Testing tividenofusp alfa vs idursulfase for MPS II
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child’s age is 2 to under 6, or 6 to under 26
- They have a confirmed diagnosis of MPS II (either neuronopathic or non-neuronopathic)
- They have not had enzyme replacement treatment, or haven’t had it continuously for 4 months before screening, or they’ve been on idursulfase for at least 4 months and tolerated it
- They have not had gene therapy for MPS II (IDS) or any stem cell therapy for this condition
- They have no reason they can’t safely get a lumbar puncture (spinal tap) and/or MRI scan
- They haven’t joined another drug study or taken another investigational drug in the last 60 days (and won’t during this study)
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 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.
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 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.
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.