Clinical Trial

A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

Not Yet Recruiting Phase 1/2
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Summary
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.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2026-06-08; most recent amendment 2026-07-02.
Trial Details
NCT Number NCT07640984
Lead Sponsor JCR Pharmaceuticals Co., Ltd.
Collaborators: MEDIPAL HOLDINGS CORPORATION
Conditions Mucopolysaccharidosis IIIB
Enrollment 12 participants
Start Date 2026-06-18
Primary Completion 2028-06-30 (estimated)
Study Completion 2028-09-30 (estimated)
Updated on ClinicalTrials.gov 2026-07-07