Exploring an Investigational Medicine for Fragile X Syndrome
For many families affected by Fragile X syndrome, everyday moments can come with challenges that others may not always see or understand. Research is one way to learn more about Fragile X syndrome and explore potential future options for the community.
Mirum Pharmaceuticals is currently enrolling participants in the BLOOM clinical study, a Phase 2 study for Fragile X syndrome. The study is evaluating MRM-3379, an investigational medication, in males ages 13 to 45 who have been diagnosed with Fragile X syndrome.
MRM-3379 is an investigational medication being studied to better understand its safety and tolerability and whether it may help manage certain symptoms associated with Fragile X syndrome. An investigational medication means that it has not been approved by the U.S. Food and Drug Administration for Fragile X syndrome, and its safety and effectiveness are still being evaluated in clinical research.
Who May Be Eligible to Enroll?
The BLOOM study is enrolling males ages 13 to 45 who have been diagnosed with Fragile X syndrome. Additional study requirements and exclusions apply. A study representative will discuss these with families during the screening process.
Families who are interested in learning more should talk with their healthcare provider about whether participation may be appropriate.
The BLOOM study includes a brief screening period, followed by 12 weeks of treatment and a follow-up period. The full study experience lasts about 16 weeks.
What Does Participation Involve?
During the study, participants and caregivers can expect a combination of in-person visits and phone check-ins. The study includes four in-person visits with the participant and caregiver, along with four phone check-ins with the caregiver. During study visits, the clinical study team will ask general questions about the participant’s health and perform study-related tests to monitor overall health, side effects, and changes in quality of life.
Participants will receive either MRM-3379 or a placebo, depending on age and study assignment. A placebo is a capsule with no active ingredient. The study medication or placebo is taken by mouth twice a day.
Throughout the study, a dedicated medical team will monitor participants’ health, and support will be provided along the way.
Support for Study-Related Travel
Mirum is committed to helping make participation as supportive as possible. Reasonable study-related travel may be covered for the participant and caregiver, including ground transportation, air travel, and accommodations. If travel is required, related reasonable expenses, including childcare, may be reimbursed as permitted by study site policies. Study representatives can provide more details.
Participation in any clinical study is voluntary. Families should ask questions, review the study requirements carefully, and discuss the opportunity with their healthcare provider and the study team.
