Sepiapterin study for phenylketonuria
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a new medicine called sepiapterin to see if it can help control blood phenylalanine levels in people with PKU. The trial aims to find a safe and effective dose for different age groups.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be diagnosed with PKU and have high blood phenylalanine levels in your medical history.
- Your recent blood phenylalanine levels must be within a specific range (120 to 480 micromol/L).
- If you are a woman who could become pregnant, you must agree to use contraception.
- You must be willing to stick to a controlled diet (prescribed protein and phenylalanine) during the trial.
- You cannot have taken certain PKU medicines (like Kuvan) in the last 3 months.
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 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.
This study looks at how a phenylalanine-restricted diet or certain PKU medicines affect gut health in children with PKU. It also includes healthy siblings or household members as a comparison group.
This trial tests a new pill called JNT-517 to see if it can lower high blood phenylalanine (Phe) levels in people with PKU. You may be a good fit if you have PKU, are at least 18, and can stick to a steady diet.
This trial tests a new powdered protein substitute (GMP) for people with PKU or tyrosinemia. It aims to see if it is a safe and effective option to help manage their condition.
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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