Clinical Trial

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Active, Not Recruiting Phase 1/2
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Summary
A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2023-10-18; most recent amendment 2025-08-19.
Status change: Recruiting → Active, Not Recruiting 2025-08-19
Trial Details
NCT Number NCT06095388
Lead Sponsor JCR Pharmaceuticals Co., Ltd.
Conditions Mucopolysaccharidosis III-A
Enrollment 15 participants
Start Date 2023-10-04
Primary Completion 2029-10-31 (estimated)
Study Completion 2029-10-31 (estimated)
Updated on ClinicalTrials.gov 2025-08-26