Family member affected by Fragile X?

Further information about this study

Fragile X Syndrome (FXS) is a rare genetic condition that can affect learning, communication, behavior, and everyday life. There are currently no approved medicines specifically designed to treat FXS.

This clinical study is evaluating an investigational medicine for males living with Fragile X Syndrome. The study will look at how safe and well-tolerated the investigational medicine is, and will explore whether it may help improve some of the challenges associated with FXS. By participating in this study, individuals with FXS and their families can help researchers better understand this condition and support the development of potential future treatment options.

Participation includes study visits, health assessments, and ongoing monitoring by the study team. A caregiver will be involved throughout the study to help support participation and complete study-related questionnaires.

Might there be other criteria I need to fulfill?

The person you care for may be eligible if they:

• are male
• are 13–45 years of age
• have been diagnosed with Fragile X Syndrome
• meet the study requirements

There might be other eligibility criteria, and a member of the study team will explain all eligibility requirements during the screening process. For more information, please visit the BLOOM Study website here.

What will I receive if I qualify?

If the person you care for is qualified, they will receive:

• Study-related assessments, investigational treatment, and regular study visits with the healthcare professionals at no cost.
• Support from the study team throughout participation and opportunities to ask questions during the study.
• After screening, participants will be randomly assigned to receive either the investigational treatment or a placebo. Neither participants nor the study team will know which study treatment is assigned during the study.

Will I be compensated?

Your child, family member, or loved one will receive study-related medication and care at no cost. Further, study-related reimbursement may be available, including travel expenses, meals, and other costs.