Lysosomal Storage DisordersPhase 2NCT07740512
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
A Phase 2 study testing PLX-200 in people with Lysosomal Storage Disorders and Sandhoff Disease, run by Polaryx Therapeutics, Inc..
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 15 years
- Conditions the study is for
- Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease
- What is being tested?
- PLX-200
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- No sites listed yet
- Study dates
- 1 Dec 2026to 1 Feb 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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)
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
Read the detailed description
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.
- Conditions
- Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease, CLN2, CLN3
- Being tested
- PLX-200
- Keywords
- CLN3, CLN2, Krabbe Disease, Pediatric, LSD
- Medical subject headings
- Lysosomal Storage Diseases, Sandhoff Disease, Leukodystrophy, Globoid Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PLX-200Drug
Also called Gemfibrozil
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).
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: 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) Receives: 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) Receives: 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) Receives: Drug: PLX-200 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Week 102
To evaluate the change from baseline vital signs in study participants 2 to 15 years old with LSDs during the treatment period.
Measured: 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.
Measured: 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).
Measured: Week 102
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (VABS-3) Score
To evaluate the clinical activity of PLX-200 in pediatric patients with late-infantile Krabbe/Sandhoff/CLN2/CLN3 disease using the Vineland Adaptive Behavior Scales, Third Edition (VABS-3), over 101 weeks of treatment.
Measured: Week 102
Caregiver Global Impression of Severity
To evaluate clinical activity of PLX-200 as reported by the caregiver. Change from baseline to Week 102 in Caregiver Global Impression of Severity (CaGI-S).
Measured: Week 102
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study start1 Dec 2026 (estimated)
- Primary completion31 Dec 2028 (estimated)
- Study completion1 Feb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lysosomal Storage DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Polaryx Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07740512
- Sponsor's protocol ID
- PLX-200-600
- Status last verified
- Jul 2026
- First submitted
- 17 Jul 2026
- Last updated
- 31 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07740512
- Acronym / sponsor ID
- PLX-200-600
- Conditions
- Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease, CLN2, CLN3
- Keywords
- CLN3, CLN2, Krabbe Disease, Pediatric, LSD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Polaryx Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Dec 2026 (estimated)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 1 Feb 2029 (estimated)
- First posted
- 31 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| PLX-200 (CLN3 Disease Cohort) | Experimental | Drug: PLX-200An 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) |
| PLX-200 (CLN2 Disease Cohort) | Experimental | Drug: PLX-200An 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) |
| PLX-200 (Sandhoff Disease Cohort) | Experimental | Drug: PLX-200An 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) |
| PLX-200 (Krabbe Disease Cohort) | Experimental | Drug: PLX-200An 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) |
Who may qualify
- Ages
- 2 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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:
- 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.
Key exclusion criteria (23 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PLX-200Also: GemfibrozilPLX-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). | Drug | PLX-200 (CLN2 Disease Cohort), PLX-200 (CLN3 Disease Cohort), PLX-200 (Krabbe Disease Cohort), PLX-200 (Sandhoff Disease Cohort) |
Primary outcome
To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.
Time frame: Until 30 days after the last administration of the study drug.
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.
To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.
Time frame: 30 days after the last administration of the study drug
Incidence and severity of serious adverse events (SAEs) until 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.
Time frame: 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.
Time frame: Week 102
To evaluate the change from baseline vital signs in study participants 2 to 15 years old with LSDs during the treatment period.
Time frame: 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.
Time frame: Week 102
3 secondary outcomes
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).
Time frame: Week 102
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (VABS-3) Score
Time frame: Week 102
Caregiver Global Impression of Severity
Time frame: Week 102
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PLX-200 in people with Lysosomal Storage Disorders and Sandhoff Disease, run by Polaryx Therapeutics, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Minsu Kang, PhD (217-779-7736, minsukang@polaryx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period. (time frame: Until 30 days after the last administration of the study drug.). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2026.