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Clinical Trials in the UK / NCT06185764
Active, not recruiting Phase 1/2

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-670 →Placebo

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
No
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)

FacilityCityRegionStatus
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

More Vertex Pharmaceuticals Incorporated trials in the UK

Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy Recruiting
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) Active, not recruiting
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting
A Long-term Follow-up Study in Participants Who Received CTX001 Enrolling by invitation
Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease Active, not recruiting

Other trials for the same condition

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 Recruiting
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 Active, not 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