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

Phase 2Enrolling By InvitationNCT06926621

Vertex Pharmaceuticals Incorporated36 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Myotonic Dystrophy Type 1 (DM1)

Interventions

VX-670DRUG

Solution for intravenous administration.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: • Completed study drug treatment in parent study VX23-670-001 (NCT06185764) 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.

Locations (11)

Wesley Research Institute

Auchenflower, Australia

Neuroscience Clinical Trials Unit, Alfred Brain

Melbourne, Australia

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology

Leuven, Belgium

Outcomes

Primary Outcomes

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

Time frame: From Day 1 up to Week 108

Secondary Outcomes

Change From Baseline in Splicing Index in Muscle Biopsy at Week 24

Time frame: Baseline and at Week 24

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

Time frame: From Day 1 up to Week 96

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

Time frame: From Day 1 up to Week 96

Concentration of VX-670 and its Active Component in Muscle

Time frame: Baseline and at Week 24

Data from ClinicalTrials.gov

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