To those of you who have family members with Fragile X, thank you for being interested in clinical trials! This is a challenging phase for all of us, and as exciting as it is, we know many of you have questions about what it means to be in a clinical trial.

Each study is supervised by a doctor that specializes in Fragile X. These trials are free, you do not have to tell your insurance company, and you can leave any time you want. Travel costs are usually covered.

The FDA requires two successful large-scale clinical trials before it will consider approving a new treatment. If studies are successful and the FDA approves new drugs, others with Fragile X can also have access to these medicines.

Clinical Trials currently recruiting patients

 Informational webinar by Dr. Alex Kolevzon, 10/15/2014

  PowerPoint Slides (download)

  • More about this trial

    The purpose of this study is to assess the safety and efficacy of two dose levels of NNZ-2566 when compared to placebo in adolescent and adult males (ages 14-40) who have been diagnosed with FXS.

    Neuren Brochure on Fragile X Syndrome

    Brochure to download and print

    NNZ-2566 has a novel mechanism of action compared with other drugs that have been tested for Fragile X syndrome. It is a modified version of a biologically active part of Insulin-like Growth Factor (IGF-1). IGF-1 is a protein that helps cells grow and respond to stress or damage. In the brain, IGF-1 contributes to the growth of brain cells and the connections between them (synapses).

    The biologically active part of IGF-1 is called glypromate or GPE and supports the function of brain cells in a different way than IGF-1.  GPE occurs naturally in the brain. NNZ-2566 is a version of GPE modified to make it available to take as an oral medication. NNZ-2566 may help improve brain function by enhancing functions normally performed by GPE and by increasing IGF-1 levels in the brain. NNZ-2566 has been shown in animal models of Fragile X to improve performance in tests of behavior and cognition.

    FDA has granted two special designations for Neuren’s development of NNZ-2566 in Fragile X: Fast Track Designation and Orphan Drug Designation.

    This trial will include 60 participants between the ages of 14-40. Taking part in this study involves 8 visits to the doctor’s office over 10 weeks. The study medication and all study-related evaluations will be provided at no cost. Some support for travel is available. 

  • Trial Sites

    Davis, California
    UC Davis MIND Institute
    Principal Investigator: Randi Hagerman, MD
    Contact: Lindsey Partington 916-703-0427 lindsey.partington@ucdmc.ucdavis.edu
    Atlanta, Georgia
    Emory University – Recruiting
    Principal Investigator: Jeannie Visootsak, MD
    Contact: Krista Charen    404-778-8479    kharkre@emory.edu<
    Chicago, Illinois
    Rush University Medical Center
    Principal Investigator: Elizabeth M Berry-Kravis, MD
    Contact: Jamie A Chin    (312) 942-0025  Jamie_A_Chin@rush.edu
    Worcester, Massachusetts
    University of Massachusetts Medical School
    Principal Investigator: Jean Frazier, MD
    Contact: Ann Foley    774-455-4103    ann.foley@umassmed.edu
    NYC, New York     (click for flyer)
    Mount Sinai School of Medicine
    Principal Investigator: Alexander Kolevzon, MD
    Contact: Hillary Rieger  212-241-2826   hillary.rieger@mssm.edu
    Cincinnati, Ohio
    Cincinnati Children’s Hospital Medical Center
    Principal Investigator: Craig Erickson, MD
    Contact: Bridget Crippen bridget.crippen@cchmc.org or Kaela O’Brien kaela.obrien@cchmc.org
    Lewisburg, Pennsylvania
    Geisinger Autism & Developmental Medicine Institute
    Principal Investigator: Thomas Challman, M.D.
    Contact: Margaret King, Ph.D. (570)-522-9438  mking1@geisinger.edu
    Media, Pennsylvania
    Suburban Research Associates
    Principal Investigator: Shivkumar Hatti, MD
    Contact: Nathan Blubaugh    610-891-9024 ext 103    nblubaugh@suburbanresearch.com
    or Contact: Nikki Thomas    610-891-7200    Nthomas@suburbanresearch.com
    Denver, Colorado
    Children’s Hospital Colorado
    Principal Investigator: Nicole Tartaglia, MD
    Contact: Nanastasia Welnick    720-777-8608   Nanastasia.Welnick@childrenscolorado.org

 

  • Clinical trial of MDX in ages 15-55 with Fragile X – sponsored by Alcobra Ltd.
    Alcobra, an emerging biopharmaceutical company based in Israel, is conducting a clinical trial of Metadoxine Extended Release (MDX) in adults and adolescents with Fragile X Syndrome. This Phase IIb trial is a multi-center, randomized, placebo-controlled study, conducted in the US and Israel.

FRAXA hosted an Informational webinar by Dr. Elizabeth Berry-Kravis, Professor of Biochemistry, Neurological Sciences and Pediatrics at Rush University Medical Center in Chicago, on 12/12/2014

  Slides (pdf)

  • More about this trial

    The study is supported by positive data collected from multiple earlier animal studies using metadoxine. Results from these studies demonstrated significant improvement in behavioral and cognitive outcomes in mice based on testing and performance of memory, learning, and social interaction.There are few individuals in the US with Fragile X; as well as few treatment options; that is why the FDA has granted “Orphan Drug” designation. It is possible that participants may not benefit from participation in this trial. The drug is experimental and subjects will be informed of all safety concerns prior to participating in the trial.
    Specific information about center locations and the protocol can be found at Clinicaltrials.gov at http://clinicaltrials.gov/ct2/show/NCT02126995 or by contacting Anthony Robinson, Director, Clinical Operations at ARobinson@alcobra-pharma.com
  • Trial Sites

    Phoenix, AZ – Southwest Autism Research & Resource Center
    Principal Investigator: Raun Melmed, MD
    Contact: Sharman Ober-Reynolds   602-218-8225    SOberReynolds@autismcenter.org
    Aurora, CA – Children’s Hospital of Colorado
    Principal Investigator: Nicole Tartaglia, MD
    Contact: Susan Howell   720-777-8361   Susan.howell@childrenscolorado.org
    Sacramento, CA – University of California Davis
    Principal Investigator: Randi Hagerman, MD
    Contact: Lindsey Partington  916-703-0471   lindsey.partington@ucdmc.ucdavis.edu
    Chicago, IL – Rush University Medical Center
    Principal Investigator: Elizabeth Berry-Kravis, MD
    Contact: Crystal Hervey   312-942-7250   Crystal_Hervey@Rush.edu
    Boston, MA – Children’s Hospital Boston
    Principal Investigator: Lisa Albers-Prock, MD
    Contact: Lauren Voelz at 617-355-2954 or Lauren.Voelz@childrens.harvard.edu
    Baltimore, MD – Kennedy Krieger Istitute
    Principal Investigator: Robert Findling, MD
    Contact: Rebecca Hinton  443-923-9326  Hinton@kennedykrieger.org
    Media, PA – Suburban Research Associates
    Principal Investigator: Shivkumar Hatti, MD
    Contact: Nikki Thomas   610-891-7200   nthomas@suburbanresearch.com
    Houston, TX – Baylor College Of Medicine/TX Children’s Hospital Fragile X Clinic
    Principal Investigator: Diane Treadwell-Deering, MD
    Contact: Chiemenam Amaechi   832-824-4275   cxamaech@texaschildrens.org
    Seattle, WA – Seattle Children’s Hospital
    Principal Investigator: Mark Stein, PhD
    Contact: Libby Bliss   206-884-1488  elizabeth.bliss@seattlechildrens.org
  • Details

    The study enrolls eligible young men with FXS, ages 18-24, from around the world, and includes:
    · One visit, lasting several days, to the NIH Clinical Center, in Bethesda, Maryland.
    · Assessment by physicians
    · Blood draw, Two brain scans (PET & MRI), possible sedation
    · Travel, food, and lodging are covered and include the patient and one or two accompanying family members.
    · Compensation is paid for your time and assistance.Please consider enrolling your child in our clinical research study of FXS. If you would like to participate, be on our mailing list, be contacted in case of future studies, or would simply like to have more information: Please call Inna Loutaev, +1 301 496 4707, or E-mail loutaev.inna@nih.gov, or visit our website: http://patientinfo.nimh.nih.gov/Department of Health & Human Services, National Institutes of Health, National Institute of Mental Health, www.clinicaltrials.gov 06-M-0214

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Completed Clinical Trials funded by FRAXA

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For all Fragile X medication trials visit http://www.clinicaltrials.gov/ct2/results?term=fragile+x

To learn how trials work, visit http://clinicaltrials.gov/ct2/info/understand