A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1

Vertex Pharmaceuticals Incorporated

Summary

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.

Eligibility

Age range: 18–64 years
Sex: All
Healthy volunteers: No

Detailed criteria

Key Inclusion Criteria: \- Documented clinical diagnosis of DM1 with age of onset greater than (\>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100 Key Exclusion Criteria: \- History of any illness or any clinical condition as pre-specified in the protocol Other protocol defined Inclusion/Exclusion criteria may apply.

Interventions

Drug
VX-670
Solution for intravenous administration.
Drug
Placebo
Solution for intravenous administration.

Locations (26)

Stanford Neuromuscular Research
San Carlos, California
University of Florida Clinical Research Center
Gainesville, Florida
University of Kansas Medical Center
Fairway, Kansas
Boston Children's Hospital
Boston, Massachusetts
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri
Wake Forest Baptist Health
Winston-Salem, North Carolina