study details
start date: February 28, 2022
estimated completion: August 31, 2024
last updated: February 2, 2022
phase of development:
Phase 3
size / enrollment: 50
primary outcomes:
- Change in levels of Cerebrospinal Fluid Heparan Sulfate from baseline (Cohort A)
- Baseline to Week 53,105
Change in cognitive testing measured from baseline (BSID-III) (Cohort A)
- Baseline to Week 53, 105
Change in liver volume relative to body weight from baseline. (Cohort A and Cohort B)
- Baseline to Week 53
Change in spleen volume relative to body weight from baseline. (Cohort A and Cohort B)
- Baseline to Week 53
secondary outcomes:
- Change in adaptive behavioral testing measured from baseline (Vineland Adaptive Behavior Scales) (Cohort A)
Screening to Week 53, 105
- Change in levels of Cerebrospinal Fluid heparan sulfate from baseline. (Cohort B)
Baseline to Week 53
- Change in the Test of Variables of Attention (T.O.V.A.) from baseline. (Cohort B)
Screening to Week 53
- Change in the absolute Forced Vital Capacity (FVC) from baseline. (Cohort B)
Baseline to Week 53
- Change in liver volume relative to body weight from baseline. (Cohort A)
Baseline to Week 105
inclusion criteria:
• Eligible Ages: 0 - 120
• Eligible Sexes: male
Inclusion Criteria:
A patient from whom a voluntarily signed written Institutional Review Board or Independent Ethics Committee-approved informed consent form can be obtained. If the patient is aged under 18 years at the time of assent or willingness to participate in the study cannot be confirmed due to MPS II-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g. his parents or guardians) needs to be obtained instead of his consent or assent. (however, written consent should be obtained from the patient himself too, whenever possible)
Males with confirmed diagnosis of MPS II
Naïve patients or patients who are receiving stable enzyme replacement therapy with idursulfase for more than 12 weeks before starting the JR-141 administration.
Male patients whose co-partners are of child-bearing potential agree to use a medically accepted, highly effective method plus an approved method of effective contraception from the time of informed consent.
Males aged 36-71 months old whose standard score by BSID-III or KABC-II is between 65-85 at screening; OR
Males aged 30-35 months old at screening, with presence of the mutation in the IDS gene, and who are judged as having the severe phenotype by the Expert Board.
Males aged 6 years or older and whose IQ are 70 and higher.
Attenuated patients with 1 SD deficiency in the omission errors or variability domains of the TOVA test.
exclusion criteria: Criteria:
Use of gene therapy or hematopoietic stem cell transplantation (HSCT), excluding those who need enzyme replacement therapy even after HSCT.
Unable to undergo lumbar puncture.
A patient who has received other investigational product (drug or device) within 4 months of study enrollment.
Unable to comply with the protocol as determined by the principal investigator or subinvestigator.
Judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity including sensitivity to anesthesia or hypersensitivity to any component of JR-141.
A patient who has a known or suspected local or general infection or is at risk of abnormal bleeding due to a medical condition or therapy.A patient who otherwise is judged by the principal investigator or subinvestigator to be ineligible to participate in the study.
