Evaluating VX-670 for treating Myotonic Dystrophy Type 1 in adults
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
This study is testing a new drug called VX-670 to see if it can safely help adults with Myotonic Dystrophy Type 1 feel better.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 44 (estimated) |
| Ages | 18 Years to 64 Years |
| Sex | All |
| Sponsor | Vertex Pharmaceuticals Incorporated Industry-sponsored |
| Locations | 26 sites (San Carlos, California and 25 other locations) |
| Trial ID | NCT06185764 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 in adult participants diagnosed with Myotonic Dystrophy Type 1 (DM1). Participants will receive either VX-670 or a placebo in varying doses to determine the drug's effects. The study will involve both Phase 1 and Phase 2 evaluations to gather comprehensive data on the drug's performance and safety profile. The research is crucial for understanding how VX-670 may improve outcomes for individuals with this condition.
Who should consider this trial
Good fit: Ideal candidates for this study are adults with a documented clinical diagnosis of DM1 and a genetic test confirming the presence of a CTG repeat of at least 100.
Not a fit: Patients with a history of other significant illnesses or clinical conditions as outlined in the protocol may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option that significantly improves the quality of life for patients with Myotonic Dystrophy Type 1.
How similar studies have performed: While there have been studies on Myotonic Dystrophy, the specific approach of using VX-670 is novel and has not been extensively tested in this context.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
San Carlos, California and 25 other locations
- Stanford Neuromuscular Research — San Carlos, California, United States (Recruiting)
- University of Florida Clinical Research Center — Gainesville, Florida, United States (Recruiting)
- University of Kansas Medical Center — Fairway, Kansas, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- Washington University School of Medicine / St. Louis Children's Hospital — St Louis, Missouri, United States (Recruiting)
- Wake Forest Baptist Health — Winston-Salem, North Carolina, United States (Recruiting)
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (Recruiting)
- Virginia Commonwealth University (Sanger Hall) — Richmond, Virginia, United States (Recruiting)
- Wesley Research Institute — Auchenflower, Australia (Recruiting)
- Neuroscience Clinical Trials Unit, Alfred Brain — Melbourne, Australia (Recruiting)
- Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg — Leuven, Belgium (Recruiting)
- Hopital de Chicoutimi — Chicoutimi, Canada (Recruiting)
- Altasciences Montreal — Montreal, Canada (Recruiting)
- McGIll University — Montreal, Canada (Recruiting)
- University of Ottawa — Ottawa, Canada (Recruiting)
- CHU Research Centre of Quebec — Québec, Canada (Recruiting)
- Neuromuscular Reference Center Institute of Myology — Paris, France (Recruiting)
- Ludwig Maximilians Universitaet Muenchen — München, Germany (Recruiting)
- Centro Clinico NeMO — Milan, Italy (Recruiting)
- Maastricht University Medical Center — Maastricht, Netherlands (Recruiting)
- Hospital Universitario y Politécnico La Fe — Valencia, Spain (Recruiting)
- Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow, United Kingdom (Recruiting)
- Leonard Wolfson Experimental Neurology Centre CRF — London, United Kingdom (Recruiting)
- St. George's University Hospital — London, United Kingdom (Recruiting)
- Salford Royal Hospital — Salford, United Kingdom (Recruiting)
- Royal Hallamshire Hospital — Sheffield, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Medical Information
- Email: medicalinfo@vrtx.com
- Phone: 617-341-6777
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.