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Also known as [14C]-MRM-3379, Low dose of MRM-3379, Middle Dose of MRM-3379
MRM-3379 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Arm 1: Low Dose of MRM-3379 | EXPERIMENTAL | Participants 16-45 years of age randomized to this arm will receive the low dose of MRM-3379 |
| Arm 2: Middle dose of MRM-3379 | EXPERIMENTAL | Participants 16-45 years of age randomized to this arm will receive the middle dose of MRM-3379 |
| Arm 3: High dose of MRM-3379 | EXPERIMENTAL | Participants 16-45 years of age randomized to this arm will receive the high dose of MRM-3379 |
| Arm 4 :Placebo | PLACEBO_COMPARATOR | Participants 16-45 years of age randomized to this arm will receive Placebo |
| Arm 5: Low dose of MRM-3379 Open-Label | EXPERIMENTAL | Participants 13 to \< 16 years of age will receive the low dose of MRM-3370 Open-Label |
| Name | Type | Description |
|---|---|---|
| Low dose of MRM-3379 | DRUG | Oral capsule |
| Middle Dose of MRM-3379 | DRUG | Oral capsule |
| High dose of MRM-3379 | DRUG | Oral capsule |
| Placebo | DRUG | Capsules matched to study drug without the active pharmaceutical ingredient |
| Low dose of MRM-3379 Open-Label | DRUG | Oral capsule |
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 mu...
MRM-3379 is an investigational small molecule being developed by Mirum Pharmaceuticals for Fragile X Syndrome, a rare genetic condition. It is currently in Phase 2 clinical development and has not been approved by any regulatory agency. The program is being studied in male participants aged 13 years and older.
MRM-3379 is being studied as a potential treatment for Fragile X Syndrome, a rare neurodevelopmental disorder. It is an investigational therapy in Phase 2 testing and is not approved for any use. The ongoing trial enrolls male participants aged 13 years and older in the United States and Spain.
MRM-3379 targets PDE4D, an enzyme. By acting on this phosphodiesterase target, the small molecule is designed to modulate signaling pathways relevant to Fragile X Syndrome. PDE4D inhibition is the mechanism under investigation in the Phase 2 BLOOM study of male participants with the condition.
MRM-3379 is being developed by Mirum Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker MIRM. Mirum is a rare disease focused biopharmaceutical company. The company is running the Phase 2 BLOOM trial of MRM-3379 in Fragile X Syndrome.
MRM-3379 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory authority. The single ongoing trial is currently recruiting participants, with no completed trials reported for the program to date.
MRM-3379 is being evaluated in one clinical trial, NCT07209462, titled Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM). The Phase 2 study is recruiting 60 male participants aged 13 years and older across the United States and Spain. It is randomized, double blind, and placebo controlled.
Yes, [14C]-MRM-3379 is an alternative name for MRM-3379, referring to the carbon-14 labeled form of the compound. Other labels used for study arms include Low dose of MRM-3379 and Middle Dose of MRM-3379. All refer to the same investigational PDE4D-targeting small molecule from Mirum Pharmaceuticals.