Gene therapy for phenylketonuria
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy, NGGT002, which delivers a healthy gene to help people with phenylketonuria (PKU) manage their phenylalanine levels. It may reduce the need for strict diet control.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 55 years old.
- You have been diagnosed with PKU and have high blood phenylalanine levels (≥600 μmol/L) in the past two years.
- You have not responded to or cannot take current PKU medications like sapropterin or pegvaliase.
- You are willing to manage your diet and follow study procedures.
- You have no active infections, liver disease, or history of cancer.
- You have not had gene therapy before.
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 tests a one-time gene therapy called NGGT002 for adults with classic PKU who cannot take or do not respond well to current treatments. The goal is to see if it can safely lower blood phenylalanine levels.
This study tests whether a specific mix of large neutral amino acids (LNAAs) can help adults with classical PKU by lowering phenylalanine levels in the brain. It includes an MRI and PET scan to see how the treatment affects brain activity.
This study looks at whether following specific care guidelines for phenylketonuria (PKU) leads to better metabolic control (blood phenylalanine levels) and better patient well-being. You may be asked to complete questionnaires and follow the study plan while receiving the usual PKU treatment your clinic recommends.
This study looks at how nutrition affects risk factors in adults with PKU (a condition that requires a special diet to control certain blood chemicals). If you can manage your diet independently and agree to follow the study diet plan, this may help researchers find better nutritional approaches.
This study tests a new injection called GS1168 for adults with phenylketonuria (PKU) whose current treatments are not working well. The goal is to see if it can help control their PKU better.
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.
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