Testing ACER-001 for combined D,L-2 hydroxyglutaric aciduria
Treatments studied
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a drug called ACER-001 in a single patient with a rare metabolic disorder. The goal is to see if the drug helps manage symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have combined D,L-2 hydroxyglutaric aciduria (a rare metabolic condition).
- A parent or guardian gives permission at the start of the study.
- Parents agree to follow the study plan.
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 medicine called sodium phenylbutyrate (ACER-001) to see if it helps people with MCADD, a genetic condition that affects how your body breaks down fats. If you have MCADD and the common gene change, you might be able to join.
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.
This trial tests a new gene therapy (VGM-R02b) for children with Glutaric Acidemia Type 1 (GA-1) whose symptoms are not well controlled by standard treatment. The goal is to see if the therapy is safe and can help improve neurological symptoms.
This Phase 2 trial tests whether adding D-galactose can help people with a confirmed SLC35A2 genetic condition. It aims to improve symptoms such as seizures and ongoing stomach problems like vomiting or diarrhea.
This early-stage trial tests VGN-R09b, a gene therapy, given to children with AADC deficiency who are not doing well on standard medicines. It aims to improve symptoms and quality of life by addressing the underlying cause of AADC deficiency.
This trial tests whether new protein substitute tablets are acceptable for people with certain rare metabolic disorders. Participants will replace their usual protein substitute with the study tablets for 7 days to see if they can tolerate them.
Hear when a new Combined D,L-2-hydroxyglutaric Aciduria trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.