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

NCT07740512 · clinicaltrials.gov ↗

PHASE2

Phase

NOT_YET_RECRUITING

Status

24

Enrollment

INDUSTRY

Sponsor class

Conditions

Lysosomal Storage Disorders
Sandhoff Disease
Krabbe Disease
CLN2
CLN3

Interventions

DRUG: PLX-200

Sponsor

Polaryx Therapeutics, Inc.