RECRUITING

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

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Conditions

Myotonic Dystrophy Type 1 (DM1)DM1DM2Myotonic Dystrophy 1Myotonic Dystrophy 2Myotonic DystrophyDMMyotoniaDystrophy MyotonicMyotonic DisordersSteinert DiseaseVertexEntradaVX-670VX670PMOASOMyotonic Muscular DystrophyMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesMuscular Dystrophies

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official 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

Quick Facts

Study Start:2024-02-20
Study Completion:2026-12-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06185764

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 64 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Medical Information
CONTACT
617-341-6777
medicalinfo@vrtx.com

Study Locations (Sites)

Stanford Neuromuscular Research
San Carlos, California, 94070
United States
University of Florida Clinical Research Center
Gainesville, Florida, 32608
United States
University of Kansas Medical Center
Fairway, Kansas, 66205
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Washington University School of Medicine / St. Louis Children's Hospital
Saint Louis, Missouri, 63110
United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27104
United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
United States
Virginia Commonwealth University (Sanger Hall)
Richmond, Virginia, 23298
United States

Collaborators and Investigators

Sponsor: Vertex Pharmaceuticals Incorporated

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-02-20
Study Completion Date2026-12-31

Study Record Updates

Study Start Date2024-02-20
Study Completion Date2026-12-31

Terms related to this study

Keywords Provided by Researchers

  • DM1
  • DM2
  • Myotonic Dystrophy 1
  • Myotonic Dystrophy 2
  • Myotonic Dystrophy Type 1 (DM1)
  • Myotonic Dystrophy
  • DM
  • Myotonia
  • Dystrophy Myotonic
  • Myotonic Disorders
  • Steinert Disease
  • Vertex
  • Entrada
  • VX-670
  • VX670
  • PMO
  • ASO
  • Myotonic Muscular Dystrophy
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Musculoskeletal Diseases
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, Inborn
  • Heredodegenerative Disorders, Nervous System
  • Neurodegenerative Diseases
  • Muscular Dystrophies

Additional Relevant MeSH Terms

  • Myotonic Dystrophy Type 1 (DM1)