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)
JCR Pharmaceuticals Co., Ltd.
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
- Age range
- Up to 5 years
- Sex
- All
- Healthy volunteers
- No
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 \<6 years. 3. Ability to complete and achieve a Standard score ≥55, using either Bailey Scale of Infant Development-4 (BSID-4) Cognitive domain or Kaufman Assessment Battery for Children-II (KABC-II) Non-Verbal Index, whichever tool is most appropriate for the participant's chronological age. Exclusion Criteria: 1. Contraindication for lumbar puncture or MRI. 2. A participant who has a medical condition or extenuatin…