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

Vertex Pharmaceuticals

Status and phase: Invitation-only; Phase 2

Conditions: Myotonic Dystrophy Type 1 (DM1)

Treatments: Drug: VX-670

Study type: Interventional

Funder types: Industry

Identifiers

NCT06926621

2024-517983-47-00 (Other Identifier)

VX24-670-101

Details and patient eligibility

About

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).

Enrollment: 36 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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36 participants in 1 patient group

VX-670

Experimental group

Description:

Participants will receive multiple doses of VX-670.

Treatment:

Drug: VX-670