Back to resultsAn Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Primary Purpose
Mucopolysaccharidosis II
Status
Active
Phase
Phase 2
Locations
Brazil
Study Type
Interventional
Intervention
JR-141
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis II
Eligibility Criteria
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.
Sites / Locations
- Grupo de Pesquisa Clínica em Genética Médica - HCPA
- Igeim - Unifesp
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
JR-141
Arm Description
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.
Outcomes
Primary Outcome Measures
Number of participants with Adverse Events
Secondary Outcome Measures
Urinary heparan sulfate concentrations
Urinary dermatan sulfate concentrations
Serum heparan sulfate concentrations
Serum dermatan sulfate concentrations
Liver and spleen volumes (MRI)
Echocardiography
Full Information
NCT ID
NCT03708965
First Posted
September 27, 2018
Last Updated
June 22, 2023
Sponsor
JCR Pharmaceuticals Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT03708965
Brief Title
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Official Title
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 1, 2019 (Actual)
Primary Completion Date
March 31, 2024 (Anticipated)
Study Completion Date
March 31, 2024 (Anticipated)
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 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.
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
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)
8. Arms, Groups, and Interventions
Arm Title
JR-141
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
JR-141
Intervention Description
IV infusion (lyophilized powder)
Primary Outcome Measure Information:
Title
Number of participants with Adverse Events
Time Frame
From screening up to the end of study, up to approximately 5 years
Secondary Outcome Measure Information:
Title
Urinary heparan sulfate concentrations
Time Frame
through study completion, an average of 52 weeks, up to approximately 5 years
Title
Urinary dermatan sulfate concentrations
Time Frame
through study completion, an average of 52 weeks, up to approximately 5 years
Title
Serum heparan sulfate concentrations
Time Frame
through study completion, an average of 52 weeks, up to approximately 5 years
Title
Serum dermatan sulfate concentrations
Time Frame
through study completion, an average of 52 weeks, up to approximately 5 years
Title
Liver and spleen volumes (MRI)
Time Frame
through study completion, an average of 52 weeks, up to approximately 5 years
Title
Echocardiography
Time Frame
through study completion, an average of 52 weeks, up to approximately 5 years
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Grupo de Pesquisa Clínica em Genética Médica - HCPA
City
Porto Alegre
Country
Brazil
Facility Name
Igeim - Unifesp
City
São Paulo
Country
Brazil
12. IPD Sharing Statement
Learn more about this trial
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
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