UC Davis Health Clinical Studies

AFQ056 for Language Learning in Children With FXS

If your child is between the ages of 32 months and 6 years and has fragile X syndrome, we invite them to take part in this study.

There are two purposes of this study: 1.) To find out if the drug AFQ056, which is made by the pharmaceutical company named Novartis, is safe and has beneficial effects in children who have fragile X syndrome (FXS). 2.) To find out if a structured language intervention can help children with fragile X syndrome communicate better. AFQ056 is an investigational drug that is not approved for use for the treatment of people with FXS by the U.S. Food and Drug Administration (FDA). It is not currently “on the market” (available for you to buy) in any country. Fragile X syndrome is a genetic disorder – which means that your child was born with it. We all have unique characteristics that make us individual. These characteristics are carried by "genes‟ in our body's cells. In fragile X syndrome, part of a gene is damaged. The current theory is that this gene is "switched off‟ in patients with fragile X syndrome. It cannot produce enough of a protein the brain needs. This protein is called FMRP and helps brain cells respond to messages properly. AFQ056 is an oral (given by mouth) study drug. It is being developed for patients with fragile X syndrome. It blocks a specific type of glutamate (a form of brain chemical or “neurotransmitter”) receptor called the mGluR5 receptor in the brain. A receptor can be thought of as a "lock‟ and AFQ056 as a "key‟ which fits into the lock to stop it from being active. The mGluR5 receptor is thought to be overactive in patients with fragile X syndrome due to lack of FMRP. AFQ056 can block the mGluR5 receptor and decrease activity in the mGluR5 pathway. By blocking the mGluR5 receptor and pathway in the brain, it is thought that AFQ056 may be able to compensate for some of the function of the missing FMRP. Thus, symptoms of fragile X Syndrome may be reduced. We are asking if your child can take part in this research study because he/she is between the ages of 32 months and 6 years and has fragile X syndrome.

Drug study, Phase 2
Any, age 2 to 6 years old

CleanUP IPF for the Pulmonary Trials Cooperative

The study team thinks long-term antibiotic therapy can reduce hospital stays for respiratory problems.

We invite you to take part in a research study because your doctor has determined that you have idiopathic pulmonary fibrosis (IPF). Idiopathic pulmonary fibrosis is a rare but serious lung disease. It causes trouble breathing because the lungs develop increased scar tissue over time. “Idiopathic” means that we do not know what causes the scarring to develop. The study team thinks there is a relationship between long-term antibiotic therapy and a reduction in hospital stays for respiratory problems. The purpose of this study is to see if standard care plus antibiotic therapy reduces harmful infections. These infections could lead to hospitalization or death in patients diagnosed with IPF.

Drug study, Phase 3
Any, age 40 years or older
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