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

NCT06185764 | Active Not Recruiting Phase 1 FDA Drug

• 2 other identifiers: VX23-670-0012023-506028-10-00
• Study Type: interventional
• Target: 47
• Locations: 10 countries
• Sites: 26

Brief 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.

Conditions

Myotonic Dystrophy Type 1 (DM1)

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (6)

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

  From Day 1 up to Day 42

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

  From Day 1 up to Day 42

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

  From Day 1 up to Day 168

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

  From Day 1 up to Day 168

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

  Baseline, Day 15 and Day 120

Study Arms (4)

Part A: Single Ascending Dose

EXPERIMENTAL

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

Drug: VX-670

Part A: Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Part B: Single and Multiple Ascending Dose

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.

Drug: VX-670

Part B: Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

VX-670 DRUG

Solution for intravenous administration.

Part A: Single Ascending Dose; Part B: Single and Multiple Ascending Dose

Placebo DRUG

Solution for intravenous administration.

Part A: Placebo; Part B: Placebo

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• \- 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

You may not qualify if:

• \- History of any illness or any clinical condition as pre-specified in the protocol

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (26)

Stanford Neuromuscular Research

San Carlos, California, 94070, United States

Location

University of Florida Clinical Research Center

Gainesville, Florida, 32608, United States

Location

University of Kansas Medical Center

Fairway, Kansas, 66205, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University School of Medicine / St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Wake Forest Baptist Health

Winston-Salem, North Carolina, 27104, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Virginia Commonwealth University (Sanger Hall)

Richmond, Virginia, 23298, United States

Location

Wesley Research Institute

Auchenflower, Australia

Location

Neuroscience Clinical Trials Unit, Alfred Brain

Melbourne, Australia

Location

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

Leuven, Belgium

Location

Hopital de Chicoutimi

Chicoutimi, Canada

Location

Altasciences Montreal

Montreal, Canada

Location

McGIll University

Montreal, Canada

Location

University of Ottawa

Ottawa, Canada

Location

CHU Research Centre of Quebec

Québec, Canada

Location

Neuromuscular Reference Center Institute of Myology

Paris, France

Location

Ludwig Maximilians Universitaet Muenchen

München, Germany

Location

Centro Clinico NeMO

Milan, Italy

Location

Maastricht University Medical Center

Maastricht, Netherlands

Location

Hospital Universitario y Politécnico La Fe

Valencia, Spain

Location

Clinical Research Facility, Queen Elizabeth University Hospital

Glasgow, United Kingdom

Location

Leonard Wolfson Experimental Neurology Centre CRF

London, United Kingdom

Location

St. George's University Hospital

London, United Kingdom

Location

Salford Royal Hospital

Salford, United Kingdom

Location

Royal Hallamshire Hospital

Sheffield, United Kingdom

Location

MeSH Terms

Conditions

Myotonic Dystrophy; Myotonia; Myotonic Disorders; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Muscular Dystrophies

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2023
• First Posted: December 29, 2023
• Study Start: February 20, 2024
• Primary Completion (Estimated): February 2, 2027
• Study Completion (Estimated): February 2, 2027
• Last Updated: August 20, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); US (8); BE (1); CA (5); DE (1); NL (1); GB (5); IT (1); ES (1); AU (2)