Clinical trials
Mucopolysaccharidosis II clinical trials
Below are recruiting mucopolysaccharidosis ii clinical trials, each written for real people, not researchers. We’re tracking 8 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05371613RecruitingPhase 2/Phase 3
Testing tividenofusp alfa vs idursulfase for MPS II
This trial compares a new treatment (tividenofusp alfa) to the current standard treatment (idursulfase) in children and young adults with MPS II. It’s looking to see which option works better and is safe, especially based on age and whether the disease affects the brain.
Oakland, CaliforniaAges 2–25 - NCT05619900Recruiting
Register people with lysosomal storage diseases
This study is a registry that collects information from people diagnosed with lysosomal storage diseases. It helps researchers better understand these conditions and support future studies.
San Francisco, CaliforniaAges Up to 64 years - NCT06036693Recruiting
MPS Long-Term Study
This study follows people with MPS, a rare genetic condition, to learn more about the disease over time. If you have a confirmed MPS diagnosis, you may be able to join.
AngersAges Any age - NCT05208281RecruitingPhase 2/Phase 3
GNR-055 for Hunter syndrome after standard enzyme treatment
This trial tests the safety and effectiveness of a study medicine called GNR-055 in people with Hunter syndrome (MPS II). It may help researchers learn whether the treatment improves outcomes and how the body handles the medicine.
MoscowAges Any age - NCT05368038Enrolling by invitation
Newborn screening program for babies up to 4 weeks old
This trial tests a flexible newborn screening process that looks for certain conditions early in life. It may help by catching problems sooner, when treatment can be started earlier.
Brooklyn, New YorkAges Up to 4 weeks - NCT06075537Enrolling by invitationPhase 2/Phase 3
Long-term safety study of a MPS II treatment
This study is for people with MPS II (Hunter syndrome) who have already completed a previous Denali study. It will test a drug called tividenofusp alfa (DNL310) over a longer period to see how safe it is and how well it works.
Oakland, CaliforniaAges Up to 18 years - NCT05594992Enrolling by invitationPhase 3
Study extension of JR-141 for long-term Hunter syndrome safety
This is a follow-up study for people who already took JR-141 in an earlier trial for Hunter syndrome (MPS II). It checks how well the treatment works over the long term and monitors long-term safety.
Oakland, CaliforniaAges Any age - NCT02254863RecruitingPhase 1
Stem cell transplant into the spinal fluid for rare brain diseases
This Phase 1 trial tests a transplant using special donated stem-cell–like cells placed into the spinal fluid to treat certain inherited brain diseases. It is mainly designed to check safety, and it may offer benefit by targeting brain and nerve damage.
Durham, North CarolinaAges 1 week–22 years
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Mucopolysaccharidosis II trials by city
Studies with a site in or near these metro areas.
Mucopolysaccharidosis II trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for mucopolysaccharidosis ii?
- Yes. Clin2 currently lists 8 recruiting mucopolysaccharidosis ii studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a mucopolysaccharidosis ii trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a mucopolysaccharidosis ii trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.