An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: f
View:
• Chronological age of \<18 years
• Confirmed diagnosis of MPS IIIB
Locations
Other Locations
Japan
Hiroshima University Hospital
RECRUITING
Hiroshima
University of the Ryukyus Hospital
RECRUITING
Okinawa
National Center for Child Health and Development
RECRUITING
Tokyo
Contact Information
Primary
JCR Pharmaceuticals Co., Ltd.
clinical_development@jp.jcrpharm.com
+81-797-32-8582
Time Frame
Start Date: 2024-11-05
Estimated Completion Date: 2030-04-30
Participants
Target number of participants: 10
Treatments
Experimental: JR-446
Related Therapeutic Areas
Sponsors
Leads: JCR Pharmaceuticals Co., Ltd.