At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- VX-670 (drug), Placebo (drug)
- How long the study runs
- Study runs about 36 months (dates as stated)
- About the drug or intervention
- VX-670 — drug: Solution for intravenous administration. · Placebo — drug: Solution for intravenous administration.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 18 Years to 64 Years
- Who
- All
- Number of participants
- 47
- Started
- 2024-02-20
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for myotonic dystrophy type 1 (dm1)
- • Phase1/Phase2 - 47 participants
- • 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
Who can take part?
- • Ages 18 Years to 64 Years
- • Diagnosed with myotonic dystrophy type 1 (dm1)
Where?
- • Glasgow - Clinical Research Facility, Queen Elizabeth University Hospital
- • London - Leonard Wolfson Experimental Neurology Centre CRF
- • London - St. George's University Hospital
- • Salford - Salford Royal Hospital
- • +1 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 64 Years
- Who can join: All genders
Biomarkers mentioned
Who cannot take part
- ✗\- History of any illness or any clinical condition as pre-specified in the protocol
- ✗Other protocol defined Inclusion/Exclusion criteria may apply.
See the full criteria
Where Is This Study? (5 UK sites)
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
Leonard Wolfson Experimental Neurology Centre CRF
London, United Kingdom
St. George's University Hospital
London, United Kingdom
Salford Royal Hospital
Salford, United Kingdom
Royal Hallamshire Hospital
Sheffield, United Kingdom