NCT07209462 Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
| NCT ID | NCT07209462 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Mirum Pharmaceuticals, Inc. |
| Condition | Fragile X Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 60 participants |
| Start Date | 2025-11-22 |
| Primary Completion | 2027-10 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 60 participants in total. It began in 2025-11-22 with a primary completion date of 2027-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This study is a multicenter, double-blind, randomized, placebo-controlled study to assess the safety and tolerability of 3 doses of MRM-3379 in male participants with Fragile X Syndrome ages 16 to 45 (inclusive). In addition, a parallel cohort of participants ages 13 to \<16 will receive open-label MRM-3379. All participants will participate for 12 weeks of treatment. The study is also intended as a proof-of-concept investigation to evaluate whether MRM-3379 can improve FXS symptoms
Eligibility Criteria
Inclusion Criteria: * Willing and able to provide signed informed consent/assent. Where local regulations permit inclusion of participants deemed unable to provide informed consent, a legally authorized representative must provide informed consent on the participant's behalf, and the participant must provide assent if applicable. * Male, 13-45 years of age (inclusive) * Weight ≥30 kg and BMI between 18 and 36 kg/m2 (inclusive) at the screening visit * Diagnosis of FXS with a molecular genetic ≥200 CGG repetitions . * Able to perform the PVT and ORRT of the NIH-TCB * Have a consistent caregiver(s) who is willing and able to be present regularly and reliably with the participant * Able to swallow tablets or capsules Exclusion Criteria: History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation
Frequently Asked Questions
Who can join the NCT07209462 clinical trial?
This trial is open to male participants only, aged 13 Years or older, up to 45 Years, studying Fragile X Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07209462 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT07209462 currently recruiting?
Yes, NCT07209462 is actively recruiting participants. Visit ClinicalTrials.gov or contact Mirum Pharmaceuticals, Inc. to inquire about joining.
Where is the NCT07209462 trial being conducted?
This trial is being conducted at Phoenix, United States, Irvine, United States, Orange, United States, Sacramento, United States and 11 additional locations.
Who is sponsoring the NCT07209462 clinical trial?
NCT07209462 is sponsored by Mirum Pharmaceuticals, Inc.. The trial plans to enroll 60 participants.