Mucopolysaccharidosis II
Conditions
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.
Interventions
DRUGJR-141
IV infusion (lyophilized powder)
Sponsors
JCR Pharmaceuticals Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
0 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of participants with Adverse Events | From screening up to the end of study, up to approximately 5 years |
Secondary
| Measure | Time frame |
|---|---|
| Urinary heparan sulfate concentrations | through study completion, an average of 52 weeks, up to approximately 5 years |
| Urinary dermatan sulfate concentrations | through study completion, an average of 52 weeks, up to approximately 5 years |
| Serum heparan sulfate concentrations | through study completion, an average of 52 weeks, up to approximately 5 years |
| Serum dermatan sulfate concentrations | through study completion, an average of 52 weeks, up to approximately 5 years |
| Liver and spleen volumes (MRI) | through study completion, an average of 52 weeks, up to approximately 5 years |
| Echocardiography | through study completion, an average of 52 weeks, up to approximately 5 years |
Countries
Brazil
Outcome results
None listed