Status: Not yet recruiting
A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.
Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Age: Up to 5Biological sex: AllType: InterventionalSponsor: JCR Pharmaceuticals Co., Ltd.Updated: Jul 7, 2026Locations: 5
Eligibility criteria
Patients with confirmed MPS IIIB with a phenotype of MPS IIIB that is not classi... [+2]
Contraindication for lumbar puncture or MRI. [+9]