A Phase 1/2 Study of VX-670 in Adult Subjects with Myotonic Dystrophy Type 1
Research type
Research Study
Full 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
IRAS ID
1008324
Contact name
Alicia Lee
Contact email
Sponsor organisation
Vertex Pharmaceuticals Inc.
Eudract number
2023-506028-10
Clinicaltrials.gov Identifier
Research summary
This study VX23-670-001 is a Phase 1/2, first-in-human (FIH) randomized, double-blind, placebo controlled, 2 part study to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of a single ascending dose (Part A) and single and multiple ascending doses (Part B) of VX-670 in subjects with Myotonic Dystrophy Type 1.VX-670, is designed to target the cause of DM1 by directly binding the increased repeats in the DMPK transcript. There are two parts to this study, Parts A (`12 subjects) & B (24 subjects) will participate in this study. Part A: after the Screening/Baseline Period, participants will receive a single dose of study drug on Day 1 followed by 24 hours safety monitoring. They will remain in the study for 6 weeks after dosing. Eligible participants will be assigned to a study treatment group or cohort. They will be informed which study treatment group they are assigned to and it is possible that they will know whether they are receiving active study drug. If they are assigned to cohort A1 or A2, they will receive the active study drug. In cohort A3, it is possible that they will instead receive placebo. If they are assigned to cohort A3, they have a 3 out of 4 (75%) chance of being assigned to receive the study drug. Part B: after the Screening/Baseline Period, participants will receive a single dose of study drug on Day 1 followed by 24 hours safety monitoring. They will receive a second dose on Day 43 and third dose on Day 85. After each of these doses they will remain in the clinic for approx 4-6 hours after the study drug administration for safety monitoring. They will remain in the study for 24 weeks. Eligible participants will be assigned to 1of 3 cohorts. They will know their treatment group. They will not know whether they are receiving active study drug or placebo. Participants have a 75% chance of being assigned to receive VX-670 and a 25% chance of being assigned to receive placebo on Day 1, Day 43, and Day 85.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
23/SC/0387
Date of REC Opinion
13 Dec 2023
REC opinion
Further Information Favourable Opinion