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, check out Tetra’s 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 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 | 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) | Adoley Allotey | aallotey@suburbanresearch.com | 610-891-9024 x 1112 |
| Greenwood Genetic Center (SC) | Nicole Johnston | njohnston@ggc.org | 864-672-6896 |
| CHOC Thompson Autism Center (CA) | Elyssa Smith | elyssa.smith@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) | Karlla Brigatti | kbrigatti@clinicforspecialchildren.org | 717-687-9407 |
Interested in Participating?
Our Most Recent Opportunities
Study: Recording the Experiences of Black and Hispanic Mothers who have Children with Fragile X Syndrome
Are you a Black or Hispanic woman raising a child with Fragile X syndrome? Researchers want to hear about your experiences. The South Carolina Family Experiences Lab is conducting 30-minute interviews to learn about the experiences of Black and Hispanic women raising a child with Fragile X syndrome. About the Study Who can participate? Black and Hispanic women of any age, who have a child with Fragile X syndrome, may [...]
Survey: What do you think about at-home research visits?
Dr. Schmitt and the Cincinnati Fragile X Center are conducting a survey to learn about potential barriers and advantages of conducting research visits in the home.
Study: Single Dose Study for Adult Women and Men with FXS
Dr. Craig Erickson at Cincinnati Children’s Hospital Medical Center is conducting a clinical trial to learn about if medication can change the brain’s response to sound, which may be abnormal in FXS.
Project WellCAST: Caregivers of Children with Fragile X Needed for a Research Study
The Kelleher Lab at Purdue University is conducting an NIH-funded clinical trial called Project WellCAST. Project WellCAST aims to understand how to best support caregivers of children with rare neurogenetic conditions, including Fragile X syndrome.
Study: Language, executive function, and quality of life of those with the Fragile X premutation
Researchers at the Waisman Center at UW-Madison are recruiting adults with the Fragile X premutation for a study on language, executive function, and quality of life.
Views and Experiences of Caregiver Coaching in Early Childhood Speech-Language Services: A Survey of Caregivers and Speech-Language Pathologists
The Research in Developmental Disabilities and Language Lab at The University of Wisconsin-Madison is conducting a survey to learn about you and your child’s experiences in speech therapy during early childhood.