First human test of potential muscle disease treatment
NCT ID NCT06204809
Summary
This early-stage study tested the safety of a new drug called PGN-EDODM1 in 24 adults with Myotonic Dystrophy Type 1 (DM1), a genetic muscle-wasting disease. The main goal was to see if single doses given through an IV were safe and well-tolerated. Researchers also measured how the drug moved through and was processed by the body over 16 weeks.
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Contacts and locations
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Locations
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CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec, Canada
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario, Canada
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Salford Royal Hospital
Salford, United Kingdom
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Stanford University
Palo Alto, California, 94304, United States
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UCI Center for Clinical Research
Irvine, California, 92697, United States
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University College London Hospital
London, UK, NW1 2PG, United Kingdom
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University of Calgary
Calgary, Alberta, T3M 1M4, Canada
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University of Kansas Medical Center
Fairway, Kansas, 66205, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Conditions
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