Opportunities for Families

Drug Studies

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.

Alternative Types of Studies

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.

Summary

At the NFXF, we post all opportunities for families that meet General Guidelines for posting established by our Scientific and Clinical Advisory Committee and by our Ethics Committee.

The NFXF also periodically assists clinics, by request, with recruitment through a variety of means including mailings, email blasts and Facebook postings. This is a fee-based service that was developed in consultation with the NFXF Ethics Committee and is designed to promote the advancement of research on behalf of the pharmaceutical companies while compensating the NFXF for the associated staff and materials expenses. 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.

Recent Opportunities & Clinical Trials

• Join the Fragile X Clinical & Research Consortium Registry and Database

  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

• Ongoing Clinical Trials for Fragile X

  The NFXF database for all current clinical trials for Fragile X-associated Disorders.

  . . . Read More: Ongoing Clinical Trials for Fragile X

• Online Survey: Usage of Tablet App Technology by Individuals with Fragile X Syndrome

  Has app technology made a qualitative difference for your affected family member? Please help us share valuable information about use of app technology by individuals with fragile X syndrome by completing the following survey developed by Laurie Yankowitz, EdD, and Vicki Sudhalter, PhD. Results will be presented at the upcoming International Fragile X Conference in July.   Usage of Tablet

  . . . Read More: Online Survey: Usage of Tablet App Technology by Individuals with Fragile X Syndrome

  Posted at May 24, 2012 | Categories : Opportunities for families | 5 Comments
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  Study Recruitment: When Do Seizures Occur in People with Epilepsy and Intellectual Disabilities?

  Researchers from the University of Cambridge (UK) are carrying out an online survey about situations that might increase or decrease the chances of children/adults with epilepsy and intellectual disability having a seizure. We hope that learning more about this will lead to improved seizure management for people with Fragile X and other syndromes with high rates of epilepsy and

  . . . Read More: Study Recruitment: When Do Seizures Occur in People with Epilepsy and Intellectual Disabilities?

  Posted at May 23, 2012 | Categories : Opportunities for families | 4 Comments
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  You Can Help Our Fragile X World

  Your answers will help give a better understanding of how fragile X syndrome affects families and children What job opportunities are available to young adults with fragile X syndrome? When do parents tell a daughter that she is a carrier of fragile X syndrome? How knowledgeable is your child’s doctor about fragile X syndrome? How do you plan for the long term care of adult children with fragile X

  . . . Read More: You Can Help Our Fragile X World

  Posted at April 30, 2012 | Categories : Fragile Xtras,Opportunities for families | 2 Comments
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  Investigational Drug Study for Children with Fragile X Syndrome – Baltimore

  Kennedy Krieger Institute is looking for children, ages 5 through 11 years, with fragile X syndrome to participate in a research study. The purpose of this study is to determine if an investigational drug, arbaclofen, is an effective treatment for social withdrawal symptoms associated with fragile X syndrome. This study will also assess whether arbaclofen is safe and tolerated by individuals with fragile X syndrome. Social

  . . . Read More: Investigational Drug Study for Children with Fragile X Syndrome – Baltimore

  Posted at March 4, 2012 | Categories : Opportunities for families | 0 Comment
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  Does Your Adolescent or Young Adult Have Fragile X Syndrome?

  New Stanford Treatment Study Focused on Fragile X Syndrome Adolescents and Young Adults with Fragile X Syndrome Age 12-29 Years Needed We are recruiting females and males between the ages of 12 and 29 years to participate in a newly funded medication study in fragile X syndrome. Participants will have the opportunity to receive the medication, donepezil, which may improve cognition and behavior in individuals with this important condition.

  . . . Read More: Does Your Adolescent or Young Adult Have Fragile X Syndrome?

  Posted at March 1, 2012 | Categories : Opportunities for families | 1 Comment

• Does Your Child Need Help to Improve His/Her Math Skills?

  Standford University Research Individuals with Fragile X Syndrome Age 9-23 Years Needed We are conducting a behavioral treatment study utilizing a fun, reward-based learning paradigm to maximize your child’s learning potential. Participation involves: Coming to Stanford for 3 days Cognitive testing MRI scan Intensive behavioral intervention Inclusion criteria for participation – child must be: Between 9-23 years of age Diagnosed with fragile X syndrome Does not experience seizures Right handed (or favor his/her right hand) Able to lie still for up to 10 min with instruction Benefits of

  . . . Read More: Does Your Child Need Help to Improve His/Her Math Skills?

  Posted at February 14, 2012 | Categories : Opportunities for families | 1 Comment

• Study of Fragile X Treatment Preferences

  RTI International{{1}}, an independent, nonprofit research organization, is conducting an important study. The purpose of this study is to better understand the views of people who provide care to a son or male with fragile X syndrome who is at least 5 years of age. The results of the study will be used to help make better decisions about developing medications to treat the most pressing needs of people with fragile X syndrome.{{2}} RTI

  . . . Read More: Study of Fragile X Treatment Preferences

  Posted at January 12, 2012 | Categories : Opportunities for families | 4 Comments

• Study recruitment: Parent Mediated Language Intervention for Young Children with FXS

  Young children with fragile X syndrome and their biological mothers are being recruited to participate in a language intervention study conducted by the research team of Dr. Leonard Abbeduto at the MIND Institute, University of California-Davis. Children should be between 2- and 6-years of age and have little spoken language. The project will teach mothers to interact with their children in ways that support communication and language development. Participation in the project may

  . . . Read More: Study recruitment: Parent Mediated Language Intervention for Young Children with FXS

  Posted at December 17, 2011 | Categories : Opportunities for families | 19 Comments

• Study Recruitment: Children & Adults with Fragile X Syndrome Nashville’s Fragile X Treatment Research Program, Vanderbilt University

  This announcement has been posted as a courtesy by the National Fragile X Foundation (NFXF) on behalf of Nashville's Fragile X Treatment Research Program at Vanderbilt University. The announcement and the parties conducting and sponsoring the research meet guidelines established by the NFXF regarding relevancy and appropriateness to Fragile X. The NFXF takes no position and makes no claims as to the potential benefits of the study including those studies involving medications, and cannot

  . . . Read More: Study Recruitment: Children & Adults with Fragile X Syndrome Nashville's Fragile X Treatment Research Program, Vanderbilt University

  Posted at October 13, 2011 | Categories : Opportunities for families | 0 Comment

• Research Study for Premutation Males Ages 18-50

  The University of California, Davis MIND Institute is currently recruiting men with the fragile X premutation between the ages of 18-50 to participate in a research study focusing on emotion and memory. Participation includes 2 full days at the MIND Institute Fragile X Research and Treatment Center in Sacramento, CA. Travel and lodging expenses will be covered and participants will receive $100 in compensation. For men who have not yet been tested, FMR1 DNA

  . . . Read More: Research Study for Premutation Males Ages 18-50

  Posted at April 25, 2011 | Categories : Opportunities for families | 2 Comments