Clinical trials
Mucopolysaccharidosis I clinical trials
Below are recruiting mucopolysaccharidosis i clinical trials, each written for real people, not researchers. We’re tracking 19 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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 - 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 - 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 - 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 - 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 - 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+ - NCT06488924RecruitingPhase 1/Phase 2
Study of JR-446 for Sanfilippo syndrome type B
This trial tests an experimental drug, JR-446, for children with Sanfilippo syndrome type B (MPS IIIB). The goal is to see if it is safe and can help with symptoms.
HiroshimaAges Up to 17 years - NCT06519552RecruitingPhase 1
Gene therapy study for adults with MPS type I
This study tests a new gene therapy called JWK008 for adults with MPS type I. The goal is to see if it is safe and can help improve symptoms. You may be able to join if you are 18 or older and have not had serious side effects from enzyme replacement therapy.
Chengdu, SichuanAges 18 years+ - 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 - 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+ - 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 - NCT06333041RecruitingPhase 2/Phase 3
CBD study for Sanfilippo syndrome
This study tests whether a cannabis-derived medicine called cannabidiol (CBD) can help children with Sanfilippo syndrome, a rare genetic disorder. It may be a good fit for children who have already tried other treatments or whose disease is more advanced.
Torrance, CaliforniaAges 4 years+ - 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 - 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 - 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 - NCT06103487Enrolling by invitation
Long-term follow-up for RGX-111 treatment
This study follows participants who previously received the RGX-111 treatment to monitor their health over time. It helps researchers understand the long-term effects and safety of the therapy.
Orange, CaliforniaAges Any age - 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 - 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
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Common questions
- Are there clinical trials for mucopolysaccharidosis i?
- Yes. Clin2 currently lists 19 recruiting mucopolysaccharidosis 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 mucopolysaccharidosis 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 mucopolysaccharidosis 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.