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

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.

The study involves 3 visits to the MIND Institute over 20 weeks as well as weekly video conferencing sessions in the home to learn strategies to help the child with language. Study visits include behavioral and language assessments, a medical history, physical exams, parent questionnaires, and blood and urine sampling.

Please contact the study coordinator Erika Bickel at or (916) 703-0281.

Worcester, MA

MRI Study in Children with Fragile X Syndrome or Down Syndrome - U Mass Medical Center

Bethesda, MD

PET and MRI Fragile X Study - National Institute of Mental Health

Researchers will measure the rate at which the brain makes proteins (protein synthesis) and will identify specific parts of the brain affected in Fragile X.  The study enrolls eligible young men with FXS, ages 18-24, from around the world.
Please call Inna Loutaev, +1 301 496 4707, or E-mail, or visit website.

Cincinnati, OH

Trial of acamprosate


Recent Trials and Studies Awaiting Results


Completed Clinical Trials With Results

Trofinetide 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. IGF-1 contributes to the growth of brain cells and the connections between them (synapses).
FDA has granted two special designations for trofinetide in Fragile X: Fast Track Designation and Orphan Drug Designation. Trofinetide has a different mechanism of action from other drugs that have been tested for Fragile X syndrome.


For all Fragile X medication trials visit

To learn how trials work, visit