Fragile X syndrome

This trial tests whether metformin, a common diabetes medication, can improve behavior, thinking, and language in children with fragile X syndrome. Children aged 2 to 16 will receive either metformin or a placebo for six months. Researchers will measure changes in behavior using …

This study tests an experimental drug called BPN14770 (zatolmilast) in men aged 18 to 45 with Fragile X syndrome, a genetic condition that causes intellectual disability and behavioral challenges. The trial compares the drug against a placebo to see if it improves cognitive abili…

This study aimed to develop a non-invasive prenatal test using fetal cells from a mother's blood to detect triplet repeat diseases like Huntington's disease, Fragile X syndrome, and certain types of muscular dystrophy and ataxia. Researchers enrolled 60 pregnant women at risk and…

This study tested a cannabidiol (CBD) gel, called ZYN002, applied to the skin in 215 children, teens, and young adults with Fragile X syndrome. The goal was to see if it could improve behavioral symptoms like irritability and social withdrawal. Participants received either the CB…

This study tested a drug called zatolmilast (BPN14770) in 163 male adolescents aged 9 to 18 with Fragile X syndrome. The trial had two parts: one to check how the drug moves through the body, and another where some teens got the drug and others got a placebo. Researchers measured…

This study tested a single dose of SPG601 in 10 adult men with Fragile X syndrome to see if it improves symptoms like attention and behavior. Participants were randomly assigned to receive either the drug or a placebo, and neither they nor their doctors knew which they got. The g…

This study tested whether metformin, a common diabetes medication, can help improve language, behavior, and weight problems in people with fragile X syndrome. 125 participants aged 6 to 35 took either metformin or a placebo for 4 months. Researchers measured changes in language s…

This study follows nearly 600 people with Smith-Magenis syndrome (SMS), a rare genetic condition, to track how their health, behavior, and development change over time. Researchers will perform detailed medical exams, genetic tests, and surveys to better understand the syndrome's…

This completed study at the NIH enrolled 96 children with known or suspected genetic disorders, including autism and Fragile X syndrome. The main goal was to give clinic staff hands-on experience and explore new research ideas. Participants received medical exams, lab tests, and …

This completed study at Rutgers University looked at how the brain and body communicate in 30 people with conditions like autism, ADHD, Alzheimer's, and Parkinson's. Participants performed natural movements like walking and pointing while their brain, heart, and motion signals we…

This study used special PET and MRI scans to look at brain activity in 27 men with autism, Fragile X syndrome, or no condition. The goal was to measure a brain chemical called glutamate, which helps nerve cells communicate. Researchers hope this will reveal how brain signaling di…

This study looked at how a single dose of gaboxadol affects brain activity in adult males with Fragile X syndrome. Ten participants aged 18 to 40 took either the drug or a placebo during home and clinic visits. The main goal was to measure changes in brain waves using EEG, not to…