Opportunities for Families

Join the FORWARD 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 FORWARD Registry and Database.

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 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.

Recent Opportunities & Clinical Trials

If you are having trouble funding your trip to this clinic, ask the clinic coordinator about financial assistance through the Justin Silver Fly With Me Fund.
Page 1 of 512345