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Completed

NCT Number: NCT03359213

A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)

A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.

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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

0 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Grupo de Pesquisa Clínica em Genética Médica - HCPA, Porto Alegre, Brazil

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Males with confirmed diagnosis of MPS II, based on deficient activity of IDS in leucocytes or fibroblasts and/or pathogenic mutations identified in the IDS gene (if enzyme diagnosis was in dried blood spots or plasma, molecular genetics confirmation is mandatory).
  • One of the following age groups:
  • 0 to 3 years and 11 months old (6 patients, 2 in each dose)
  • 4 years to 7 years and 11 months old (6 patients, 2 in each dose)
  • 8 years or older (6 patients, 2 in each dose)
  • Capable of providing written consent by himself, unless the patient is under the age of 18 years at the time of informed consent process, or it is not possible to obtain consent from the patient himself due to his intellectual disabilities associated with MPS II.
  • In the case of a patient who is under the age of 18 years or from whom it is not possible to obtain consent due to his intellectual disabilities associated with MSP II, he may be included if written consent can be provided by legal representative; however written consent should be obtained from the patient himself too, wherever possible.
  • Naïve patients or patients who are receiving enzyme replacement therapy with idursulfase could be included if provided treatment has been stable in the last 6 months and agree to interrupt the treatment at least one week before the first study drug infusion, and agree in suspending this treatment for the duration of the trial.

Exclusion criteria

  • Refusal to sign the informed consent form.
  • Unable to perform the study procedures, except for neurocognitive testing.
  • Previous engrafted BMT/HSCT.
  • Surgical or other major medical intervention planned to occur before week 26.
  • Participation in a clinical trial with an investigational drug in the last 12 months.
  • Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumber puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process.
  • Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or sensitivity.
  • Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study out of consideration for the subject safety.

Treatment and study plan

JR-141

Drug

IV infusion (lyophilized powder), 1.0 mg/kg/week

Primary outcomes

  1. Number of participants with Adverse Events

    Time frame: 26 weeks

    • Adverse events
    • Antidrug antibodies
    • Blood pressures in mmHg
    • Heart rate in beats/minute
    • Respiratory rate in breaths/minute
    • Temperature in °C
    • Presence or absence of abnormalities for physical examination
    • Presence or absence of abnormalities for 12-lead electrocardiogram
    • Routine laboratory tests in blood (hematology, liver function, renal function, iron-related levels) and urine (urinalysis)

Secondary outcomes

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

    Time frame: 21 hours after dosing at the first and last infusions

    Plasma concentration of JR-141

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

    Time frame: 21 hours after dosing at the first and last infusions

    Plasma concentration of JR-141

  3. Liver and spleen volumes (MRI)

    Time frame: 26 weeks

  4. Left ventricular mass by a standard 2-dimensional Doppler echocardiogram

    Time frame: 26 weeks

  5. Urinary heparan sulfate concentrations

    Time frame: 26 weeks

  6. Urinary dermatan sulfate concentrations

    Time frame: 26 weeks

  7. Serum heparan sulfate concentrations

    Time frame: 26 weeks

  8. Serum dermatan sulfate concentrations

    Time frame: 26 weeks

Sponsors and collaborators

Lead sponsor

JCR Pharmaceuticals Co., Ltd.

Industry

Registry information

Official study title

Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II

Important dates

Study start
2018
Primary completion
2019
Study completion
2019
First posted
Dec 2, 2017
Registry last updated
Jul 24, 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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