Conference Presentation

Keynote: Clinical Trials in Fragile X Syndrome

01 h 18 m

This session will be a family-friendly joint presentation of results from recently completed, and a description of all currently active clinical trials and development programs for new medications in fragile X syndrome (FXS).

About the Webinar

Presented by Elizabeth Berry-Kravis, MD, PhD, Craig A. Erickson, MD, and Randi J. Hagerman, MD
Learn more about the presenters

Trials to be covered include acamprosate, single dose EEG-focused challenge studies, AFQ056 (FXLEARN), ergoloid mesylates/5HTP, metformin, cannabidiol, BAER-101, BPN 14770, sulindac and Anavex 2-73. A basic explanation of how each medication works and the results of trials previously conducted will be discussed. For each trial currently recruiting, requirements for participation, sites where patients can participate, and logistical information will be given, and presenters will answer any questions about trials.

Additional Resources

Fragile X and Puberty & Sexuality
Depending on their age, it’s necessary to approach teaching children about relationships, the difference between public and private, body changes, feelings and emotions, reproduction, STDs, birth control, and how to keep themselves safe. You may even find you need a refresher for yourself before approaching the subject with them.

About the Speakers

Elizabeth Berry-Kravis

Elizabeth Berry-Kravis, MD, PhD, established the Fragile X Clinic and Research Program at Rush University Medical Center in 1992. She studies Fragile X syndrome medical issues, epilepsy, and psychopharmacology and provides care to over 700 patients with FXS. She has been a leader in translational research, including the development of outcome measures and biomarkers, natural history studies, newborn screening, and particularly clinical trials of new targeted treatments.

Dr. Berry-Kravis’s laboratory studies the cellular roles of the Fragile X protein (FMRP), its relationship to phenotypes, and the optimization of genetic testing methods. She is a longstanding member of the NFXF Scientific and Clinical Advisory Committee, and Clinical Trials Committee, and is the principal investigator of the CDC-funded FORWARD-MARCH natural history project for Fragile X.

Dr. Berry-Kravis attended the University of Notre Dame for her undergraduate studies and the University of Chicago for her doctoral degrees (MD and PhD) and training in pediatric neurology.

Craig A. Erickson

Craig A. Erickson, MD, is a professor of Psychiatry at Cincinnati Children’s Hospital Medical Center and the University of Cincinnati College of Medicine-Affiliated. Dr. Erickson leads a neurodevelopmental clinical and research group focused on improving clinical care through research discovery. He is the director of the Cincinnati Fragile X Research and Treatment Center, one of the largest such programs in the world. He serves as the chair of the Clinical Trials Committee organized by the National Fragile X Foundation and is a leader in translational medicine efforts in Fragile X syndrome, autism, and related disorders. Additionally, he is the director of research in the Division of Psychiatry at Cincinnati Children’s Hospital. 

Randi J. Hagerman

Developmental pediatrician Randi J. Hagerman, MD, FAAP, is a highly regarded professional within the Fragile X community. She co-founded the National Fragile X Foundation in 1984 and served on the board for 25 years. In 2009, she decided it was time to step aside and let others bring their expertise to the board, though she continues to help guide the foundation to this day.

There is no aspect of the NFXF that Randi has not helped shape. Her strength as both a clinician and researcher has informed the NFXF mission and strategic plan. She and her husband, Dr. Paul Hagerman, generously support the NFXF mission. While Randi is no longer a board member, she is active on the NFXF Scientific & Clinical Advisory Committee and the Fragile X Clinical & Research Consortium where she represents the UC Davis Medical Center’s MIND Institute as medical director of the Fragile X Clinic.

Randi continues to be a sought-after speaker and her work has expanded from Fragile X syndrome to all Fragile X-associated disorders. Randi is continually on the go as an international spokesperson for Fragile X. Fortunately, she’s only seconds away by phone when we need to call upon her for her guidance, advice, and wisdom.

Hilary Rosselot

Hilary joined the NFXF team in 2019. Prior to joining the NFXF team, she worked at the Cincinnati Fragile X Research and Treatment Center for several years. She has experience as a clinical research coordinator across many types of clinical trials and served as the clinical research manager for the Cincinnati program. She earned a bachelor’s in psychology, an MBA, and was previously a SOCRA-certified clinical research professional (CCRP). She enjoys spending time with her family, including her young daughter and Boston Terrier, as well as curling up with a good book.