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

JCR Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3; Phase 2

Conditions: Mucopolysaccharidosis II

Treatments: Drug: JR-141

Study type: Interventional

Funder types: Industry

Identifiers

NCT04348136

JR-141-302

Details and patient eligibility

About

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

Enrollment: 27 patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion criteria

1. A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator.
2. A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.)
3. For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.)
4. Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception.

Exclusion criteria

1. A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator.
2. A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator.
3. A patient judged to be ineligible by the investigator or subinvestigator for other reasons.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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27 participants in 1 patient group

JR-141

Experimental group

Treatment:

Drug: JR-141