Recruiting

GalileoVX-670 for Myotonic Dystrophy Type 1

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Study Aim

This study aims to evaluate the safety and tolerability of VX-670 in individuals with Myotonic Dystrophy Type 1 by monitoring for adverse events.

What is being tested

VX-670

+ Placebo

Drug
Who is being recruted

Muscular Diseases+14

+ Muscular Dystrophies

+ Musculoskeletal Diseases

From 18 to 64 Years
+3 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Placebo-ControlledPhase 1 & 2
Interventional
Study Start: February 2024
See protocol details

Summary

Principal SponsorVertex Pharmaceuticals Incorporated
Study ContactMedical Information
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: February 20, 2024

Actual date on which the first participant was enrolled.

This clinical trial aims to explore the safety and effects of a new drug, VX-670, in adults with Myotonic Dystrophy Type 1 (DM1). DM1 is a genetic disorder that affects muscle function, leading to various health challenges. The study is crucial as it seeks to understand how the body processes VX-670 and its impact on DM1 symptoms. Understanding the safety and effectiveness of this medication could lead to improved treatment options for those living with this condition. Participants in the trial will receive either VX-670 or a placebo, which is a substance with no active drug, given in varying doses. The study will assess how well participants tolerate the medication and monitor how the drug moves through and affects the body. Observations will focus on any side effects and changes in disease symptoms to ensure the treatment is safe and potentially beneficial for future use.

Official TitleA 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
NCT06185764
Principal SponsorVertex Pharmaceuticals Incorporated
Study ContactMedical Information
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

36 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 64 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Muscular DiseasesMuscular DystrophiesMusculoskeletal DiseasesMyotoniaMyotonic DystrophyCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsNeuromuscular DiseasesSigns and SymptomsPathological Conditions, Signs and SymptomsNeurodegenerative DiseasesHeredodegenerative Disorders, Nervous SystemNeuromuscular ManifestationsMuscular Disorders, AtrophicMyotonic DisordersGenetic Diseases, Inborn

Criteria

2 inclusion criteria required to participate
Other protocol defined Inclusion/Exclusion criteria may apply.
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
1 exclusion criteria prevent from participating
History of any illness or any clinical condition as pre-specified in the protocol

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

4 intervention groups are designated in this study

50% chance of being blinded to the placebo group

Treatment Groups

Group I

Experimental
Participants will be randomized to receive a single dose of different dose levels of VX-670.

Group II

Experimental
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.

Group III

Placebo
Participants will be randomized to receive single dose of placebo matched to VX-670.

Group IV

Placebo
Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 26 locations

Recruiting

Stanford Neuromuscular Research

San Carlos, United StatesOpen Stanford Neuromuscular Research in Google Maps
Recruiting

University of Florida Clinical Research Center

Gainesville, United States
Recruiting

University of Kansas Medical Center

Fairway, United States
Recruiting

Boston Children's Hospital

Boston, United States
Recruiting
26 Study Centers