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

August 25, 2025 updated by: JCR Pharmaceuticals Co., Ltd.
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Japan
      • Hiroshima, Japan
        • Recruiting
        • Hiroshima University Hospital
        • Contact:
          • Satoshi Okada
          • Phone Number: +81-82-257-5555
      • Okinawa, Japan
        • Recruiting
        • University of the Ryukyus Hospital
        • Contact:
          • Koichi Nakanishi
          • Phone Number: +81-98-895-3331
      • Tokyo, Japan
        • Recruiting
        • National Center for Child Health and Development
        • Contact:
          • Yuichi Abe
          • Phone Number: +81-3-3416-0181

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: JR-446
Drug: JR-446
IV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To establish the safety and tolerability of JR-446 in MPSIIIB patients
Time Frame: up to 4 years (multiple visits)
up to 4 years (multiple visits)
To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients
Time Frame: up to 4 years (multiple visits)
up to 4 years (multiple visits)

Secondary Outcome Measures

Outcome Measure
Time Frame
To assess PK profile of JR-446 in MPSIIIB patients
Time Frame: up to 4 years (multiple visits)
up to 4 years (multiple visits)
To explore the potential efficacy of JR-446 in the treatment of patients with MPS IIIB
Time Frame: up to 4 years (multiple visits)
up to 4 years (multiple visits)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

JCR Pharmaceuticals Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 5, 2024

Primary Completion (Estimated)

April 30, 2030

Study Completion (Estimated)

April 30, 2030

Study Registration Dates

First Submitted

June 28, 2024

First Submitted That Met QC Criteria

June 28, 2024

First Posted (Actual)

July 5, 2024

Study Record Updates

Last Update Posted (Estimated)

September 2, 2025

Last Update Submitted That Met QC Criteria

August 25, 2025

Last Verified

August 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • JR-446-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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