Vertex
Are You Eligible?To evaluate the safety and tolerability of an investigational study drug compared to a placebo in adults with Myotonic Dystrophy (DM1).
Research Objectives
Status
Recruitment
Total participants required
Study location
Study type
Patient group
About this clinical trial
To purpose of this clinical trial is to test the safety and tolerability of an investigational study drug compared to a placebo in adults with Myotonic Dystrophy (DM1). “Investigational” means the drug is not approved by regulatory health authorities for the treatment of DM1, and is still being tested for safety and effectiveness.
For more information visit https://clinicaltrials.gov/study/NCT06185764
Eligibility
- Be between the ages of 18 and 64 (male & female)
- Be willing and able to comply with scheduled visits, a treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures
- Weigh more than 40 kg and have a BMI of less than 35 kg/m2
- Have a documented diagnosis of DM1 with onset of symptoms at older than one year of age (genotype testing can be performed as part of study participation at no additional cost to you)
- Have evidence of myotonia, as defined by hand opening time of more than two seconds
- Be ambulant (able to walk without assistance from an orthotic device or brace)
Lead investigator
- A/Prof Robert Henderson
Clinical trial coordinators
- Jacqui Langton
- Emma Brownrigg
Technical title
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
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