Recruiting: Mirum Launches Clinical Trial of MRM-3379 for Fragile X Syndrome
Mirum Pharmaceuticals has launched a Phase 2 clinical trial of MRM-3379, a selective PDE4D inhibitor, as a potential treatment for Fragile X syndrome. The study is now open for males ages 13 to 45 with Fragile X syndrome.
The trial (ClinicalTrials.gov ID: NCT07209462) will evaluate the safety, tolerability, and early efficacy signals of MRM-3379, focusing on improvements in cognitive function, behavior, and daily living. This marks an important step forward in developing targeted treatments for Fragile X syndrome.
It’s incredibly encouraging to see a company like Mirum step into the Fragile X space with a potential therapy that holds real promise. This study is actively enrolling, and we encourage eligible families to consider participation in this important research.
Michael Tranfaglia, MD
Medical Director and Co-Founder
FRAXA Research Foundation
A Promising Path Toward Advancing PDE4D Therapies in Fragile X
MRM-3379 targets the phosphodiesterase type 4D (PDE4D) enzyme. This approach builds on years of science, much of it spearheaded and funded by FRAXA, which identified PDE4D inhibitors as a promising treatment strategy based on their ability to restore signaling pathways disrupted in Fragile X.
FRAXA-funded studies helped support the development of Shionogi’s zatolmilast, another PDE4D inhibitor that showed cognitive improvement in a Phase 2 trial and is currently in Phase 3 development.
Mirum’s MRM-3379 potentially offers a new generation of PDE4D-targeting therapy with differentiated pharmacological properties.
Understanding the MRM-3379 Trial and How to Take Part
The clinical trial will run for 12 weeks, during which males ages 16 to 45 receive MRM-3379 or placebo. In addition, males ages 13 to <16 can receive open-label MRM-3379. Sites in California and Pennsylvania are recruiting now and additional sites (including Boston and Cincinnati) will be coming on line soon.
For complete eligibility details and the full list of recruiting sites, see the MRM-3379 entry on ClinicalTrials.gov.
Enrollment Information
Southwest Autism Research & Resource Center
Phoenix, Arizona
Riley Stark
(480) 351-0567
stark@autismcenter.org
Amnova Clinical Research, LLC
Irvine, California
Maggie Wang
(949) 358-7447
maggie@amnovaresearch.com
Rush University Medical Center-Clinical Operation
Chicago, Illinois
Lorelai Konen
(312) 942-2815
Lorelai_Konen@rush.edu
Suburban Research Associates
Media, Pennsylvania
Alex Hollis
(610) 891-9024
ahollis@suburbanresearch.com
Children's Hospital of Orange County - Pediatric Subspecialty
Not Yet Recruiting
Orange, California
Sailaja Golla
UC Davis Mind Institute
Not Yet Recruiting
Sacramento, California
Melissa Heys
(916) 703-0301
meheys@health.ucdavis.edu
Children's Hospital Colorado
Not Yet Recruiting
Colorado Springs, Colorado
Nicole Tartaglia
(720) 777-6895
Nicole.Tartaglia@childrenscolorado.org
Neurology
Not Yet Recruiting
Miami, Florida
Dr. Barbara Coffey
(305) 243-2759
bjc134@miami.edu
Emory University - The Emory Clinic - Clifton Road
Not Yet Recruiting
Atlanta, Georgia
Robin Vinson
(404) 778-8533
robin.vinson@emory.edu
Kennedy Krieger Institute, John Hopkins School of Medicine
Not Yet Recruiting
Baltimore, Maryland
Kennedy Krieger
(443) 923-3850
ResearchTrials@KennedyKrieger.org
Boston Children's Hospital
Not Yet Recruiting
Boston, Massachusetts
Michelle Coughlin
(857) 218-3842
michelle.Coughlin@childrens.harvard.edu
UMass Memorial Health
Not Yet Recruiting
Worcester, Massachusetts
Taylor Merk
(774) 455-4119
Taylor.merk@umassmed.edu
University of Minnesota Department of Medicine
Not Yet Recruiting
Minneapolis, Minnesota
Rachel Floersch
(612) 626-8424
Floer013@umn.edu
Cincinnati Children's Hospital Medical Center
Not Yet Recruiting
Cincinnati, Ohio
Hannah Sachs
(513) 803-1150
Hannah.Sachs@cchmc.org
Intermountain Healthcare - Park City Specialty Clinic
Not Yet Recruiting
Salt Lake City, Utah
Peter Farr
(801) 587-2103
peter.farrell@hsc.utah.edu
Why Advancing Multiple Treatment Options Is Critical in Fragile X
The launch of this trial is a major milestone for the Fragile X community. MRM-3379 adds to a growing pipeline of therapies that aim to address the root biological mechanisms of Fragile X, not just the symptoms. No single medicine will be right for everyone, so it is important to have multiple drugs in development. FRAXA is proud to see our early investments in Fragile X research helping drive PDE4D discoveries into clinical trials. As more biotech companies like Mirum enter this space, we move closer to real-world treatments.
As with all trials, progress depends on participation. To stay up to date on this and other clinical trials, visit our Clinical Trials Hub or reach out to our team at info@fraxa.org.