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

U.S., June 12 -- ClinicalTrials.gov registry received information related to the study (NCT07640984) titled 'A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)' on May 14.

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.

Study Start Date: June 18

Study Type: INTERVENTIONAL

Condition: Mucopolysaccharidosis IIIB

Intervention: DRUG: JR-446

IV infusion

Recruitment Status: NOT_YET_RECRUITING

Sponsor: JCR ...