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

NCT03708965 | Active Not Recruiting Phase 2

• 1 other identifier: JR-141-BR22
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Mucopolysaccharidosis II

Outcome Measures

Primary Outcomes (1)

• Number of participants with Adverse Events

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

Secondary Outcomes (6)

• Urinary heparan sulfate concentrations

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

• Urinary dermatan sulfate concentrations

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

• Serum heparan sulfate concentrations

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

• Serum dermatan sulfate concentrations

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

• Liver and spleen volumes (MRI)

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

Study Arms (1)

JR-141

EXPERIMENTAL

Subjects will be assigned to 1.0, 2.0 or 4.0 mg of JR-141 per kg of body weight once every week (the same dose taken during the previous study) in the beginning of the study. During the study, the dose of all subjects will be switched to the selected one\*. \* The dose was determined to be 2.0 mg/kg/week based on the safety and efficacy data of JR-141-BR21 study.

Drug: JR-141

Interventions

JR-141 DRUG

IV infusion (lyophilized powder)

JR-141

Eligibility Criteria

• Age: 0 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• JCR Pharmaceuticals Co., Ltd.: lead

Study Sites (2)

Grupo de Pesquisa Clínica em Genética Médica - HCPA

Porto Alegre, Brazil

Location

Igeim - Unifesp

São Paulo, Brazil

Location

MeSH Terms

Conditions

Mucopolysaccharidosis II

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2018
• First Posted: October 17, 2018
• Study Start: January 1, 2019
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: July 24, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

BR (2)