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NCT Number: NCT03128593

A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,

* to collect the safety information of JR-141 * to evaluate the plasma pharmacokinetics of JR-141 * to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms

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Key information

Conditions

Mucopolysaccharidosis II Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases X-Linked Intellectual Disability

Age range

6 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Gifu Clinical site, Gifu, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Patients aged 6 years or older at the time of informed consent.
  • Patients diagnosed with MPS II.
  • Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.

Exclusion criteria

  • Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation.
  • Patients in whom lumbar puncture cannot be performed.
  • Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study.
  • Patients who have received other investigational products within 4 months before enrollment in the study.

Treatment and study plan

JR-141

Drug

IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week

Primary outcomes

  1. Number of participants with Adverse Events

    Time frame: 4 weeks

    • Adverse events
    • Laboratory tests
    • Vital signs
    • 12-lead electrocardiogram
    • Antibody
    • Infusion associated reaction

Secondary outcomes

  1. Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]

    Time frame: 4 weeks

    Plasma concentration of JR-141

  2. Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]

    Time frame: 4 weeks

    Plasma concentration of JR-141

  3. Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)

    Time frame: 4 weeks

  4. HS/DS in CSF

    Time frame: 4 weeks

    JR-141 concentration in CSF

  5. Urinary total GAG

    Time frame: 4 weeks

  6. Liver and spleen volumes

    Time frame: 4 weeks

  7. Cardiac function

    Time frame: 4 weeks

Sponsors and collaborators

Lead sponsor

JCR Pharmaceuticals Co., Ltd.

Industry

Registry information

Official study title

A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Important dates

Study start
2017
Primary completion
2017
Study completion
2017
First posted
Apr 25, 2017
Registry last updated
Nov 14, 2022

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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