A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Purpose
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.
Condition
- Myotonic Dystrophy Type 1 (DM1)
Eligibility
- Eligible Ages
- Between 18 Years and 64 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
Exclusion Criteria
- History of any illness or any clinical condition as pre-specified in the protocol Other protocol defined Inclusion/Exclusion criteria may apply.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Part A: Single Ascending Dose |
Participants will be randomized to receive a single dose of different dose levels of VX-670. |
|
|
Placebo Comparator Part A: Placebo |
Participants will be randomized to receive single dose of placebo matched to VX-670. |
|
|
Experimental Part B: Single and Multiple Ascending Dose |
Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A. |
|
|
Placebo Comparator Part B: Placebo |
Participants will be randomized to receive single or multiple doses of placebo matched to VX-670. |
|
Recruiting Locations
University of Kansas Medical Center
Fairway, Kansas 66205
Fairway, Kansas 66205
More Details
- Status
- Recruiting
- Sponsor
- Vertex Pharmaceuticals Incorporated