An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

June 22, 2023 updated by: JCR Pharmaceuticals Co., Ltd.
A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

19

Phase

  • Phase 2

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

  • Brazil
      • Porto Alegre, Brazil
        • Grupo de Pesquisa Clínica em Genética Médica - HCPA
      • São Paulo, Brazil
        • Igeim - Unifesp

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

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients who will have completed clinical trial JR-141-BR21.
  • Capable of providing written consent by himself, unless the patient is under the age of 18 years at the time of informed consent process, or it is not possible to obtain consent from the patient himself due to his intellectual disabilities associated with MPS II.
  • In the case of a patient who is under the age of 18 years or from whom it is not possible to obtain consent due to his intellectual disabilities associated with MSP 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, wherever possible.

Exclusion Criteria:

  • Refusal to sign the informed consent form.
  • Unable to perform the study procedures, except for neurocognitive testing.
  • Previous engrafted BMT/HSCT.
  • Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumber puncture due to joint contracture or those who are likely to 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 a serious drug allergy or sensitivity.
  • Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study out of consideration for the subject safety.

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
Intervention / Treatment
Experimental: JR-141

Subjects will be assigned to 1.0, 2.0 or 4.0 mg of JR-141 per kg of body weight once every week (the same dose taken during the previous study) in the beginning of the study.

During the study, the dose of all subjects will be switched to the selected one*.

* The dose was determined to be 2.0 mg/kg/week based on the safety and efficacy data of JR-141-BR21 study.
Drug: JR-141
IV infusion (lyophilized powder)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of participants with Adverse Events
Time Frame: From screening up to the end of study, up to approximately 5 years
From screening up to the end of study, up to approximately 5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Urinary heparan sulfate concentrations
Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years
through study completion, an average of 52 weeks, up to approximately 5 years
Urinary dermatan sulfate concentrations
Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years
through study completion, an average of 52 weeks, up to approximately 5 years
Serum heparan sulfate concentrations
Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years
through study completion, an average of 52 weeks, up to approximately 5 years
Serum dermatan sulfate concentrations
Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years
through study completion, an average of 52 weeks, up to approximately 5 years
Liver and spleen volumes (MRI)
Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years
through study completion, an average of 52 weeks, up to approximately 5 years
Echocardiography
Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years
through study completion, an average of 52 weeks, up to approximately 5 years

Collaborators and Investigators

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

Sponsor

JCR Pharmaceuticals Co., Ltd.

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)

January 1, 2019

Primary Completion (Estimated)

March 31, 2024

Study Completion (Estimated)

March 31, 2024

Study Registration Dates

First Submitted

September 27, 2018

First Submitted That Met QC Criteria

October 16, 2018

First Posted (Actual)

October 17, 2018

Study Record Updates

Last Update Posted (Actual)

June 23, 2023

Last Update Submitted That Met QC Criteria

June 22, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • JR-141-BR22

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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