clinical trial · NCT07640984
A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
JCR Pharmaceuticals Co., Ltd.·phase2·not yet recruiting·n = 12
Mucopolysaccharidosis IIIBJR-446
brief 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.
started
Jun 18, 2026
primary completion
Jun 30, 2028
completion
Sep 30, 2028
last updated
Jun 11, 2026
official title
A Global, Open-Label, Phase I/II Trial, to Evaluate Safety, Tolerability, Pharmacodynamic and Preliminary Efficacy of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol