Vertex
Are You Eligible?The purpose of this study is to assess the long-term safety, tolerability and effectiveness of multiple doses of VX-670, an investigational drug, in people with Myotonic Dystrophy Type 1 (DM1) over a period of up to two years.
Research Objectives
Status
Recruitment
Total participants required
Study location
Study type
Patient group
About this clinical trial
This study will help determine how well VX-670 works and how safe it is for long-term use in individuals with DM1. DM1 is caused by a genetic mutation in the DMPK gene, where certain DNA sequences (CTG repeats) are abnormally increased. These extra repeats interfere with the production of essential proteins in cells, leading to the symptoms of DM1. VX-670 is designed to specifically target and bind to these CTG repeats in the DMPK gene, aiming to correct the underlying cause of the disease. If you meet the requirements and want to participate in the study, you will be enrolled into the study and you will be assigned to a study treatment dose corresponding with your dose in the previous (parent) study. You will remain in the study for approximately 2 years.
Trial Information –https://clinicaltrials.gov/study/NCT06926621
Eligibility
- Have previously participated in a VX-670 study
- Be willing and able to comply with scheduled visits, a treatment plan, study restrictions, safety tests, contraceptive guidelines and other study procedures
Lead investigator
- Dr Po Sheng Yang
Clinical trial coordinators
- Jacqui Langton
- Emma Craig
Technical title
Title - Scientific: An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type I
Register your interest
If you would like more information about this clinical trial, please complete the contact form below.