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
Overview
- Phase
- Phase 1
- Intervention
- VX-670
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Enrollment
- 44
- Locations
- 34
- Primary Endpoint
- Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
- Status
- Recruiting
- Last Updated
- 2 months ago
Overview
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.
Investigators
Eligibility Criteria
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.
Arms & Interventions
Part A: Single Ascending Dose
Participants will be randomized to receive a single dose of different dose levels of VX-670.
Intervention: VX-670
Part A: Placebo
Participants will be randomized to receive single dose of placebo matched to VX-670.
Intervention: Placebo
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.
Intervention: VX-670
Part B: Placebo
Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.
Intervention: Placebo
Outcomes
Primary Outcomes
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
Secondary Outcomes
- 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 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: 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 A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma(From Day 1 up to Day 42)
- 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)