We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study of JR-141 in Patients With Mucopolysaccharidosis II

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03568175
Recruitment Status : Completed
First Posted : June 26, 2018
Last Update Posted : November 14, 2022
Sponsor:
Information provided by (Responsible Party):
JCR Pharmaceuticals Co., Ltd.

Tracking Information
First Submitted Date  ICMJE June 1, 2018
First Posted Date  ICMJE June 26, 2018
Last Update Posted Date November 14, 2022
Actual Study Start Date  ICMJE August 1, 2018
Actual Primary Completion Date February 20, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 13, 2018)
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid [ Time Frame: Baseline to 52 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 28, 2018)
  • Change From Baseline in Serum Heparan Sulfate Levels. [ Time Frame: Baseline, 24-26 weeks, 50-52 weeks ]
  • Change From Baseline in Serum Dermatan Sulfate Levels. [ Time Frame: Baseline, 24-26 weeks, 50-52 weeks ]
  • Change From Baseline in Urinary Heparan Sulfate Levels. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Urinary Dermatan Sulfate Levels. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Liver Volumes. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Spleen Volumes. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Cardiac Function. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in 6-minute Walk Test Distance. [ Time Frame: 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. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: Baseline to 25 weeks ]
  • Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001) [ Time Frame: 25, 52 weeks ]
  • Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. ) [ Time Frame: 25, 52 weeks ]
  • Drug concentration in Cerebrospinal Fluid. [ Time Frame: 25, 52*weeks ]
    *Drug concentration in Cerebrospinal Fluid at 52 Weeks is applicable only for subjects to be enrolled in extension study .
Original Secondary Outcome Measures  ICMJE
 (submitted: June 13, 2018)
  • Change From Baseline in Serum Heparan Sulfate Levels. [ Time Frame: Baseline, 24-26 weeks, 50-52 weeks ]
  • Change From Baseline in Serum Dermatan Sulfate Levels. [ Time Frame: Baseline, 24-26 weeks, 50-52 weeks ]
  • Change From Baseline in Urinary Heparan Sulfate Levels. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Urinary Dermatan Sulfate Levels. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Liver Volumes. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Spleen Volumes. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Cardiac Function. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in 6-minute Walk Test Distance. [ Time Frame: 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. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: Baseline to 25 weeks ]
  • Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  • Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001) [ Time Frame: 25, 52 weeks ]
  • Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. ) [ Time Frame: 25, 52 weeks ]
    *Drug concentration in CSF at 52 Weeks is applicable only for subjects who enroll in extension study.
  • Drug concentration in CSF. [ Time Frame: 25, 52*weeks ]
    *Drug concentration in CSF at 52 Weeks is applicable only for subjects who enroll in extension study.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of JR-141 in Patients With Mucopolysaccharidosis II
Official Title  ICMJE A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Mucopolysaccharidosis II
Intervention  ICMJE Drug: JR-141
IV infusion, 2.0 mg/kg/week
Study Arms  ICMJE Experimental: JR-141 2.0 mg/kg/week
Intervention: Drug: JR-141
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 31, 2021)
28
Original Estimated Enrollment  ICMJE
 (submitted: June 13, 2018)
20
Actual Study Completion Date  ICMJE February 20, 2020
Actual Primary Completion Date February 20, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03568175
Other Study ID Numbers  ICMJE JR-141-301
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party JCR Pharmaceuticals Co., Ltd.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE JCR Pharmaceuticals Co., Ltd.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account JCR Pharmaceuticals Co., Ltd.
Verification Date November 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP