Long-term safety study of a MPS II treatment
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have completed the previous MPS II study (DNLI-E-0002 or DNLI-E-0007).
- From study DNLI-E-0002: you must have finished at least the Week 49 visit and not stopped treatment early.
- From study DNLI-E-0007: you must have finished the full treatment period (96 weeks for some, 48 weeks for others).
- You cannot have other unstable medical conditions that would make participation unsafe.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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.
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.
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.
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.
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.
This trial tests a new medicine called GC1130A for children with Sanfilippo syndrome type A (MPS IIIA). The goal is to see if it is safe and if it might help with symptoms.
Hear when a new Mucopolysaccharidosis II trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.