UC Davis Health Clinical Studies

A Study of the Long-Term Safety and Tolerability of Experimental ZYN002 in Children and Adolescents With Fragile X Syndrome (FXS)

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Fragile X Syndrome (FXS) is a genetic condition caused by a mutation in the Fragile X mental retardation 1 (FMR1) gene. This mutation is thought to cause changes to the “endocannabinoid system.” This is a certain pathway in the central nervous system. Disruption in this pathway can lead to FXS symptoms such as: - mild to severe intellectual or developmental disability - speech and language difficulties - behavioral, social and emotional difficulties. Cannabidiol (CBD) is part of the Cannabis/Marijuana plant. However, it does not have the same effects on the brain that recreational marijuana has. It may affect the endocannabinoid pathway. The Drug Product ZYN002 is a pharmaceutically manufactured CBD. It is being developed as a clear gel that can be applied to the skin (called transdermal delivery). This provides consistent, controlled levels of CBD in the blood when it is given once or twice a day. The purpose of this study is to evaluate the long-term safety, tolerability and effectiveness of the experimental drug ZYN002. It will be used in the treatment of symptoms of Fragile X Syndrome (FXS). There are currently no FDA approved medications shown to treat FXS. ZYN002 is an experimental treatment. This means that it is not approved by the FDA and must be tested to see if it is an effective treatment.

Drug study, Phase [/2/,/ /3/]
Any, age 3 to 18 years old

Telehealth study about a parent-implemented language test for individuals with Autism Spectrum Disorder

Estudio de telesalud sobre una prueba de lenguaje aplicada por los padres en el hogar para personas con Trastorno del Espectro Autista

We are now recruiting parents along with their sons/daughters with ASD for a new study! The study aims to teach parents to administer a spoken language test to their sons/daughters in their homes via telecommunication technology (i.e. Skype). We also will be adapting the language test for use with Spanish-speaking families. Therefore, we are recruiting both families who primarily speak Spanish as well as families who primarily speak English with their children. Estamos reclutando padres junto con sus hijos/as con TEA para un nuevo estudio! El objetivo del estudio es enseñar a padres a dar una prueba de lenguaje a sus hijos/hijas en sus hogares a través de las tecnologías de telecomunicaciones (ej., Skype). También adaptaremos la prueba al español. Por lo tanto, estamos reclutando tanto a familias que hablen principalmente inglés en casa con sus hijos como a familias que hablen principalmente español con sus hijos.

outcome measures/ medidas de resultado
Any, age 6 to 23

Study on Language Development in Young Adults with Fragile X Syndrome

The goal of the study is to learn more about how certain abilities (e.g. memory), certain types of experiences (e.g. interactions with family), and biology affect language abilities in adulthood for both males and females with fragile X syndrome. We are also interested in understanding how these factors and language/literacy skills affect the abilities of these young adults to function independently. Such information will help us suggest ways to improve instruction, therapy, and support to help youth fragile X to successfully transition from school to the demands of adult life.

Any, age in, entering, or just out of last year of HS

A Study of the Experimental Medicine OV101 For Fragile X Syndrome

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We invite you to take part in a research study because your child has a rare genetic disorder called Fragile X syndrome (FXS). You are being asked to give permission for your child to take part in the study. If you are a parent/legally authorized representative (LAR), you are being asked to take part in this study as detailed in this consent form. Before you decide if you want to take part, it is important for you to understand why the research is being done and what it will involve. Please read the following information carefully and discuss it with friends, family and physician if you wish. The study doctor or study staff will also explain the study to you.

Drug study, Phase 2
Male, age 13 to 22 years old

In-Home Autism Study

Are you a parent of a child with Autism?

Researchers at the UC Davis MIND Institute are collaborating with the Hartwell Foundation, Stanford University and Case Western University to reach thousands of families who are managing the challenges of caring for a child with an Autism Spectrum Disorder diagnosis. As its name implies, Autism Spectrum Disorder (ASD) represents a spectrum of symptoms, behaviors and associated features that medical science generally refers to as autism. It is the goal of the "Kids First" initiative to create a detailed classification of ASD that will more accurately reflect each individual's behavior and personality. With this new approach, researchers will be able to dig beyond the surface features of the disorder, to enact personalized, targeted approaches for care and interventions that will be more successful than what is available today.

Any, age 1-17

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