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

JCR Pharmaceuticals

Status and phase: Active, not recruiting; Phase 2

Conditions: Mucopolysaccharidosis II

Treatments: Drug: JR-141

Study type: Interventional

Funder types: Industry

Identifiers

NCT03708965

JR-141-BR22

Details and patient eligibility

About

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.

Enrollment: 19 patients

Sex: Male

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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19 participants in 1 patient group

JR-141

Experimental group

Description:

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.

Treatment:

Drug: JR-141