Diagnosis must be supported by all of the following:
• Age of symptom onset consistent with the targeted subtype,
• Relevant clinical manifestations, and
• Documented genotype at Screening or prior to enrollment. If no genotype is available at Screening, blood samples will be collected for genetic analysis as part of study procedures.
• Written informed consent must be obtained from the participant's parent(s) or legal guardian(s). Assent must also be obtained from the participant, when applicable, in accordance with local regulations and the participant's developmental status.
• Parent(s) or legal guardian(s) must demonstrate willingness and ability to comply with the protocol, including adherence to all required baseline, treatment, and follow-up assessments.

You may not qualify if:

• The participant has a known inherited neurologic disease other than the targeted lysosomal storage disorder subtype.
• The participant has a neurological illness unrelated to the study indication that may independently cause cognitive or motor decline.
• The participant requires ventilatory support, except for noninvasive support during sleep (e.g., Continuous Positive Airway Pressure \[CPAP\], Bilevel Positive Airway Pressure \[BiPAP\]).
• The participant has moderate or severe hepatic dysfunction, defined as alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin greater than 3 times the upper limit of normal (ULN), except in cases of Gilbert syndrome. The participant has a diagnosis of primary biliary cirrhosis.
• The participant has clinically significant anemia
• The participant has a body surface area (BSA)-adjusted eGFR \<90 mL/min/1.73m2 at Screening or baseline.
• The participant has a history or current diagnosis of gallbladder disease (e.g., cholelithiasis or cholecystitis).
• The participant has a known hypersensitivity to gemfibrozil or any component of the study drug.
• The participant is currently using, or is expected to require during the study, any of the following medications which are contraindicated with PLX-200:
• HMG-CoA reductase inhibitors
• Repaglinide (Prandin®)
• Dasabuvir (Exviera®)
• Selexipag (Uptravi®)
• Pioglitazone (Actos®)
• Fibrate medication (e.g., gemfibrozil, fenofibrate). Participants must not have received gemfibrozil or other fibrates for at least 2 weeks or five half-lives, whichever is shorter, before Visit 2 (Day 1). They may not receive gemfibrozil or other fibrates during the study

Sponsors & Collaborators

• Polaryx Therapeutics, Inc.: lead

MeSH Terms

Conditions

Lysosomal Storage Diseases; Sandhoff Disease; Leukodystrophy, Globoid Cell

Interventions

Gemfibrozil

Condition Hierarchy (Ancestors)

Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Gangliosidoses, GM2; Gangliosidoses; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating 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

Central Study Contacts

Minsu Kang, PhD

CONTACT

217-779-7736; minsukang@polaryx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 17, 2026
• First Posted: July 31, 2026
• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): February 1, 2029
• Last Updated: July 31, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share