Gene therapy injection for adults with phenylketonuria
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a new one-time gene therapy injection (called GS1168) for adults with phenylketonuria (PKU). It aims to help your body break down the amino acid phenylalanine better, so your diet may become less strict.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 to 55 years old.
- You must have PKU caused by a change in the PAH gene.
- Your PKU must not be well controlled with your current diet or other treatments.
- If you take Kuvan or Palynziq, you must have stopped them at least 28 days before joining.
- You need to weigh less than 100 kg and have a BMI below 30.
- You must agree to avoid alcohol for the study duration and use birth control if you could become pregnant.
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 tests a new injection called PL54 to see if it is safe and tolerable for adults with phenylketonuria (PKU), a condition where the body cannot break down an amino acid called phenylalanine. It is designed for people with high blood phenylalanine levels and may offer a new treatment option.
This trial tests an experimental medication taken as a pill to see if it can help people with PKU manage their condition. The study will check if the pill is safe and how it affects blood phenylalanine levels.
This study tests a new medicine called JNT-517 for people with phenylketonuria (PKU) over a long period. It aims to see if the drug can help manage PKU safely and effectively.
This study is testing whether a new microtablet form of protein substitute is easy and acceptable for people with PKU to take. If you qualify, you'll take these tablets instead of your current protein substitute and share your experience with the research team.
This study looks at whether checking phenylalanine levels at home more often helps people with classic PKU keep their levels in a healthy range. It may help you manage your diet and treatment more effectively.
This study looks at adults with phenylketonuria (PKU) using advanced testing (called multiomics) to understand how the condition affects the body. If you took part in a previous PKU study (ECOPHEN) and are willing to give a saliva sample for DNA analysis, this trial may be for you.
Hear when a new Phenylketonuria (PKU) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.