Status:
RECRUITING
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Lead Sponsor:
JCR Pharmaceuticals Co., Ltd.
Conditions:
Mucopolysaccharidosis III-B
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE1
PHASE2
Brief 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 Criteria
Inclusion
- Chronological age of \<18 years
- Confirmed diagnosis of MPS IIIB
Exclusion
- 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
- The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
Key Trial Info
Start Date :
November 5 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 30 2030
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT06488924
Start Date
November 5 2024
End Date
April 30 2030
Last Update
September 2 2025
Active Locations (3)
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1
Hiroshima University Hospital
Hiroshima, Japan
2
University of the Ryukyus Hospital
Okinawa, Japan
3
National Center for Child Health and Development
Tokyo, Japan