Not Yet RecruitingPhase 2

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

24 participantsStarted 2026-12-01

Plain-language 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.

Who can participate

Age range

2 Years – 15 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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:
. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: 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.

Timeframe: 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.

Timeframe: 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.

Timeframe: Week 102

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

Timeframe: 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.

Timeframe: Week 102

Trial details

NCT IDNCT07740512

SponsorPolaryx Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-12-31

Contact for this trial

Minsu Kang, PhD

217-779-7736 minsukang@polaryx.com

View on ClinicalTrials.gov More Lysosomal Storage Disorders trials

Who can participate

Age range

2 Years – 15 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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:
. 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.

What they're measuring

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

Timeframe: 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.

Timeframe: 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.

Timeframe: 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.

Timeframe: Week 102

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

Timeframe: 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.

Timeframe: Week 102