Study of an experimental drug for phenylketonuria (PKU)
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests an investigational drug (AG-181) for people with PKU who have a specific genetic change (R408W mutation). It aims to check if the drug is safe and how it works in your body.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have PKU with at least one R408W gene mutation.
- Your blood phenylalanine (Phe) levels must have been above 600 µmol/L in the past year and during screening.
- Your body mass index (BMI) must be between 18 and 35, and you must weigh at least 50 kg (110 lbs).
- A dietitian must approve your ability to keep a stable diet during the study.
- You cannot have taken the study drug before, and you must stop certain other medications (like some stomach acid reducers or PKU treatments) before starting.
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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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 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.
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