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NCT Number: NCT06488924

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

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.

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Key information

Conditions

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

Age range

Up to 17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Hiroshima University Hospital, Hiroshima, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

JR-446

Drug

IV infusion

Primary outcomes

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

    Time frame: up to 4 years (multiple visits)

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

    Time frame: up to 4 years (multiple visits)

Secondary outcomes

  1. To assess PK profile of JR-446 in MPSIIIB patients

    Time frame: up to 4 years (multiple visits)

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

    Time frame: up to 4 years (multiple visits)

Study contacts

Contact information is provided by the study sponsor or research team.

JCR Pharmaceuticals Co., Ltd.

CONTACT

[email protected]

+81-797-32-8582

Sponsors and collaborators

Lead sponsor

JCR Pharmaceuticals Co., Ltd.

Industry

Registry information

Important dates

Study start
2024
Primary completion
2030
Study completion
2030
First posted
Jul 5, 2024
Registry last updated
Sep 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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