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! 💪💚

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