Cincinnati Children’s Hospital is conducting a research study to learn about a medication called AZD7325 to treat impairments associated with Fragile X syndrome (FXS). AZD7325 is not currently approved by the Food and Drug Administration.
We are asking you and other people with FXS to be in this research because AZD7325 may be helpful for treatment of FXS, but it needs to be tested to be sure. We hope to learn more about the medicine’s safety and if it works on symptoms of FXS.
About the AZD7325 Clinical Trial
Q: Who can participate?
Adults 18-50 years old who have been diagnosed with FXS may be eligible to participate.
Q: What will happen in the study?
If the adult with FXS qualifies and decides to be in this research study, they will come to Cincinnati Children’s for eight visits over the next 12-16 weeks. All participants complete three treatment phases that will include a two-week treatment with 5mg AZD7325, two weeks of 15mg AZD7325, and two weeks of placebo. There will be a two-week washout period between each treatment phase, meaning there will be no medication for two weeks. The order in which the treatment phases occur is randomized, which means neither you nor the researchers conducting this study will know what phase of treatment the participant is on. But, if there is an emergency, they are able to get this information.
The following is a list of some of the assessments, tests, and procedures that will happen during the study:
- Review of your child’s medical and psychiatric history (including medications).
- Developmental/cognitive testing.
- Blood samples will be drawn.
- Drug treatment will be taken during the research visit and then sent home for regular morning and evening dosing.
- Vital signs and EKG to measure heart rhythm.
- Watch a video with faces on a screen to track eye movement.
- Tasks that test thinking skills, memory, and concentration.
- A caregiver will be asked questions about behavior.
Q: What are the good things that can happen from this research?
We hope that this treatment will make participants feel better or help reduce symptoms, but we don’t know if it will. When we finish this research, we expect that we will know more about AZD7325 and FXS. This may help other people with FXS later on.
You may request to receive a report of Fragile X testing if you want that information. Reports of methylation and repeat counts will be signed by the principal investigator and sent out by mail to families.
Q: What are the bad things that can happen from this research?
AZD7325 side effects seen commonly with 15mg dose are dizziness, tiredness, excitement, nausea, and headache. Less commonly seen side effects are dry mouth, confusion, anxiety, and sleepiness.
- EEG: The saline solution used during EEG testing may irritate sensitive skin.
- Blood Draw: The blood draw may include some mild discomfort, hematoma, infection, bleeding, or fainting. Standard methods and precautions will be used to protect the puncture site from bleeding and infection.
- Psychological Stress: Some of the questions may cause the participant or caregiver to become uncomfortable.
- Other Risks: Every effort is made to keep information confidential; however, this cannot be guaranteed.
There may be other risks that we do not know about yet.
Q: Will I and/or my child be paid to be in this research study?
Participants receive $100 for time and travel for the first visit and $50 for each of the following 7 visits. Travel reimbursement may be available for eligible families.
Q: How can I participate?
Complete the form on this page to have your information sent to the study coordinator Stephanie Booker. She will be in contact with you as soon as possible.
Craig Erickson, MD
Cincinnati Children’s Hospital Medical Center
If you’d like to learn more or apply for this clinical trial, please fill out the form to send your contact information to the study coordinator.
Our Most Recent Opportunities
A Mount Sinai Genetic Counseling graduate student is conducting a research study to learn about possible benefits and side effects of CBD supplements when used to treat FXS symptoms. This information will be obtained by using an anonymous online parental and caregiver survey. About the Survey Who can participate? Parents and caregivers caring for somebody with FXS who uses, or has used, CBD supplements may be eligible to participate. The parent or caregiver responding to the survey must be at least 18 years old. The individual with »
Researchers at Teachers College at Columbia University are conducting a survey to learn about the impact that the COVID-19 pandemic has had on multiple aspects of the lives of people with Fragile X Syndrome. We want to see how the Fragile X community specifically was impacted so that we can have a more focused plan of action for helping these families. The overall goal of this survey is to distribute our findings to medical professionals so that they can see what gaps have been created in their practices from the pandemic.
Participants of this study will explore what information is considered most important to recently diagnosed fragile X premutation carriers. Open to premutation carriers 18 and over who were diagnosed sometime since January 2018.
Kaylynn Shuleski, a master’s in genetic counseling candidate, is conducting a research study to explore the concerns and challenges caregivers may face when planning long-term supports and living arrangements for their adult children with Fragile X syndrome (FXS). This information will be valuable for current and future families when considering long-term supports, as well as for healthcare professionals, counselors, and policy makers. About the Study Who can participate? Parents and caregivers of adults with full mutation »
Males between the ages of 18 and 30 years with a diagnosis of Fragile X syndrome (full mutation) may be eligible to participate.
The Research in Neurodevelopmental Disabilities Lab at the Waisman Center at the University of Wisconsin-Madison is looking for parents of male or female children 6 to 17 years old with Fragile X syndrome for an online survey.