A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
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
44 participants
Feb 20, 2024
INTERVENTIONAL
Conditions
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
Inclusion Criteria1
- \- 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
Exclusion Criteria1
- \- History of any illness or any clinical condition as pre-specified in the protocol
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Interventions
Solution for intravenous administration.
Solution for intravenous administration.
Locations(26)
View Full Details on ClinicalTrials.gov
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NCT06185764