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

NCT06488924 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-09-02

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis III-B

Interventions

DRUG

JR-446

IV infusion

Sponsors & Collaborators

  • JCR Pharmaceuticals Co., Ltd.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-05
Primary Completion
2030-04-30
Completion
2030-04-30

Countries

  • Japan

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06488924 on ClinicalTrials.gov