A Study of JR-141 in Patients With Mucopolysaccharidosis II

Phase 2

Completed

Brief Summary: A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.

Recruitment & Eligibility

Inclusion Criteria

Capable of providing written consent by himself (not mandatory for those aged under 20 years at the time of informed consent process, or those who is impossible to obtain consent from the patient himself due to intellectual disabilities associated with MPS II.)
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 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, whenever possible)
Males with confirmed diagnosis of MPS II, based on deficient activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic mutations identified in the IDS gene, etc.
Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.

Exclusion Criteria

Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely 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 serious drug allergy or sensitivity.
Patients who have received other investigational product within 4 months before enrollment in the study.
Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study.

Arm && Interventions

Group	Intervention	Description
JR-141 2.0 mg/kg/week	JR-141	-

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid	Baseline to 52 weeks

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Urinary Heparan Sulfate Levels.	Baseline, 25 weeks, 52 weeks
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.	Baseline to 25 weeks
Change From Baseline in Serum Dermatan Sulfate Levels.	Baseline, 24-26 weeks, 50-52 weeks
Change From Baseline in Liver Volumes.	Baseline, 25 weeks, 52 weeks
Change From Baseline in 6-minute Walk Test Distance.	Baseline, 25 weeks, 52 weeks	Item 9 will be administrated only in patients judged by the investigator or subinvestigator to be possible to perform the 6-minutes walk test
Change From Baseline in Joint Range of Motion.	Baseline, 25 weeks, 52 weeks
Change From Baseline in Urinary Dermatan Sulfate Levels.	Baseline, 25 weeks, 52 weeks
Change From Baseline in Spleen Volumes.	Baseline, 25 weeks, 52 weeks
Drug concentration in Cerebrospinal Fluid.	25, 52*weeks	\*Drug concentration in Cerebrospinal Fluid at 52 Weeks is applicable only for subjects to be enrolled in extension study .
Change From Baseline in Cardiac Function.	Baseline, 25 weeks, 52 weeks
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.	Baseline, 25 weeks, 52 weeks
Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)	25, 52 weeks
Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )	25, 52 weeks
Change From Baseline in Serum Heparan Sulfate Levels.	Baseline, 24-26 weeks, 50-52 weeks

Locations (19): Osaka Clinical site
🇯🇵
Osaka, Japan
Saitama Clinical site
🇯🇵
Saitama, Japan
Fukui Clinical site
🇯🇵
Fukui, Japan
Fukuoka Clinical site 2
🇯🇵
Fukuoka, Japan
Fukuoka Clinical site
🇯🇵
Fukuoka, Japan
Gifu Clinical site
🇯🇵
Gifu, Japan
Kumamoto Clinical site
🇯🇵
Kumamoto, Japan
Kanagawa Clinical site
🇯🇵
Kanagawa, Japan
Okinawa Clinical site
🇯🇵
Okinawa, Japan
Okayama Clinical site
🇯🇵
Okayama, Japan
Okayama Clinical site 2
🇯🇵
Okayama, Japan
Osaka Clinical site 3
🇯🇵
Osaka, Japan
Shizuoka Clinical site
🇯🇵
Shizuoka, Japan
Tochigi Clinical site
🇯🇵
Tochigi, Japan
Hokkaido Clinical site
🇯🇵
Hokkaido, Japan
Osaka Clinical site 2
🇯🇵
Osaka, Japan
Shizuoka Clinical site 2
🇯🇵
Shizuoka, Japan
Tokyo Clinical site
🇯🇵
Tokyo, Japan
Tottori Clinical site
🇯🇵
Tottori, Japan