Active — Not RecruitingPhase 2/3

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Japan27 participantsStarted 2019-09-01

Plain-language summary

Multicenter, open-label, single-group, designed to evaluate the long term 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

. A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator.
. A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.)
. For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.)
. Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception.

Exclusion criteria

. A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator.
. A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator.
. A patient judged to be ineligible by the investigator or subinvestigator for other reasons.

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

Occurrence of adverse events

Timeframe: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years

Occurrence of adverse reactions

Timeframe: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years

Incidence of abnormal vital signs

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Incidence of abnormal vital signs

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Incidence of abnormal vital signs

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Laboratory tests (urinalysis)

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Vital signs (pulse rate)

Trial details

NCT IDNCT04348136

SponsorJCR Pharmaceuticals Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-03-31

View on ClinicalTrials.gov More Mucopolysaccharidosis II trials

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

. A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator.
. A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.)
. For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.)
. Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception.

Exclusion criteria

. A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator.
. A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator.
. A patient judged to be ineligible by the investigator or subinvestigator for other reasons.

What they're measuring

Occurrence of adverse events

Timeframe: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years

Occurrence of adverse reactions

Timeframe: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years

Incidence of abnormal vital signs

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Incidence of abnormal vital signs

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Incidence of abnormal vital signs

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Laboratory tests (urinalysis)

Timeframe: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Vital signs (pulse rate)