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

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Dec 15, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called VX-670 to see how safe it is and how well it works for adults with Myotonic Dystrophy Type 1 (DM1). DM1 is a genetic condition that affects muscle control and strength. The researchers will give participants different doses of VX-670 and observe its effects to find out the best way to use it. The trial is currently recruiting participants aged 18 to 75 who have been diagnosed with DM1 and have a specific genetic marker related to the condition.

To join the study, participants need to have been diagnosed with DM1 at age one or older and have a genetic test showing a certain number of repeats in their DNA. Participants will be closely monitored during the trial, and they can expect regular check-ups to assess their health and how they respond to the medication. It’s important to note that individuals with certain medical conditions may not be eligible to participate. Overall, this study aims to gather important information about VX-670 that could help improve treatment options for people living with DM1.

Gender

ALL

Eligibility 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.