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Study identifier: NCT07740512 Synced from ClinicalTrials.gov · July 31, 2026
● Not Yet Recruiting

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

Condition: Lysosomal Storage Disorders · Sandhoff Disease · Krabbe Disease  ·  Sponsor: Polaryx Therapeutics, Inc.

PhasePhase 2
Planned participants24
Who can joinAll sexes, 2 Years to 15 Years
Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Minsu Kang, PhD  ·  217-779-7736  ·  minsukang@polaryx.com

Always discuss trial participation with your own doctor first.

Locations (0)

Locations not yet listed on the registry record.

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Source record: clinicaltrials.gov/study/NCT07740512