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

NCT06185764 · tracked via the Priya Life Science UK tracker

Sponsor

Vertex Pharmaceuticals Incorporated

Phase

Phase 1/2

Started

2024-02-20

Last updated

2026-08-20

Condition(s) studied

Myotonic Dystrophy Type 1 (DM1)

Investigational drug(s) / intervention(s)

VX-670Placebo

VX-670: Solution for intravenous administration.

Placebo: Solution for intravenous administration.

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

64 Years

Healthy volunteers

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/Exclusion criteria may apply.

Primary outcome measure(s)

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

Trial sites (26)

Facility	City	Region
Stanford Neuromuscular Research	San Carlos	California
University of Florida Clinical Research Center	Gainesville	Florida
University of Kansas Medical Center	Fairway	Kansas
Boston Children's Hospital	Boston	Massachusetts
Washington University School of Medicine / St. Louis Children's Hospital	St Louis	Missouri
Wake Forest Baptist Health	Winston-Salem	North Carolina
University of Pennsylvania	Philadelphia	Pennsylvania
Virginia Commonwealth University (Sanger Hall)	Richmond	Virginia
Wesley Research Institute	Auchenflower	Australia
Neuroscience Clinical Trials Unit, Alfred Brain	Melbourne	Australia
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg	Leuven	Belgium
Hopital de Chicoutimi	Chicoutimi	Canada
Altasciences Montreal	Montreal	Canada
McGIll University	Montreal	Canada
University of Ottawa	Ottawa	Canada
CHU Research Centre of Quebec	Québec	Canada
Neuromuscular Reference Center Institute of Myology	Paris	France
Ludwig Maximilians Universitaet Muenchen	München	Germany
Centro Clinico NeMO	Milan	Italy
Maastricht University Medical Center	Maastricht	Netherlands
Hospital Universitario y Politécnico La Fe	Valencia	Spain
Clinical Research Facility, Queen Elizabeth University Hospital	Glasgow	United Kingdom
Leonard Wolfson Experimental Neurology Centre CRF	London	United Kingdom
St. George's University Hospital	London	United Kingdom
Salford Royal Hospital	Salford	United Kingdom
Royal Hallamshire Hospital	Sheffield	United Kingdom

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Other trials for the same condition

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 Recruiting

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I Enrolling by invitation

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06185764 on ClinicalTrials.gov ↗ ← All trials in the UK