Age 12–45 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 12 to <18 years old and the other trial is for adult males 18-45 years.
About the Study
Who can participate?
Males ages 12–45 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, check out Tetra’s study flyer or 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 reimbursement is available for eligible families.
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 | Phone | |
|---|---|---|---|
| Rush University Medical Center (IL) | Abigail Ayemoba or Loren Escot | Abigail_Ayemoba@rush.edu Loren_Escot@rush.edu |
312-563-9495 |
| Cincinnati Children’s Hospital Medical Center (OH) | Ashley Tyree | ashley.tyree@CCHMC.org | 513-517-1550 |
| UC Davis Health System (CA) | Ellery Santos | ersantos@ucdavis.edu | 916-703-0200 |
| Children’s Hospital Colorado (CO) | Sailor Brukardt | sailor.brukardt@childrenscolorado.org | 920-810-9347 |
| Kennedy Krieger Institute (MD) | Kristen Judy | judy@kennedykrieger.org | 443-923-3826 |
| Icahn School of Medicine T Mount Sinai (NY) | Venus Fan | venus.fan@mssm.edu | 212-659-1456 |
| University of Utah and Primary Children’s Hospital | Carly Straley | carly.straley@hsc.utah.edu | 801-587-3136 |
| University of Massachusetts Chan Medical School (MA) | Lauren Venuti | lauren.venuti@umassmed.edu | 774-455-4113 |
| Suburban Research Associates (PA) | Megan Verano | mvarano@suburbanresearch.com | 610-891-9024 |
| Greenwood Genetic Center (SC) | Caleb Hinzman | chinzman@ggc.org | 864-672-6912 |
| CHOC Thompson Autism Center (CA) | April Soriano | April.soriano@choc.org | 714-509-8300 x 32683 |
| Emory University School of Medicine (GA) | Jean Luan McColl | jean.luan@emory.edu | 404-778-8619 |
Interested in Participating?
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