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

NCT07740512 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2026-07-31

No results posted yet for this study

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

Interventions

DRUG

PLX-200

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).

Sponsors & Collaborators

  • Polaryx Therapeutics, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-12-01
Primary Completion
2028-12-31
Completion
2029-02-01
FDA Drug
Yes

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07740512 on ClinicalTrials.gov