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NCT Number: NCT03708965

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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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

  • Patients who will have completed clinical trial JR-141-BR21.
  • 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.

Exclusion criteria

  • Refusal to sign the informed consent form.
  • Unable to perform the study procedures, except for neurocognitive testing.
  • Previous engrafted BMT/HSCT.
  • 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)

Primary outcomes

  1. Number of participants with Adverse Events

    Time frame: From screening up to the end of study, up to approximately 5 years

Secondary outcomes

  1. Urinary heparan sulfate concentrations

    Time frame: through study completion, an average of 52 weeks, up to approximately 5 years

  2. Urinary dermatan sulfate concentrations

    Time frame: through study completion, an average of 52 weeks, up to approximately 5 years

  3. Serum heparan sulfate concentrations

    Time frame: through study completion, an average of 52 weeks, up to approximately 5 years

  4. Serum dermatan sulfate concentrations

    Time frame: through study completion, an average of 52 weeks, up to approximately 5 years

  5. Liver and spleen volumes (MRI)

    Time frame: through study completion, an average of 52 weeks, up to approximately 5 years

  6. Echocardiography

    Time frame: through study completion, an average of 52 weeks, up to approximately 5 years

Sponsors and collaborators

Lead sponsor

JCR Pharmaceuticals Co., Ltd.

Industry

Registry information

Important dates

Study start
2019
Primary completion
2026
Study completion
2027
First posted
Oct 17, 2018
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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