CompletedPhase 2/3

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Japan28 participantsStarted 2018-08-01

Plain-language summary

A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.

Who can participate

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Capable of providing written consent by himself (not mandatory for those aged under 20 years at the time of informed consent process, or those who is impossible to obtain consent from the patient himself due to intellectual disabilities associated with MPS II.)
. In the case of a patient who is under the age of 20 years or from whom it is not possible to obtain consent due to intellectual disabilities associated with MPS 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, whenever possible)
. Males with confirmed diagnosis of MPS II, based on deficient activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic mutations identified in the IDS gene, etc.
. Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.

Exclusion criteria

. Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
. Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely 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 serious drug allergy or sensitivity.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid

Timeframe: Baseline to 52 weeks

Trial details

NCT IDNCT03568175

SponsorJCR Pharmaceuticals Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-02-20

View on ClinicalTrials.gov More Mucopolysaccharidosis II trials