ClinicalTrials.gov record NCT07740512

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

An Open-Label, Multicenter, Phase 2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients With Lysosomal Storage Disorders (SOTERIA)

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age2 years old to 15 years old
SexAll
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label Phase 2 basket trial studies PLX-200 in children with select lysosomal storage disorders (CLN2, CLN3, Sandhoff, Krabbe). Participants receive oral PLX-200 twice daily for about 101 weeks (a 5-week titration then a 96-week maintenance). The study collects safety data, drug levels, and measures of clinical activity compared against synthetic controls.

What it is studying. The study is testing the safety and tolerability of PLX-200 in pediatric patients with specified lysosomal storage disorders, measuring pharmacokinetics (drug levels) and looking for signals of clinical activity using disease-specific assessments.

What the researchers hope to learn. Scientific goals are to evaluate safety and tolerability, characterize pharmacokinetics, track changes in lab tests, physical exam findings, vital signs, and ECG, and assess preliminary clinical activity using instruments such as the Vineland Adaptive Behavior Scales, Third Edition (VABS-3) and Caregiver Global Impression of Severity (CaGI-S). The study aims to generate data to inform future research in these disorders.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • PLX-200

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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
  • Participants receiving Zavesca® (miglustat) or any other prohibited therapies must be willing to discontinue these therapies, complete a washout period (2 weeks or five half-lives, whichever is shorter) prior to Visit 2 (Day 1), and refrain from receiving them while they are participating in the study. If participants were previously on Brineura®, they must complete a 3-month washout period prior to Visit 2 (Day 1) and refrain from receiving it while they are participating in the study.
  • The participant has a medical condition or personal circumstance that, in the opinion of the Investigator or Sponsor, could compromise safety, protocol compliance, or the interpretability of study data.
  • The participant has received any investigational product or medical device within 30 days prior to the baseline visit that could confound study results or pose additional risk. All participants who have previously received stem cell or gene therapy are excluded regardless of timing.
  • The participant receives systemic anticoagulant therapy (e.g., warfarin) and is unable or unwilling to comply with increased frequency of INR monitoring during study participation. Note: Participants may be eligible if receiving anticoagulants (e.g., warfarin) provided that INR can be monitored with increased frequency and dose adjustments are implemented to maintain therapeutic range and avoid bleeding complications.
  • The participant has uncontrolled seizures, defined as ≥4 generalized tonic-clonic seizures per month or a recent episode of status epilepticus.
  • The participant has severe central nervous system abnormalities (e.g., hydrocephalus, intracranial shunt).
  • The participant has a history of clinically significant arrhythmia or QTc prolongation at Screening or baseline.
  • The participant is pregnant or breastfeeding or is a female showing signs of pubertal development (e.g., Tanner Stage ≥2) who is unable or unwilling to undergo pregnancy testing at Screening or baseline. All such determinations must involve consultation with the Medical Monitor and follow the procedures outlined in each ISA.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This is an open-label, proof-of-concept, Phase 2 basket trial evaluating the safety, tolerability, and clinical activity of PLX-200 in pediatric participants with lysosomal storage disorders (LSDs) including CLN2, CLN3, Sandhoff disease, and Krabbe disease. Participants will receive PLX-200 oral solution twice daily (BID) for approximately 101 weeks, comprising of a 5-week Titration Period and 96-week Maintenance Period. Clinical activity will be evaluated using synthetic control comparisons. Dosing strategies and study durations for all other indications will be defined in their respective ISAs.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will take PLX-200 oral solution twice daily for about 101 weeks (a 5-week titration period followed by a 96-week maintenance period), with dosing based on each participant's weight. They will attend regular clinic visits for safety monitoring that include blood tests, physical exams, vital signs, and 12-lead ECGs, and study staff will record any side effects. Clinical activity will be measured with caregiver questionnaires and standardized assessments such as the Vineland Adaptive Behavior Scales (VABS-3) and the Caregiver Global Impression of Severity; if no prior genetic test is available, a blood sample will be taken for genetic analysis. Participants may need to stop certain prohibited medications and complete required washout periods before starting the study drug.

How the study is structured. This is an open-label, non-randomized, single-group Phase 2 master protocol with separate disease-specific cohorts (CLN2, CLN3, Sandhoff, Krabbe). All participants receive PLX-200 oral solution twice daily (about 12 hours apart, 30 minutes before meals) for approximately 101 weeks, starting with a 5-week titration period followed by a 96-week maintenance period. Clinical activity will be evaluated using synthetic control comparisons and by disease-specific Intervention Specific Assessments (ISAs). Dosing is weight-based as specified in the protocol.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • No sites are listed on the registry yet. Where will the study take place?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Krabbe disease

Clinical trials8 trials listed for Krabbe diseaseEvery trial the registry lists for Krabbe disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Krabbe disease researchLatest monthly update: September 2026. Includes: Transplant survival. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 1, 2026 (estimated)
Primary completion
Dec 31, 2028 (estimated)
Study completion
Feb 1, 2029 (estimated)
Target enrollment
24 participants (estimated)
Last updated on registry
Jul 31, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Polaryx Therapeutics, Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
at Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
legal guardian
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
Assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
eGFR
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
contraindicated
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
five half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
washout period
A period during which a participant stops a previous treatment before starting the study treatment, so the earlier treatment does not affect the results.
QTc
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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