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

NCT ID: NCT06926621

Last Updated: 2026-01-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2

Total Enrollment

36 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-04-28

Study Completion Date

2028-07-24

Brief Summary

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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).

Detailed Description

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Conditions

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Myotonic Dystrophy Type 1 (DM1)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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VX-670

Participants will receive multiple doses of VX-670.

Group Type EXPERIMENTAL

VX-670

Intervention Type DRUG

Solution for intravenous administration.

Interventions

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VX-670

Solution for intravenous administration.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

• History of any illness or any clinical condition as pre-specified in the protocol
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Wesley Research Institute

Auchenflower, , Australia

Site Status

Neuroscience Clinical Trials Unit, Alfred Brain

Melbourne, , Australia

Site Status

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology

Leuven, , Belgium

Site Status

Altasciences Montreal

Montreal, , Canada

Site Status

Montreal Neurological Institute-Hospital

Montreal, , Canada

Site Status

University of Ottawa

Ottawa, , Canada

Site Status

Universite Laval - Neurology

Québec, , Canada

Site Status

Hospital Universitario y Politecnico La Fe - Neurology

Valencia, , Spain

Site Status

Queen Elizabeth University Hospital - Neurology

Glasgow, , United Kingdom

Site Status

Leonard Wolfson Experimental Neurology Centre CRF

London, , United Kingdom

Site Status

Royal Hallamshire Hospital - Neurology

Sheffield, , United Kingdom

Site Status

Countries

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Australia Belgium Canada Spain United Kingdom

Other Identifiers

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2024-517983-47-00

Identifier Type: OTHER

Identifier Source: secondary_id

VX24-670-101

Identifier Type: -

Identifier Source: org_study_id

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