Actively Recruiting
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Led by JCR Pharmaceuticals Co., Ltd. · Updated on 2025-09-02
10
Participants Needed
3
Research Sites
286 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Chronological age of <18 years
- Confirmed diagnosis of MPS IIIB
You will not qualify if you...
- Prior experience to gene therapy or HSCT with successful engraftment
- Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF
- Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures
- Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF
- Serious drug allergy or hypersensitivity
- Contraindication for lumbar puncture or MRI
- History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 3 locations
1
Hiroshima University Hospital
Hiroshima, Japan
Actively Recruiting
2
University of the Ryukyus Hospital
Okinawa, Japan
Actively Recruiting
3
National Center for Child Health and Development
Tokyo, Japan
Actively Recruiting
Research Team
J
JCR Pharmaceuticals Co., Ltd.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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