Phase III Open-label Study of JR-446 Infusion for Children With Mucopolysaccharidosis Type IIIB

What this Trial Is About

Researchers are evaluating JR-446, a study drug, in patients with Mucopolysaccharidosis type IIIB MPS IIIB, a rare genetic condition. This open-label Phase III trial aims to assess the safety and tolerability of JR-446 while exploring its potential effects in treating MPS IIIB. The study is sponsored by JCR Pharmaceuticals Co., Ltd. and includes children up to 17 years old. Participants will receive JR-446 through intravenous infusion. The study will last up to 4 years with multiple visits during this period to closely monitor patients. The trial does not include a placebo group and does not involve masking or blinding. This long-term study will observe how the drug behaves in the body and evaluate its pharmacodynamic effects. During participation, patients will undergo safety assessments and tests to track the drugs impact, including pharmacokinetic profiling. Researchers will also explore the drugs potential to benefit MPS IIIB symptoms. Visits will include various evaluations and monitoring to ensure participant safety throughout the trial period, which concludes by April 2030.

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB