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

Phase 2

Active, not recruiting

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

• Registration Number: NCT04348136
• Lead Sponsor: JCR Pharmaceuticals Co., Ltd.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-09-01
• Study First Submitted: 2019-12-18
• Study First Submitted QC: 2020-04-13
• Study First Posted: 2020-04-16
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-13
• Last Update Posted: 2024-03-15
• Primary Completion: 2030-03-31
• Study Completion: 2030-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 27

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
JR-141	JR-141	-

Primary Outcome Measures

Name	Time	Method
Time course of serum HS and DS concentrations from initial dosing in the preceding study	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of uronic acid concentration from initial dosing in the preceding study	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (blood pressure)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
12-lead electrocardiogram	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years	The presence or absence of abnormal findings (if present, specific findings and whether or not they are reported as adverse events)
Time course of developmental assessment data (Vineland-II) from initial dosing in the preceding study	Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of CSF substrate (HS and DS) concentrations from initial dosing in the preceding study	Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of urinary DS concentration from initial dosing in the preceding study	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of spleen volume (assessed by CT or MRI) from initial dosing in the preceding study	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Occurrence of adverse events	From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Occurrence of adverse reactions	From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Incidence of abnormal vital signs	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years	Laboratory tests (iron-related tests)
Laboratory tests (urinalysis)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (pulse rate)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (body temperature)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Antibody tests (anti-JR-141 antibodies)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
IAR	From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Time course of developmental assessment data (Kyoto Scale of Psychological Development 2001) from initial dosing in the preceding study	Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of urinary HS concentration from initial dosing in the preceding study	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of 6-minute walk test distance from initial dosing in the preceding study	Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of developmental assessment data (Bayley-III or KABC-II) from initial dosing in the preceding study	Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of liver volume (assessed by CT or MRI) from initial dosing in the preceding study	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of cardiac function (assessed by echocardiography) from initial dosing in the preceding study	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of joint range of motion from initial dosing in the preceding study	Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Trial Locations

Locations (20)

Fukui Clinical site; 🇯🇵 Fukui, Japan

Fukuoka Clinical site 2; 🇯🇵 Fukuoka, Japan

Fukuoka Clinical site; 🇯🇵 Fukuoka, Japan

Gifu Clinical site; 🇯🇵 Gifu, Japan

Shizuoka Clinical site; 🇯🇵 Shizuoka, Japan

Kananagawa Ckinical site; 🇯🇵 Kanagawa, Japan

Kumamoto Clinical site; 🇯🇵 Kumamoto, Japan

Hiroshima Prefectural Hospital; 🇯🇵 Hiroshima, Japan

Hokkaido Clinical site; 🇯🇵 Hokkaido, Japan

Okayama Clinical site 2; 🇯🇵 Okayama, Japan

Okayama Clinical site; 🇯🇵 Okayama, Japan

Okinawa Clinical site; 🇯🇵 Okinawa, Japan

Osaka Clinical site 3; 🇯🇵 Osaka, Japan

Osaka Clinical site 2; 🇯🇵 Osaka, Japan

Saitama Clinical site; 🇯🇵 Saitama, Japan

Osaka Clinical site; 🇯🇵 Osaka, Japan

Shizuoka Clinical site 2; 🇯🇵 Shizuoka, Japan

Tottori Clinical site; 🇯🇵 Tottori, Japan

Tochigi Clinical site; 🇯🇵 Tochigi, Japan

Tokyo Clinical site; 🇯🇵 Tokyo, Japan