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

• Status: Active, not recruiting
• Trial end date: 2030-03-31
• Target enrollment: 0
• Participant gender: Male

Summary

Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: JCR Pharmaceuticals Co., Ltd.

Criteria

Inclusion Criteria:

1. Patients who will have completed clinical trial JR-141-301 and judged by the investigator or subinvestigator to be able to participate in the study from safety aspects.

2. Capable of providing written consent by himself, unless the patient is under the age of 20 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.

3. In the case of a patient who is under the age of 20 years or from whom it is not possible to obtain consent due to his intellectual disabilities associated with MPS 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:

1. Judged by the investigator or subinvestigator to be ineligible to participate in the study due to receive concomitant therapy which affectseffect the clinical evaluation.

2. 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.

3. Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study.