A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

NCT06926621 | Enrolling By Invitation Phase 2 FDA Drug

• 2 other identifiers: VX24-670-1012024-517983-47-00
• Study Type: interventional
• Target: 44
• Locations: 5 countries
• Sites: 11

Brief Summary

The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).

Conditions

Myotonic Dystrophy Type 1 (DM1)

Keywords

DM 1; Myotonic Dystrophy 1; Myotonic Dystrophy Type 1 (DM1); VX-670; Myotonic Dystrophy; DM; Myotonia; Dystrophy Myotonic; Myotonic Disorders; Steinert Disease; Vertex; Entrada; 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)

• Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  From Day 1 up to Week 108

Secondary Outcomes (2)

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

  From Day 1 up to Week 96

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

  From Day 1 up to Week 96

Study Arms (1)

VX-670

EXPERIMENTAL

Participants will receive multiple doses of VX-670.

Drug: VX-670

Interventions

VX-670 DRUG

Solution for intravenous administration.

VX-670

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Completed study drug treatment in parent study VX23-670-001 (NCT06185764)

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 (11)

Wesley Research Institute

Auchenflower, Australia

Location

Neuroscience Clinical Trials Unit, Alfred Brain

Melbourne, Australia

Location

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology

Leuven, Belgium

Location

Altasciences Montreal

Montreal, Canada

Location

Montreal Neurological Institute-Hospital

Montreal, Canada

Location

University of Ottawa

Ottawa, Canada

Location

Universite Laval - Neurology

Québec, Canada

Location

Hospital Universitario y Politecnico La Fe - Neurology

Valencia, Spain

Location

Queen Elizabeth University Hospital - Neurology

Glasgow, United Kingdom

Location

Leonard Wolfson Experimental Neurology Centre CRF

London, United Kingdom

Location

Royal Hallamshire Hospital - Neurology

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 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2025
• First Posted: April 14, 2025
• Study Start: April 28, 2025
• Primary Completion (Estimated): January 30, 2029
• Study Completion (Estimated): January 30, 2029
• Last Updated: February 27, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

GB (3); CA (4); ES (1); AU (2); BE (1)