Age 9–45 males with FXS with a molecular genetic confirmation FMR1 >200 CGG repetitions needed for clinical trials of new drug zatolmilast.

Tetra Therapeutics 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, Tetra 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.

About the Study

Who can participate?

Males ages 9–45 may be eligible to participate with a molecular genetic confirmation of FXS: FMR1 >200 CGG repetitions.

What will happen in the study?

If the individual qualifies and decides to be in this research study, they will conduct eight visits (at clinic and at home) over 13 weeks.

The following is a list of some of the study procedures that will happen during the study:

  • Participant cognitive and behavior testing
  • Blood draws
  • Parent/caregiver questionnaires

For more information, visit Tetra’s website.

What are the good things that can happen from this research?

Potential benefits are improvement in cognitive (language and vocabulary) function and improvement in daily activities/behavior.

What are the bad things that can happen from this research?

Potential drug side effects include mild nausea and diarrhea.

There may be other risks that we do not know about yet.

Will I or my child be paid to complete this study?

Participants receive $50 per visit (6).

Travel can be coordinated and paid for by Tetra or reimbursed for eligible families, even if there is not a site nearby. 

Who can I contact for more information about this study?

There are several sites across the U.S. Complete the contact form below to notify the site nearest you!

Site Coordinator Email Phone
Rush University Medical Center (IL) Abigail Ayemoba abigail_ayemoba@rush.edu 312-942-2815
Cincinnati Children’s Hospital Medical Center (OH) Jessie Michael jessica.michael@cchmc.org 513-517-1550
UC Davis Health System (CA) Abigail Higareda Borbe amoradelhigareda@ucdavis.edu 916-703-0281
Children’s Hospital Colorado (CO) Erika Peterson erika.peterson@childrenscolorado.org 720-777-8590
Kennedy Krieger Institute (MD) Maria Halaguena halaguena@kennedykrieger.org 443-923-3826
Icahn School of Medicine at Mount Sinai (NY) Venus Fan venus.fan@mssm.edu 929-989-7016
University of Utah and Primary Children’s Hospital (UT) Hina Yazdani hina.yazdani@hsc.utah.edu 801-581-7234
University of Massachusetts Medical School (MA) Taylor Merk taylor.merk@umassmed.edu 774-455-4119
Suburban Research Associates (PA) Augusto Sanchez asanchez@suburbanresearch.com 610-891-9024 x 1112
Greenwood Genetic Center (SC) Nicole Johnston njohnston@ggc.org 864-672-6896
CHOC Thompson Autism Center (CA) Mai Ngo pngo@choc.org 714-509-8300 x 32683
Emory University School of Medicine (GA) Jean Luan McColl jean.luan@emory.edu 404-778-8619
University of Kansas Medical Center (KS) Jasli Bonilla jbonilla@kumc.edu 913-945-9790
Amnova Clinical Research (CA) Maggie Wang maggie@amnovaresearch.com 323-348-0956
Clinic for Special Children (PA) Joelle Williamson jwilliamson@clinicforspecialchildren.org 717-687-9407

Interested in Participating?

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