Status: Recruiting
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
Participants needed: 10
Trial details
Phase: Phase 1, Phase 2Age: Up to 17Biological sex: AllType: InterventionalSponsor: JCR Pharmaceuticals Co., Ltd.Updated: Sep 2, 2025Locations: 3
Eligibility criteria
Chronological age of <18 years [+1]
Prior experience to gene therapy or HSCT with successful engraftment [+6]