Myotonic Dystrophy Type 1 (DM1)
Conditions
Keywords
DM1, DM2, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Myotonia, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Vertex, Entrada, VX-670, VX670, PMO, ASO, Myotonic Muscular Dystrophy, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases, Muscular Dystrophies
Brief summary
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.
Interventions
DRUGVX-670
Solution for intravenous administration.
DRUGPlacebo
Solution for intravenous administration.
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 64 Years
Healthy volunteers
No
Inclusion 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) | Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168 |
Secondary
| Measure | Time frame |
|---|---|
| Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma | From Day 1 up to Day 42 |
| Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma | From Day 1 up to Day 42 |
| Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose | From Day 1 up to Day 168 |
| Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose | From Day 1 up to Day 168 |
| Part B: Concentration of VX-670 and its Active Component in Muscle | Baseline, Day 15 and Day 120 |
| Part B: Change in Splicing Index in Muscle Biopsy | Baseline, Day 15 and Day 120 |
Countries
Australia, Belgium, Canada, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States
Outcome results
None listed