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

Phase 2

Active, not recruiting

• Conditions: Mucopolysaccharidosis II
• Interventions: Drug: JR-141

• Registration Number: NCT03708965
• Lead Sponsor: JCR Pharmaceuticals Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-09-27
• Study First Submitted QC: 2018-10-16
• Study First Posted: 2018-10-17
• Study Start: 2019-01-01
• Status Verified: 2023-06
• Last Update Submitted: 2023-06-22
• Last Update Posted: 2023-06-23
• Primary Completion: 2024-03-31
• Study Completion: 2024-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 19

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
JR-141	JR-141	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.

Primary Outcome Measures

Name	Time	Method
Number of participants with Adverse Events	From screening up to the end of study, up to approximately 5 years

Secondary Outcome Measures

Name	Time	Method
Urinary 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
Echocardiography	through study completion, an average of 52 weeks, up to approximately 5 years
Urinary heparan 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

Trial Locations

Locations (2)

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

Igeim - Unifesp; 🇧🇷 São Paulo, Brazil