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NCT07640984
A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
Conditions: Mucopolysaccharidosis IIIB
Sex: All
Ages: N/A – 5 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 12
Sponsor: JCR Pharmaceuticals Co., Ltd.
Location: Children's Hospital Oakland Research Institution, Attn: 2900 Wing Oakland California
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.
Eligibility Criteria
Inclusion Criteria:
1. Patients with confirmed MPS IIIB with a phenotype of MPS IIIB that is not classified as slowly progressive (i.e., not attenuated).
2. A chronological age \
Source: ClinicalTrials.gov (NCT07640984). StuddyBuddy aggregates publicly available trial information.
