Clinical Trials in Fragile X Syndrome

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 and Studies recruiting patients

Nashville, TN

Phase I Trial of Gaboxadol

Looking for children ages 13-17 who have been clinically and molecularly diagnosed with Fragile X Syndrome or Angelman Syndrome. There will be two clinic visits at Vanderbilt’s Children’s Hospital. This trial is sponsored by Ovid Therapeutics.  View/download flyer.  

Quebec, Canada

Combining Lovastatin and Minocycline

This is a pilot randomized Phase II trial, exploring the tolerability and the synergistic effect of a combined therapy. They will combine minocycline, which is often used to treat acne, and lovastatin, which is used to lower cholesterol. Both drugs target specific alterations in the brain of fragile X patients that would potentially have a combined powerful effect on their behavior. If you would like further information regarding the trial, which is accepting participants, please email

You can also view information at or 


Sacramento, CA

Combining Lovastatin and a Parent-Implemented Language Intervention in a Multimodal Treatment for Fragile X Syndrome - UC Davis MIND Institute

The study is testing the efficacy of a multi-modal treatment comprised of the medication lovastatin or placebo, and a Parent-Implemented Language Intervention (PILI) in children and adolescents with FXS between the ages of 10 and 17.  Please contact the study coordinator Erika Bickel at or (916) 703-0281.


Cincinnati, OH

Trial of acamprosate


Recent Studies Awaiting Results


Completed Clinical Trials With Results

  • 2/2016 - Double Blind Crossover Trial of Ganaxolone - Investigators: Frank Kooy and Anke Van Dijck- Antwerp, Belgium, sponsored by FRAXA Research Foundation. Dr. Randi Hagerman conducted a part of this trial in Davis, CA, sponsored by Marinus Pharmaceuticals.

    "Consistent with the mechanism of action and preclinical studies, anxious FXS patients treated with ganaxolone showed clinically relevant reductions in anxiety across several scales and subscales sensitive to measuring changes in anxious behavior. These results are encouraging and suggest that ganaxolone may improve anxiety in children with FXS, which could potentially allow them, as well as their families, to experience a more stabilized lifestyle." - Frank Kooy, PhD.

  • 12/2015 - Neuren’s trofinetide showed some success in small Phase 2 clinical trials in Fragile X syndrome and in Rett Syndrome
  • Alcobra's metadoxine trial has completed. Learn more at
  • 2012-13:  Open-Label Pilot Trial of Lovastatin - Francois Corbin, MD, PhD — Sherbrooke, Canada
  • 2008:  Minocycline trial - Carlo Paribello, MD - Toronto, Canada.   Positive results were published
  • a pilot lithium trial — Elizabeth Berry-Kravis, MD, PhD, and funded by FRAXA.  Positive results were published
  • pilot trial of CX516 —  Elizabeth Berry-Kravis, and funded by FRAXA.
  • Phase III trials of arbaclofen were completed by Seaside Therapeutics but results were disappointing in both Fragile X and autism. However, baclofen is an available drug.


For all Fragile X medication trials visit

To learn how trials work, visit