Glycerol phenylbutyrate for children with urea cycle disorders
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a medicine called glycerol phenylbutyrate in children with urea cycle disorders, which are genetic conditions that make it hard for the body to get rid of extra ammonia. The goal is to see if the medicine can help lower ammonia levels and manage the condition safely.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Children from birth to 18 years old can join.
- You must have one of these urea cycle disorder types: CPS1 deficiency, ornithine translocase deficiency, citrullinemia type I, argininosuccinic aciduria, argininemia, or HHH syndrome.
- You have not taken glycerol phenylbutyrate in the past 3 months.
- If you are old enough to have children, you must agree to use effective birth control during the study and for 1 month after the last dose.
- You must be willing to follow the study's diet and visit schedule.
- You cannot be allergic to any ingredients in the medicine.
- You cannot take certain other drugs (like valproate or corticosteroids) within 24 hours before starting the study.
- You cannot be pregnant or breastfeeding.
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 new medicine called LNP.UCD.ABE that aims to fix a specific genetic change causing a severe urea cycle disorder. It uses a technique called base editing to correct the gene mutation, which may help your body break down ammonia better.
This study measures how your body breaks down protein-related waste and makes urea. It includes both healthy volunteers and people with urea cycle disorders, which may help researchers better understand the condition and improve testing.
This trial tests a device for measuring ammonia levels at home in people with rare metabolic conditions that cause high ammonia. It aims to make monitoring easier and more convenient for patients.
This study tests an investigational drug called KRRO-121. It is first tested in healthy volunteers to check safety, and then in patients with urea cycle disorder (a condition where the body can't remove ammonia properly) to see if it helps.
This trial studies blood and other measurable signs (biomarkers) that may reflect brain injury after certain inherited metabolic conditions. Results may help doctors better detect and understand brain effects from these illnesses, especially episodes that affect the brain.
This study tests if a medicine called Glycerol Phenylbutyrate (RAVICTI) is safe and helpful for children and young adults with a specific type of PDH deficiency, a rare genetic condition affecting energy use. If you qualify, you may get a new treatment option.
Hear when a new Urea Cycle Disorders trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.