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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.
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 FXCRC Registry and Database.
. . . Read More: Fragile X Clinical & Research Consortium
Got Fragile X? Participate in a New Clinical Trial for Children with Fragile X Syndrome! Kennedy Krieger Institute is looking for children, ages 5 through 13 years, with fragile X syndrome to participate in a research study. Purpose The purpose of this study is to determine if an investigational drug is a safe and effective treatment for symptoms associated with fragile X syndrome. Procedures
Sertraline Study - Clinical Trials 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 reuptake inhibitor (SSRI) is beneficial for langauge development in young children with FXS. It is our hope that this study will lead to a
You can help us discover more about fragile X syndrome and the FMR1 premutation! Our research team at Northwestern University is studying language and social characteristics related to the gene involved in fragile X syndrome, FMR1. We are looking at very subtle characteristics among carriers of the FMR1 premutation to help us understand more clearly how
. . . Read More: Research: Did You Grow Up in Iowa, or Know Someone Who Did?
The Carolina Institute for Developmental Disabilities (CIDD) at the University of North Carolina at Chapel Hill would like to inform you of an exciting new research opportunity available to families with infants who have a child diagnosed with fragile X. This multi-center study involves the collaboration of two sites which are part of an
Fragile X Spectrum as a Model to Explore Neurogenetic Mechanisms of Cognitive Dysfunction Funded by the National Institutes of Health Approved by the University of California, Davis Institutional Review Board (#221039-3) Researchers at the UC Davis MIND Institute (Tony J. Simon, PhD, Principal Investigator and Susan M. Rivera, PhD, Co-Principal Investigator) invite you to consider participating in a research
Do you have fragile X syndrome? Are you the parent or caregiver of a person with fragile X syndrome, or know someone who is? The Kosair Charities Pediatric Clinical Research Unit at the University of Louisville invites adolescents and adults with Fragile X Syndrome to take part in a research study for a medication that
. . . Read More: Research: Study for Medication at University of Louisville
107 Chesley Drive, Unit 4 Media,PA19063 (610) 891-9024 (610) 891-9699 fax www.suburbanresearch.com Dear Parent or Caregiver, We are currently seeking volunteers who have fragile X syndrome to take part in a research study. The purpose of the study is to investigate how a new investigational medicine called RO4917523 works in treating people with fragile X syndrome. It is possible that your son or daughter may be suitable to
. . . Read More: Suburban Research Associates: Investigational Medicine RO4917523
Dear Friend of National Fragile X Foundation: We would like to inform you of clinical research studies to find out if a new investigational drug is safe and has beneficial effects in people who have fragile X syndrome. This investigational drug is not yet available on the market, because it has not been approved in the United States by the Food and Drug Administration (FDA), or any
. . . Read More: Opportunity from Novartis Pharmaceuticals
Seaside Therapeutics is studying the science of the brain and working to deliver treatments that improve the underlying causes of developmental disorders like fragile X syndrome. Right now, Seaside is testing an investigational medicine called STX209 (arbaclofen) for individuals with fragile X syndrome with social impairment. Thanks to the dedication and efforts of families, one of the clinical trials is almost complete.
. . . Read More: Seaside Therapeutics Harbor-C Trials
Teaching parents how to use an autism-based intervention with their child at home Funded by the National Fragile X Foundation Researchers at the UC Davis MIND Institute (David Hessl, Ph.D., Principal Investigator and Laurie A. Vismara, Ph.D., Co-Principal Investigator) are testing an autism-specific, behavioral intervention model with families of young children with fragile X syndrome. Parents will learn
. . . Read More: Helping Young Children With Fragile X At Home