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

NCT06488924 | Recruiting Phase 1

• 1 other identifier: JR-446-101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 3

Brief 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

Outcome Measures

Primary Outcomes (2)

• To establish the safety and tolerability of JR-446 in MPSIIIB patients

  up to 4 years (multiple visits)

• To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients

  up to 4 years (multiple visits)

Secondary Outcomes (2)

• To assess PK profile of JR-446 in MPSIIIB patients

  up to 4 years (multiple visits)

• To explore the potential efficacy of JR-446 in the treatment of patients with MPS IIIB

  up to 4 years (multiple visits)

Study Arms (1)

JR-446

EXPERIMENTAL

Drug: JR-446

Interventions

JR-446 DRUG

IV infusion

JR-446

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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.

Sponsors & Collaborators

• JCR Pharmaceuticals Co., Ltd.: lead

Study Sites (3)

Hiroshima University Hospital

Hiroshima, Japan

RECRUITING

University of the Ryukyus Hospital

Okinawa, Japan

RECRUITING

National Center for Child Health and Development

Tokyo, Japan

RECRUITING

MeSH Terms

Conditions

Mucopolysaccharidosis III

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

JCR Pharmaceuticals Co., Ltd.

CONTACT

+81-797-32-8582; clinical_development@jp.jcrpharm.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2024
• First Posted: July 5, 2024
• Study Start: November 5, 2024
• Primary Completion (Estimated): April 30, 2030
• Study Completion (Estimated): April 30, 2030
• Last Updated: September 2, 2025

Record last verified: 2025-08

Locations

JP (3)