A Study of JR-141 in Patients With Mucopolysaccharidosis II

November 10, 2022 updated by: JCR Pharmaceuticals Co., Ltd.

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
28

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Fukui, Japan, 910-1193
        • Fukui Clinical site
      • Fukuoka, Japan, 813-0017
        • Fukuoka Clinical Site 2
      • Fukuoka, Japan, 830-0011
        • Fukuoka Clinical site
      • Gifu, Japan, 501-1194
        • Gifu Clinical site
      • Hokkaido, Japan, 063-0005
        • Hokkaido Clinical site
      • Kanagawa, Japan, 232-8555
        • Kanagawa Clinical Site
      • Kumamoto, Japan, 860-8556
        • Kumamoto Clinical site
      • Okayama, Japan, 701-1192
        • Okayama Clinical site
      • Okayama, Japan, 710-8602
        • Okayama Clinical site 2
      • Okinawa, Japan, 903-0215
        • Okinawa Clinical site
      • Osaka, Japan, 534-0021
        • Osaka Clinical Site 3
      • Osaka, Japan, 545-8586
        • Osaka Clinical Site 2
      • Osaka, Japan, 565-0871
        • Osaka Clinical site
      • Saitama, Japan, 330-8777
        • Saitama Clinical site
      • Shizuoka, Japan, 420-8660
        • Shizuoka Clinical site
      • Shizuoka, Japan, 426-8677
        • Shizuoka Clinical site 2
      • Tochigi, Japan, 329-0498
        • Tochigi Clinical site
      • Tokyo, Japan, 157-8535
        • Tokyo Clinical site
      • Tottori, Japan, 683-8504
        • Tottori Clinical site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: JR-141 2.0 mg/kg/week
Drug: JR-141
IV infusion, 2.0 mg/kg/week

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
Time Frame: Baseline to 52 weeks
Baseline to 52 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Serum Heparan Sulfate Levels.
Time Frame: Baseline, 24-26 weeks, 50-52 weeks
Baseline, 24-26 weeks, 50-52 weeks
Change From Baseline in Serum Dermatan Sulfate Levels.
Time Frame: Baseline, 24-26 weeks, 50-52 weeks
Baseline, 24-26 weeks, 50-52 weeks
Change From Baseline in Urinary Heparan Sulfate Levels.
Time Frame: Baseline, 25 weeks, 52 weeks
Baseline, 25 weeks, 52 weeks
Change From Baseline in Urinary Dermatan Sulfate Levels.
Time Frame: Baseline, 25 weeks, 52 weeks
Baseline, 25 weeks, 52 weeks
Change From Baseline in Liver Volumes.
Time Frame: Baseline, 25 weeks, 52 weeks
Baseline, 25 weeks, 52 weeks
Change From Baseline in Spleen Volumes.
Time Frame: Baseline, 25 weeks, 52 weeks
Baseline, 25 weeks, 52 weeks
Change From Baseline in Cardiac Function.
Time Frame: Baseline, 25 weeks, 52 weeks
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
Baseline, 25 weeks, 52 weeks
Change From Baseline in Joint Range of Motion.
Time Frame: Baseline, 25 weeks, 52 weeks
Baseline, 25 weeks, 52 weeks
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.
Time Frame: Baseline to 25 weeks
Baseline to 25 weeks
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.
Time Frame: Baseline, 25 weeks, 52 weeks
Baseline, 25 weeks, 52 weeks
Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)
Time Frame: 25, 52 weeks
25, 52 weeks
Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )
Time Frame: 25, 52 weeks
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 .
25, 52*weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
JCR Pharmaceuticals Co., Ltd.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 1, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 20, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 20, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 1, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 13, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 26, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 14, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 10, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • JR-141-301

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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