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NCT Number: NCT06185764

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

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.

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Key information

Conditions

Myotonic Dystrophy Type 1 (DM1) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonia Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Neuromuscular Diseases Neuromuscular Manifestations Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

18 year–64 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Wesley Research Institute, Auchenflower, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

VX-670

Drug

Solution for intravenous administration.

Placebo

Drug

Solution for intravenous administration.

Primary outcomes

  1. 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

Secondary outcomes

  1. Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma

    Time frame: From Day 1 up to Day 42

  2. Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma

    Time frame: From Day 1 up to Day 42

  3. Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose

    Time frame: From Day 1 up to Day 168

  4. Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose

    Time frame: From Day 1 up to Day 168

  5. Part B: Concentration of VX-670 and its Active Component in Muscle

    Time frame: Baseline, Day 15 and Day 120

  6. Part B: Change in Splicing Index in Muscle Biopsy

    Time frame: Baseline, Day 15 and Day 120

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Information

CONTACT

[email protected]

617-341-6777

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study 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

Acronym: Galileo

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Dec 29, 2023
Registry last updated
Jun 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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