RecruitingPhase 1Phase 2NCT06488924
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Sponsor
JCR Pharmaceuticals Co., Ltd.
Enrollment
10 participants
Start Date
Nov 5, 2024
Study Type
INTERVENTIONAL
Conditions
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
Max Age: 17 Years
Inclusion Criteria2
- Chronological age of \<18 years
- Confirmed diagnosis of MPS IIIB
Exclusion Criteria8
- 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.
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Interventions
DRUGJR-446
IV infusion
Locations(3)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06488924