Clinical trials
Mucopolysaccharidosis III clinical trials
Below are recruiting mucopolysaccharidosis iii clinical trials, each written for real people, not researchers. We’re tracking 5 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 - 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 - 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 - 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+ - 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 mucopolysaccharidosis iii?
- Yes. Clin2 currently lists 5 recruiting mucopolysaccharidosis iii 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 iii 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 iii 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.