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

NCT ID: NCT04348136

Last Updated: 2025-07-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-09-01

Study Completion Date

2030-03-31

Brief Summary

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

Detailed Description

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Conditions

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Mucopolysaccharidosis II

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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JR-141

Group Type EXPERIMENTAL

JR-141

Intervention Type DRUG

IV infusion, 2.0 mg/kg/week

Interventions

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JR-141

IV infusion, 2.0 mg/kg/week

Intervention Type DRUG

Eligibility Criteria

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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.
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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JCR Pharmaceuticals Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Fukui Clinical site

Fukui, , Japan

Site Status

Fukuoka Clinical site 2

Fukuoka, , Japan

Site Status

Fukuoka Clinical site

Fukuoka, , Japan

Site Status

Gifu Clinical site

Gifu, , Japan

Site Status

Hiroshima Prefectural Hospital

Hiroshima, , Japan

Site Status

Hokkaido Clinical site

Hokkaido, , Japan

Site Status

Kananagawa Ckinical site

Kanagawa, , Japan

Site Status

Kumamoto Clinical site

Kumamoto, , Japan

Site Status

Okayama Clinical site

Okayama, , Japan

Site Status

Okayama Clinical site 2

Okayama, , Japan

Site Status

Okinawa Clinical site

Okinawa, , Japan

Site Status

Osaka Clinical site 3

Osaka, , Japan

Site Status

Osaka Clinical site 2

Osaka, , Japan

Site Status

Osaka Clinical site

Osaka, , Japan

Site Status

Saitama Clinical site

Saitama, , Japan

Site Status

Shizuoka Clinical site

Shizuoka, , Japan

Site Status

Shizuoka Clinical site 2

Shizuoka, , Japan

Site Status

Tochigi Clinical site

Tochigi, , Japan

Site Status

Tokyo Clinical site

Tokyo, , Japan

Site Status

Tottori Clinical site

Tottori, , Japan

Site Status

Countries

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Japan

Other Identifiers

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JR-141-302

Identifier Type: -

Identifier Source: org_study_id

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