An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

Recruiting now · Phase 1/Phase 2

Conditions studied: Mucopolysaccharidosis III-B

In brief

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.

Key facts

Study ID

NCT06488924

Run by

JCR Pharmaceuticals Co., Ltd.

People needed

10

Starts

2024-11-05

Expected to finish

2030-04-30

Last updated by the study team

2025-09-02

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Chronological age of <18 years
• Confirmed diagnosis of MPS IIIB

You may not qualify if…

• 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.

Where it is running

• Hiroshima University Hospital — Hiroshima, Japan (enrolling)
• University of the Ryukyus Hospital — Okinawa, Japan (enrolling)
• National Center for Child Health and Development — Tokyo, Japan (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.