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Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

NCT06095388 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-08-26

No results posted yet for this study

Summary

A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.

Conditions

  • Mucopolysaccharidosis III-A

Interventions

DRUG

JR-441

IV infusion

DRUG

JR-441

IV infusion

DRUG

JR-441

IV infusion

Sponsors & Collaborators

  • JCR Pharmaceuticals Co., Ltd.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-10-04
Primary Completion
2029-10-31
Completion
2029-10-31

Countries

  • Germany

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 NCT06095388 on ClinicalTrials.gov