Gene therapy trial for classic PKU
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 to 55 years old.
- You have classic PKU caused by a specific PAH gene mutation.
- Current PKU medications like Kuvan or Palynziq do not work well for you, or you cannot take them.
- Your blood phenylalanine level has been at least 600 μmol/L in the last 6 months while on a regular diet.
- You are willing to follow a diet plan and record what you eat during the study.
- 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.
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Other trials that look related to this one.
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