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

> **NCT07740512** · PHASE2 · NOT_YET_RECRUITING · sponsor: **Polaryx Therapeutics, Inc.** · enrollment: 24 (estimated)

## Conditions studied

- Lysosomal Storage Disorders
- Sandhoff Disease
- Krabbe Disease
- CLN2
- CLN3

## Interventions

- **DRUG:** PLX-200

## Key facts

- **NCT ID:** NCT07740512
- **Lead sponsor:** Polaryx Therapeutics, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** NOT_YET_RECRUITING
- **Start date:** 2026-12-01
- **Primary completion:** 2028-12-31
- **Final completion:** 2029-02-01
- **Target enrollment:** 24 (ESTIMATED)
- **Last updated:** 2026-07-31


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07740512

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07740512, "Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)". Retrieved via MORVS 2026-10-20 from https://api.morvs.ai/clinical/NCT07740512. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*