Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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3 July 2023 |
Main ID: |
NCT03708965 |
Date of registration:
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27/09/2018 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
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Scientific title:
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An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II |
Date of first enrolment:
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January 1, 2019 |
Target sample size:
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19 |
Recruitment status: |
Active, not recruiting |
URL:
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https://clinicaltrials.gov/show/NCT03708965 |
Study type:
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Interventional |
Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Brazil
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Key inclusion & exclusion criteria
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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.
Age minimum:
0 Years
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis II
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Intervention(s)
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Drug: JR-141
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Primary Outcome(s)
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Number of participants with Adverse Events
[Time Frame: From screening up to the end of study, up to approximately 5 years]
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Secondary Outcome(s)
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Urinary heparan sulfate concentrations
[Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years]
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Echocardiography
[Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years]
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Serum heparan sulfate concentrations
[Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years]
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Serum dermatan sulfate concentrations
[Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years]
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Liver and spleen volumes (MRI)
[Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years]
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Urinary dermatan sulfate concentrations
[Time Frame: through study completion, an average of 52 weeks, up to approximately 5 years]
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Secondary ID(s)
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JR-141-BR22
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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