A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Part of paid clinical trials in San Carlos, California.

Sponsor
Vertex Pharmaceuticals Incorporated
Study ID
NCT06185764
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Myotonic Dystrophy Type 1 (DM1)

Eligibility Criteria

Sex
ALL
Age
18 Years - 64 Years
Healthy Volunteers
Not accepted

Interventions

  • VX-670 — DRUG
    Solution for intravenous administration.
  • Placebo — DRUG
    Solution for intravenous administration.

Study Details

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.

Key Dates

Start date
Feb 20, 2024
Status verified
Jun 2026
Primary completion
Feb 2, 2027
Completion
Feb 2, 2027

Study Design

Enrollment
52 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • 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.

Primary Outcome Measure

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 ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
Stanford Neuromuscular ResearchSan CarlosCalifornia94070-
University of Florida Clinical Research CenterGainesvilleFlorida32608-
University of Kansas Medical CenterFairwayKansas66205-
Boston Children's HospitalBostonMassachusetts02115-
Washington University School of Medicine / St. Louis Children's HospitalSt LouisMissouri63110-
Wake Forest Baptist HealthWinston-SalemNorth Carolina27104-
University of PennsylvaniaPhiladelphiaPennsylvania19104-
Virginia Commonwealth University (Sanger Hall)RichmondVirginia23298-

Find similar trials in San Carlos, CA

By research site
Stanford Neuromuscular Research· San Carlos, CAUniversity of Florida Clinical Research Center· Gainesville, FLUniversity of Kansas Medical Center· Fairway, KSBoston Children's Hospital· Boston, MAWashington University School of Medicine / St. Louis Children's Hospital· St Louis, MOWake Forest Baptist Health· Winston-Salem, NC

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