- Fragile X-associated Disorders
- Treatment & Intervention
- Support the NFXF
The Fragile X Clinical & Research Consortium (FXCRC) is a group of clinics and research facilities across the United States that are dedicated to assisting families and individuals who are affected by fragile X syndrome (FXS) or one of the other Fragile X-associated Disorders. Most clinics are part of university and/or hospital settings. There, experts in the field of FXS can offer medical, therapeutic, and educational evaluations, treatment recommendations, and referrals to other important resources. The information gathered from patients and families can help expand knowledge about FXS to guide future treatment.
One of the best ways for families to help their doctors gather this information is to become part of the FORWARD Registry and Database.
Clinical trials (studies involving humans with the actual condition) of new medications for the treatment of Fragile X-associated Disorders (FXD) are currently underway at many sites throughout the U.S. and the world. These trials involve individuals with a FXD, including fragile X syndrome. Typically, these trials are sponsored by a pharmaceutical company seeking to determine the effectiveness of a new drug. Sometimes the studies involve older drugs previously used for treatment of conditions other than Fragile X. Studies typically go through three phases prior to being considered for approval by the government. Phase III studies begin only after effectiveness has been shown to occur in animals and after safety has been proven in individuals without a Fragile X disorder.
To ensure that the effects of the drugs are not influenced by the researchers hopes or expectations, most studies are “double-blind, placebo controlled.” That is, the researcher/clinician, prescribing the drug does not know who is receiving the actual drug and who is receiving a placebo (sugar pill). The parents or the actual participant also does not know. This process is considered the “gold-standard” of medical studies and helps make sure that only truly effective medicines end up as treatments.
Since these are studies only, following the conclusion of the study, in most cases the participants must stop taking the drug. However, in some placebo-controlled studies, all participants are allowed to go on the medication (not placebo) through a so-called “extension.” In this case usually the company is collecting data about long-term safety of the drug and the extension may go on for variable amounts of time, sometimes even until the drug is approved by the government. Should the studies prove that the drug is safe and effective, and once the drug is approved by the U.S. Food and Drug Administration (FDA), the individual may be prescribed the medication by his or her physician.
Not all studies involve drugs. Studies can involve behavior, language development and other important areas of intervention and development. These studies are as important as drug studies and will help doctors, therapists, educators and counselors improve treatments as well as take better advantage of new opportunities that may result from new drugs.
The NFXF also periodically assists with research recruitment through a variety of means including mailings, email blasts and Facebook postings. This is a fee-based service designed to promote the advancement of Fragile X research including on behalf of university-based researchers, pharmaceutical companies, and others who meet our general guidelines, while compensating the NFXF for the associated staff time and materials expense. If you prefer to not receive these notifications please click here.
If you are a family member and have any questions about a specific study, please use the contact information listed on the clinic’s announcement. If you are a professional and have any questions about how the NFXF can assist you with recruitment, please call (925) 938-9300.
We are recruiting a small group of mothers and their children with fragile X syndrome, between the ages of 10- and 17-years, to participate in a pilot study of a parent-implemented language intervention. The goal of this project is to teach each mother strategies which can be used to support spoken language development in their child with fragile X syndrome. » FULL ARTICLE
If you have a son with fragile X syndrome or a son with autism between 15 and 22 years of age, you and your son are invited to participate in a research study being conducted by Dr. Leonard Abbeduto at the MIND Institute University of California, Davis. The project is a collaborative effort with researchers at the University of South Carolina and at the New York State Institute for Basic Research on Developmental Disabilities. The goals of this study are to learn... » FULL ARTICLE
Genetic disorders in children: An assessment of parental coping strategies and positive growth Following on from our earlier study of how parents disclose a diagnosis of fragile X syndrome to their children, we would like to further understand your experiences surrounding your child, including your emotional reaction and psychological well-being. In particular, we are interested in the support that you as a caregiver have available, and how this helped you to cope with the experience. This is a questionnaire that you can do online. You can do this study even if... » FULL ARTICLE
We are searching for men who are carriers of the Fragile X premutation between the ages of 40 and 69 to participate in a longitudinal UC Davis research project, studying changes in brain and cognition associated with aging. For men who have not yet been tested, FMR1 DNA testing is completed to help determine eligibility for the study. » FULL ARTICLE
Do you have a daughter with fragile X syndrome? I am a graduate student in the Genetic Counseling Program at Brandeis University. I am seeking volunteers to participate in a qualitative research project. The goal of this study is to explore the experiences of parents of symptomatic girls with FMR1 full mutations who have discussed the diagnosis with their daughter. Only parents who have gone through this process, like you, have this knowledge. It... » FULL ARTICLE
We are studying an experimental medication for fragile X syndrome in children and adolescents. We hope to learn if this experimental medication is safe, how well your body manages the medication (tolerability), how much medication is in your body (pharmacokinetics) and if it works on symptoms of Fragile X Syndrome. Children and adolescents with Fragile X Syndrome (between 5 and 17 years of age) may participate. Costs for travel, accommodations, and food for clinic visits will be reimbursed. Please contact your local research center to receive more information or to ask any questions. A list... » FULL ARTICLE
The Carolina Institute for Developmental Disabilities (CIDD) at the University of North Carolina at Chapel Hill and the Center for Interdisciplinary Brain Sciences Research (CIBSR) at Stanford University would like to inform you of an exciting new research project involving families who have a child diagnosed with fragile X syndrome. This innovative, five-year, NIMH funded research project was awarded $5 million in funding in the fall of 2012 and the study is now underway. The... » FULL ARTICLE
We invite your child with fragile X syndrome to join in the foXtail study This invitation tells you a more about the study We want to see if a new medication, RO4917523, helps people with fragile X syndrome Have a talk about the foXtail study with your family/child’s caregivers and child It is your and your child’s choice if you want your child to take part in this study Please consider participating Please contact us if you want to ask questions Invitation We are inviting children ages 5 to 8 years old with fragile X syndrome... » FULL ARTICLE
We are actively recruiting children diagnosed with FXS between 24-60 months old to participate in a groundbreaking study! We have preliminary evidence that sertraline, a selective serotonin re-uptake inhibitor (SSRI) is beneficial for language development in young children with FXS. It is our hope that this study will lead to a better understanding of the benefits of early treatment with sertraline for young children with FXS and inform future treatment and interventions. Who can participate? Children between the ages of 24-60 months of age diagnosed... » FULL ARTICLE
The Kosair Charities Pediatric Clinical Research Unit at the University of Louisville invites adolescents between the ages of 14 and 17 with fragile X syndrome to take part in a research study for a medication that is being developed to treat the symptoms of fragile X syndrome. We hope to learn more about its safety, how well your body manages it (tolerability), and how much medicine is in your body (pharmacokinetics). This study is being sponsored (organized and paid for)... » FULL ARTICLE
The Waisman Center at the University of Wisconsin-Madison is recruiting boys with fragile X syndrome for a study on language development. Looking For Boys should be between 9 and 16 years of age, and speaking in at least 2-3 word phrases. Purpose The purpose of this research is to better understand how children with FXS use language. The Study Participants will complete standardized tests, a conversation language sample, as well as an autism diagnostic evaluation. Parents will complete an interview about their son’s behaviors and communication abilities. All... » FULL ARTICLE
HELP EXPLORE THE ETHICAL, LEGAL AND SOCIAL IMPLICATIONS OF NEW PRENATAL GENETIC TESTS WITH THE STANFORD UNIVERSITY FORUM ON BIOMEDICAL ETHICS The INVEST forum is designed to help people deliberate over the ethical, legal and social impact of emerging technologies. The opinions you express in this forum, a project of the Stanford Center for Integration of Research on Genetics and Ethics, will be used to make informed recommendations to technology developers, policy-makers and regulators. The current scenario deals with a new technology for... » FULL ARTICLE
Cognitive Training for Fragile X Syndrome We are conducting a study to determine if a computer-based and game-oriented training program will enhance the working memory skills of children and adolescents with fragile X syndrome (FXS). This paradigm is being tested as a potential intervention to improve certain cognitive and behavioral skills in FXS, such as paying attention and resisting distractions. Who can participate? Individuals with a confirmed diagnosis of FXS. Males and females, ages 8-17 years old. Where does the study take place? A qualified UC Davis staff... » FULL ARTICLE
The Center for Interdisciplinary Brain Sciences Research (CIBSR) at Stanford University would like to invite you to take part in an exciting new research opportunity available to families who have an adolescent child with fragile X syndrome. We are conducting a study to evaluate how environmental and biological factors affect the display of severe problem behavior (e.g., aggression, self-injury, etc.) in adolescents with fragile X syndrome. The lead investigator of this study is Scott Hall, PhD, BCBA-D. Eligible participants will travel to Stanford for a comprehensive behavioral assessment... » FULL ARTICLE
If you have a son or daughter with fragile X syndrome between 6 and 23 years of age, you and your child are invited to participate in a research study being conducted by Dr. Leonard Abbeduto at the MIND Institute University of California, Davis, and four other sites across the United States. The goal of this study is to learn more about how samples of spoken language can be used to measure change over time in the spoken language, problem solving, and... » FULL ARTICLE