A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Phase 1/2 – combined early trial
Conditions:
Myotonic Dystrophy Type 1 (DM1)
Sponsor: Vertex Pharmaceuticals Incorporated
trial.available_in:
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Overview
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.
Who can participate
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.
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