Clinical trials
MPS I clinical trials
Below are recruiting mps i clinical trials, each written for real people, not researchers. We’re tracking 17 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07361536Recruiting
Heart health in MPS
This study looks at heart structure and function in people with mucopolysaccharidosis (MPS), a rare genetic disorder. It aims to understand how MPS affects the heart over time.
Orange, CaliforniaAges birth–99 years - NCT04532047RecruitingPhase 1
Fetal enzyme therapy trial for certain inherited storage disorders
This Phase 1 trial tests an enzyme replacement treatment given to a developing baby before birth for specific inherited “lysosomal storage” diseases. The goal is to see if the treatment is safe and can improve outcomes for babies diagnosed in pregnancy.
San Francisco, CaliforniaAges 18–50 - NCT03333200Recruiting
Study of genetic brain diseases over time
This trial follows people with genetic neurodegenerative (brain-wasting) disorders over time to better understand how the condition changes. The goal is to learn patterns that could help future treatments or care plans.
Pittsburgh, PennsylvaniaAges Any age - NCT07449143Recruiting
Physical activity program for adults with MPS
This study tests a physical activity program for adults with MPS in the UK. It aims to help with movement and daily function through group workshops.
LondonAges 18 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 - NCT05492799Enrolling by invitationPhase 4
Study of AX 250 for MPS IIIB in children
This Phase 4 trial studies how safe, tolerable, and helpful ICV AX 250 is for people with MPS IIIB. It is for participants who already finished a prior AX 250 study and meet specific health and eligibility checks.
Oakland, CaliforniaAges Any age - NCT04360265Enrolling by invitationPhase 3
Follow-up gene therapy study for MPS IIIA participants
This follow-up study looks at how a past gene therapy treatment (UX111) is affecting you over time. It mainly involves continued visits and safety checks, and it helps researchers understand long-term benefits and risks.
Columbus, OhioAges Any age - NCT03604835Recruiting
MPS VII monitoring program for patients and caregivers
This trial enrolls people with mucopolysaccharidosis type VII (MPS VII) to track their health over time. It may help your care team better understand how the condition behaves and how monitoring should be done.
Orange, CaliforniaAges Any age - NCT07640984RecruitingPhase 1/Phase 2
Gene therapy for rare childhood brain disorder MPS IIIB
This early-stage trial tests JR-446, a new treatment for MPS IIIB, a rare genetic disorder that affects the brain and body. The treatment is delivered directly into the fluid around the spinal cord and aims to slow or improve symptoms in young children.
Oakland, CaliforniaAges Up to 5 years - NCT07494162Recruiting
Acupuncture for chronic low back pain with muscle knots
This trial tests if acupuncture can help chronic low back pain by looking at changes in muscle metabolism and blood flow. It compares real acupuncture with a sham (fake) procedure to see if there is a true benefit.
Sacramento, CaliforniaAges 18 years+ - NCT07098754Recruiting
Physical therapy for neck pain and posture improvement
This study compares different physical therapy techniques to improve neck posture and reduce pain in people with myofascial pain syndrome. It may help if you have mild to moderate neck pain from muscle knots and are open to physiotherapy.
Bahawalpur, Punjab ProvinceAges 20–40 - NCT07082972RecruitingPhase 4
Study of splint therapy and Botox for jaw muscle pain
This trial compares two treatments—a dental splint and Botox injections into the jaw muscle (masseter)—to see which one better relaxes head and neck muscles in people with myofascial pain syndrome. It may help you if you have facial pain related to muscle tension.
Istanbul, VeznecilerAges 18–55 - 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 - NCT05682144RecruitingPhase 1
Cell therapy for MPS I to improve immune function
This Phase 1 study tests an engineered cell therapy made to help the body in people with MPS I (Hurler-Scheie or Scheie). It is early-stage research, so the main goal is to learn about safety and the best way to treat.
Oakland, CaliforniaAges 10 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 - NCT05284006Recruiting
Non-invasive tests for Morquio A
This trial studies Morquio A (MPS IVA) using non-invasive ways to measure how the body is working, and to better understand why the condition happens. If you have Morquio A, this may help researchers learn more and improve care.
Wilmington, DelawareAges Any age - NCT02716246RecruitingPhase 2/Phase 3
Gene therapy for MPS IIIA to slow brain decline
This trial tests a gene-transfer treatment (delivered into the spinal fluid) for children with MPS IIIA, a rare genetic condition. It aims to improve or slow down brain and developmental changes caused by a missing enzyme.
Columbus, OhioAges Any age
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Common questions
- Are there clinical trials for mps i?
- Yes. Clin2 currently lists 17 recruiting mps i 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 mps i 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 mps i 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.