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
N/A
Total Duration
On this page
AI-Summary
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 I/II 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 drug's impact, including pharmacokinetic profiling. Researchers will also explore the drug’s 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.
CONDITIONS
Brief 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 less than 18 years
- Confirmed diagnosis of Mucopolysaccharidosis IIIB (MPS IIIB)
You will not qualify if you...
- Prior gene therapy or hematopoietic stem cell transplantation with successful engraftment
- Use of other investigational drugs or products within 4 months or 5 half-lives before signing consent
- Participation in another clinical trial involving invasive procedures within 30 days before enrollment
- Use of Genistein or Kineret (anakinra) within 4 months before signing consent
- Serious drug allergy or hypersensitivity
- Contraindications for lumbar puncture or MRI
- History of bleeding disorder or current use of medications increasing bleeding risk after lumbar puncture
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 4 years
Participants receive JR-446 through IV infusion as part of the study treatment.
Multiple visits over the treatment period
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.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
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