RecruitingPhase 1/2

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

United States44 participantsStarted 2024-02-20

Plain-language summary

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.

Who can participate

Age range18 Years – 64 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Key Inclusion Criteria: \- Documented clinical diagnosis of DM1 with age of onset greater than (\>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100 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.

What they're measuring

Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Timeframe: Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168

Trial details

NCT IDNCT06185764

SponsorVertex Pharmaceuticals Incorporated

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-12-22

Contact for this trial

Medical Information

617-341-6777 medicalinfo@vrtx.com

View on ClinicalTrials.gov More Myotonic Dystrophy Type 1 (DM1) trials