Clinical trials for males aged 9–45 living with FXS with a molecular genetic confirmation FMR1 >200 CGG repetitions for new drug zatolmilast.
You may have heard about EXPERIENCE (Evaluation of Fragile X Experience in Cognition Expression) clinical trials as the Tetra studies or the studies of BPN14770 in Fragile X syndrome.
The EXPERIENCE clinical studies are now being managed by Shionogi (the company that acquired Tetra Therapeutics Inc. in 2020). Shionogi is conducting two randomized, placebo-controlled studies of BPN14770 (zatolmilast) to learn about the experimental drug’s safety and efficacy. Both trials will be followed by an open label extension study. If results are positive, Shionogi will be seeking approval of the drug with the FDA.
One trial is for adolescent males ages 9 to <18 years old and the other trial is for adult males 18-45 years.
Exciting update from Shionogi on the progress of the EXPERIENCE clinical trials (also known as BPN14770-CNS-204, BPN14770-CNS-301, BPN14770-CNS-302, and the Tetra studies):
“As a result of an unprecedented surge of interest by this incredible community, enrollment for the adult study in Fragile X syndrome (EXPERIENCE-301) is nearly at capacity and screening will close [March 21, 2025]. Clinical trial sites are working with Shionogi to accommodate previously scheduled appointments for potential new study participants. Notably, clinical trial sites are no longer able to accommodate new appointments as the clinical trial cannot recruit far beyond the planned 150 individuals without putting the protocol integrity of the trial at risk and exhausting the total clinical trial drug supply.
The adolescent study in Fragile X syndrome (EXPERIENCE-204) is in its final phase of enrollment. All scheduled screening appointments will proceed as planned; however, no additional screening appointments are available. On behalf of our team and our partners at Shionogi, we are grateful for the support of the community in both raising awareness of and participating in these studies.
Please note the open-label study (EXPERIENCE-302) is ongoing for individuals who have completed EXPERIENCE-301 or 204. W e look forward to sharing the results from these studies.”
Congratulations to the Fragile X community for participating in these trials and getting us one step closer to a potential treatment for Fragile X! 💪💚
Our Most Recent Opportunities
Study: Gathering Evidence to Optimize Decisions (GEODE)
The Center for Autism Research Excellence and the Center for Systems Neuroscience at Boston University are conducting a research study to explore decision-making and sensory perception in adolescents.
Single-Dose Study for Adult Men with FXS
Cincinnati Children's Hospital Medical Center is looking for males ages 18-40 with FXS to participate in a single-dose clinical trial that is studying a drug called Gaboxadol.
Study: Somatic symptom development in 6-12 year old females with an FMR1 mutation (SoS Study)
Researchers at the New York State Institute for Basic Research are conducting a study to better understand the development of physical (somatic) symptoms in females living with and without an FMR1 gene variation.
FXS TECH Study
Researchers at Rush University Medical Center are working on technology to improve how to identify and track progress in children living with autism and Fragile X syndrome. The study is currently recruiting children ages 18 months to 5 years, and 12-18 years.
Study: Web Intervention for Parents of Youth with Genetic Syndromes (WINGS)
Researchers at the Autism Assessment, Research, Treatment & Services (AARTS) Center at Rush University Medical Center are currently conducting a fully-virtual research study that is testing two telehealth interventions that are designed to help parents of children with genetic syndromes and intellectual disabilities gain strategies to manage challenging behaviors.
Neural Underpinnings of the Relationship Between Cognition and Gait Dysfunction in Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS)
Movement disorders researchers at Rush University Medical Center are conducting a research study to learn about brain activation in people living with FXTAS during tasks like walking and thinking. This study is currently recruiting adults ages 50+ who are living with FXTAS.