Immune therapy during Palynziq treatment for PKU
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests whether combining a drug called methotrexate with Palynziq (a treatment for PKU) can help control immune responses and improve treatment outcomes. It may be an option for adults with PKU who have never used Palynziq or who have not reached target blood levels after at least 24 weeks.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 65 years old
- You have been diagnosed with PKU (phenylketonuria)
- You have not taken Palynziq before and are willing to start it during the study, OR you have been on Palynziq for at least 24 weeks with high blood levels despite taking at least 20 mg daily
- You must carry two epinephrine auto-injectors at all times while on Palynziq
- You agree to use effective birth control if pregnancy is possible
- You do not have severe allergies to methotrexate, Palynziq, or PEG-containing medications
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.
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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 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.
This study looks at how educational, social support, and nutrition help affect pregnancy outcomes and quality of life in women with PKU. It may help you better manage PKU-related needs before and during pregnancy.
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 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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