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

Phase 2Enrolling By InvitationNCT06926621

Vertex Pharmaceuticals Incorporated44 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 (15)

St. Louis Children's Hospital - Neurology

St Louis, Missouri, United States

Virginia Commonwealth University - Sanger Hall

Richmond, Virginia, United States

Wesley Research Institute

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

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

Data from ClinicalTrials.gov

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