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

Part of paid clinical trials in St Louis, Missouri.

Sponsor
Vertex Pharmaceuticals Incorporated
Study ID
NCT06926621
Phase
PHASE2
Status
Enrolling By Invitation

Conditions

  • Myotonic Dystrophy Type 1 (DM1)

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • VX-670 — DRUG
    Solution for intravenous administration.

Study Details

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).

Key Dates

First listed
Apr 14, 2025
Start date
Apr 28, 2025
Status verified
Aug 2026
Primary completion
Jan 30, 2029
Completion
Jan 30, 2029

Study Design

Enrollment
44 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: VX-670
    Participants will receive multiple doses of VX-670.

Primary Outcome Measure

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 ]

Locations (2)

FacilityCityStateZIPSite coordinators
St. Louis Children's Hospital - NeurologySt LouisMissouri63110-
Virginia Commonwealth University - Sanger HallRichmondVirginia23298-

Find similar trials in St Louis, MO

By research site
St. Louis Children's Hospital - Neurology· St Louis, MOVirginia Commonwealth University - Sanger Hall· Richmond, VA

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