A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

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

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

The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,

  • to collect the safety information of JR-141
  • to evaluate the plasma pharmacokinetics of JR-141
  • to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms

Study Overview

Status

Completed

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

14

Phase

  • Phase 2
  • Phase 1

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

      • Gifu, Japan, 501-1194
        • Gifu Clinical site
      • Kurume, Japan, 830-0011
        • Fukuoka Clinical site
      • Minato, Japan, 105-8471
        • Tokyo Clinical Site1
      • Osaka, Japan, 545-8586
        • Osaka Clinical Site1
      • Saitama, Japan, 330-8777
        • Saitama Clinical site
      • Setagaya, Japan, 157-8535
        • Tokyo Clinical Site2
      • Suita, Japan, 157-8535
        • Osaka Clinical Site2
      • Yonago, 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

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Patients aged 6 years or older at the time of informed consent.
  • Patients diagnosed with MPS II.
  • Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.

Exclusion Criteria:

  • Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation.
  • Patients in whom lumbar puncture cannot be performed.
  • Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study.
  • Patients who have received other investigational products within 4 months before enrollment 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: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental: JR-141
IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with Adverse Events
Time Frame: 4 weeks
  • Adverse events
  • Laboratory tests
  • Vital signs
  • 12-lead electrocardiogram
  • Antibody
  • Infusion associated reaction
4 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Time Frame: 4 weeks
Plasma concentration of JR-141
4 weeks
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Time Frame: 4 weeks
Plasma concentration of JR-141
4 weeks
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
Time Frame: 4 weeks
4 weeks
HS/DS in CSF
Time Frame: 4 weeks
JR-141 concentration in CSF
4 weeks
Urinary total GAG
Time Frame: 4 weeks
4 weeks
Liver and spleen volumes
Time Frame: 4 weeks
4 weeks
Cardiac function
Time Frame: 4 weeks
4 weeks

Collaborators and Investigators

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

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.

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)

March 30, 2017

Primary Completion (Actual)

October 4, 2017

Study Completion (Actual)

October 4, 2017

Study Registration Dates

First Submitted

April 10, 2017

First Submitted That Met QC Criteria

April 20, 2017

First Posted (Actual)

April 25, 2017

Study Record Updates

Last Update Posted (Actual)

November 14, 2022

Last Update Submitted That Met QC Criteria

November 10, 2022

Last Verified

November 1, 2022

More Information

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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