An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB (NCT06488924) | Clinical Trial Compass
RecruitingPhase 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Japan10 participantsStarted 2024-11-05
Plain-language summary
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
Who can participate
Age range
17 Years
Sex
ALL
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:
* Chronological age of \<18 years
* Confirmed diagnosis of MPS IIIB
Exclusion Criteria:
* Prior experience to gene therapy or HSCT with successful engraftment
* Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF
* Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures
* Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF
* Serious drug allergy or hypersensitivity
* Contraindication for lumbar puncture or MRI
* History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture
The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
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
1
To establish the safety and tolerability of JR-446 in MPSIIIB patients
Timeframe: up to 4 years (multiple visits)
2
To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients