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A Study of JR-141 in Patients With Mucopolysaccharidosis II

Primary Purpose

Mucopolysaccharidosis II

Status
Completed
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
JR-141
Sponsored by
JCR Pharmaceuticals Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis II

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Fukui Clinical site
  • Fukuoka Clinical site 2
  • Fukuoka Clinical site
  • Gifu Clinical site
  • Hokkaido Clinical site
  • Kanagawa Clinical site
  • Kumamoto Clinical site
  • Okayama Clinical site
  • Okayama Clinical site 2
  • Okinawa Clinical site
  • Osaka Clinical site 3
  • Osaka Clinical site 2
  • Osaka Clinical site
  • Saitama Clinical site
  • Shizuoka Clinical site
  • Shizuoka Clinical site 2
  • Tochigi Clinical site
  • Tokyo Clinical site
  • Tottori Clinical site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

JR-141 2.0 mg/kg/week

Arm Description

Outcomes

Primary Outcome Measures

Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid

Secondary Outcome Measures

Change From Baseline in Serum Heparan Sulfate Levels.
Change From Baseline in Serum Dermatan Sulfate Levels.
Change From Baseline in Urinary Heparan Sulfate Levels.
Change From Baseline in Urinary Dermatan Sulfate Levels.
Change From Baseline in Liver Volumes.
Change From Baseline in Spleen Volumes.
Change From Baseline in Cardiac Function.
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
Change From Baseline in Joint Range of Motion.
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.
Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)
Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )
Drug concentration in Cerebrospinal Fluid.
*Drug concentration in Cerebrospinal Fluid at 52 Weeks is applicable only for subjects to be enrolled in extension study .

Full Information

First Posted
June 1, 2018
Last Updated
November 10, 2022
Sponsor
JCR Pharmaceuticals Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT03568175
Brief Title
A Study of JR-141 in Patients With Mucopolysaccharidosis II
Official Title
A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
Study Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
August 1, 2018 (Actual)
Primary Completion Date
February 20, 2020 (Actual)
Study Completion Date
February 20, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
JCR Pharmaceuticals Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis II

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
28 (Actual)

8. Arms, Groups, and Interventions

Arm Title
JR-141 2.0 mg/kg/week
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
JR-141
Intervention Description
IV infusion, 2.0 mg/kg/week
Primary Outcome Measure Information:
Title
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
Time Frame
Baseline to 52 weeks
Secondary Outcome Measure Information:
Title
Change From Baseline in Serum Heparan Sulfate Levels.
Time Frame
Baseline, 24-26 weeks, 50-52 weeks
Title
Change From Baseline in Serum Dermatan Sulfate Levels.
Time Frame
Baseline, 24-26 weeks, 50-52 weeks
Title
Change From Baseline in Urinary Heparan Sulfate Levels.
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change From Baseline in Urinary Dermatan Sulfate Levels.
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change From Baseline in Liver Volumes.
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change From Baseline in Spleen Volumes.
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change From Baseline in Cardiac Function.
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change From Baseline in 6-minute Walk Test Distance.
Description
Item 9 will be administrated only in patients judged by the investigator or subinvestigator to be possible to perform the 6-minutes walk test
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change From Baseline in Joint Range of Motion.
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.
Time Frame
Baseline to 25 weeks
Title
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.
Time Frame
Baseline, 25 weeks, 52 weeks
Title
Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)
Time Frame
25, 52 weeks
Title
Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )
Time Frame
25, 52 weeks
Title
Drug concentration in Cerebrospinal Fluid.
Description
*Drug concentration in Cerebrospinal Fluid at 52 Weeks is applicable only for subjects to be enrolled in extension study .
Time Frame
25, 52*weeks

10. Eligibility

Sex
Male
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Fukui Clinical site
City
Fukui
ZIP/Postal Code
910-1193
Country
Japan
Facility Name
Fukuoka Clinical site 2
City
Fukuoka
ZIP/Postal Code
813-0017
Country
Japan
Facility Name
Fukuoka Clinical site
City
Fukuoka
ZIP/Postal Code
830-0011
Country
Japan
Facility Name
Gifu Clinical site
City
Gifu
ZIP/Postal Code
501-1194
Country
Japan
Facility Name
Hokkaido Clinical site
City
Hokkaido
ZIP/Postal Code
063-0005
Country
Japan
Facility Name
Kanagawa Clinical site
City
Kanagawa
ZIP/Postal Code
232-8555
Country
Japan
Facility Name
Kumamoto Clinical site
City
Kumamoto
ZIP/Postal Code
860-8556
Country
Japan
Facility Name
Okayama Clinical site
City
Okayama
ZIP/Postal Code
701-1192
Country
Japan
Facility Name
Okayama Clinical site 2
City
Okayama
ZIP/Postal Code
710-8602
Country
Japan
Facility Name
Okinawa Clinical site
City
Okinawa
ZIP/Postal Code
903-0215
Country
Japan
Facility Name
Osaka Clinical site 3
City
Osaka
ZIP/Postal Code
534-0021
Country
Japan
Facility Name
Osaka Clinical site 2
City
Osaka
ZIP/Postal Code
545-8586
Country
Japan
Facility Name
Osaka Clinical site
City
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Saitama Clinical site
City
Saitama
ZIP/Postal Code
330-8777
Country
Japan
Facility Name
Shizuoka Clinical site
City
Shizuoka
ZIP/Postal Code
420-8660
Country
Japan
Facility Name
Shizuoka Clinical site 2
City
Shizuoka
ZIP/Postal Code
426-8677
Country
Japan
Facility Name
Tochigi Clinical site
City
Tochigi
ZIP/Postal Code
329-0498
Country
Japan
Facility Name
Tokyo Clinical site
City
Tokyo
ZIP/Postal Code
157-8535
Country
Japan
Facility Name
Tottori Clinical site
City
Tottori
ZIP/Postal Code
683-8504
Country
Japan

12. IPD Sharing Statement

Links:
URL
https://pubmed.ncbi.nlm.nih.gov/33038326/
Description
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II

Learn more about this trial

A Study of JR-141 in Patients With Mucopolysaccharidosis II

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