A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II Clinical Trial

Official title:

A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03568175
• Other study ID #: JR-141-301
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: August 1, 2018
• Est. completion date: February 20, 2020

Study information

• Verified date: November 2022
• Source: JCR Pharmaceuticals Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 information / eligibility

• Status: Completed
• Enrollment: 28
• Est. completion date: February 20, 2020
• Est. primary completion date: February 20, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. 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.)

2. 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)

3. 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.

4. 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:

1. Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.

2. 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.

3. Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity.

4. Patients who have received other investigational product within 4 months before enrollment in the study.

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

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis II

Intervention

Drug:
• JR-141
IV infusion, 2.0 mg/kg/week

Locations

Country	Name	City	State
Japan	Fukui Clinical site	Fukui
Japan	Fukuoka Clinical site	Fukuoka
Japan	Fukuoka Clinical site 2	Fukuoka
Japan	Gifu Clinical site	Gifu
Japan	Hokkaido Clinical site	Hokkaido
Japan	Kanagawa Clinical site	Kanagawa
Japan	Kumamoto Clinical site	Kumamoto
Japan	Okayama Clinical site	Okayama
Japan	Okayama Clinical site 2	Okayama
Japan	Okinawa Clinical site	Okinawa
Japan	Osaka Clinical site	Osaka
Japan	Osaka Clinical site 2	Osaka
Japan	Osaka Clinical site 3	Osaka
Japan	Saitama Clinical site	Saitama
Japan	Shizuoka Clinical site	Shizuoka
Japan	Shizuoka Clinical site 2	Shizuoka
Japan	Tochigi Clinical site	Tochigi
Japan	Tokyo Clinical site	Tokyo
Japan	Tottori Clinical site	Tottori

Sponsors (1)

Lead Sponsor	Collaborator
JCR Pharmaceuticals Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

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

External Links

•   A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II