A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

• Status: Recruiting
• Trial end date: 2026-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated

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.