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A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT07640984 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-09-03

No results posted yet for this study

Summary

This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.

Conditions

  • Mucopolysaccharidosis IIIB

Interventions

DRUG

JR-446

IV infusion

Sponsors & Collaborators

  • MEDIPAL HOLDINGS CORPORATION

    collaborator UNKNOWN

  • JCR Pharmaceuticals Co., Ltd.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-04
Primary Completion
2028-06-30
Completion
2028-09-30
FDA Drug
Yes

Countries

  • United States
  • Germany
  • United Kingdom

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 NCT07640984 on ClinicalTrials.gov