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

Trial ID: NCT06185764

Interventional - Phase 1/2 - Active, not recruiting

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.

Conditions

• Myotonic Dystrophy Type 1 (DM1)

Interventions

• VX-670
• Placebo

Eligibility

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.

Sponsor

Vertex Pharmaceuticals Incorporated

Source: ClinicalTrials.gov

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