The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
Myotonic Dystrophy Type 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
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Stanford Neuromuscular Research, San Carlos, California, United States, 94070
University of Florida Clinical Research Center, Gainesville, Florida, United States, 32608
University of Kansas Medical Center, Fairway, Kansas, United States, 66205
Boston Children's Hospital, Boston, Massachusetts, United States, 02115
Washington University School of Medicine / St. Louis Children's Hospital, Saint Louis, Missouri, United States, 63110
Wake Forest Baptist Health, Winston-Salem, North Carolina, United States, 27104
University of Pennsylvania, Philadelphia, Pennsylvania, United States, 19104
Virginia Commonwealth University (Sanger Hall), Richmond, Virginia, United States, 23298
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
18 Years to 64 Years
ALL
No
Vertex Pharmaceuticals Incorporated,
2026-12-31