ENROLLING BY INVITATIONPhase 2

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

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).

Sponsor

Vertex Pharmaceuticals Incorporated

Enrollment

44 participants

Sites

12 locations

Start Date

April 2025

Completion

January 2029

Myotonic Dystrophy Type 1 (DM1)VX-670

Enrollment & Timeline

ENROLLING BY INVITATION

Target enrollment (estimated)

Target N

participants

Sites

locations

Duration

3.8 years

total trial span

Rate

pts/month needed

Apr 2025Month 14 of 45Jan 2029

31% of trial timeline elapsed31 months to primary endpoint

At 12 sites, each site needs to enroll approximately 0.1 patients/month to hit the enrollment target on schedule.

Enrollment target is estimated. ClinicalTrials.gov does not provide real-time enrollment progress.

View on ClinicalTrials.gov

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Primary Outcomes

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

From Day 1 up to Week 108

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Multi-Source Intelligencefrom PubMed · FDA FAERS · WHO ICTRP

No linked publications found in PubMed

PubMed/NCBI (daily updates) FDA FAERS (quarterly updates) WHO ICTRP (weekly updates)

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Fetching protocol from ClinicalTrials.gov…

ENROLLING BY INVITATIONPhase 2

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

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).

Sponsor

Vertex Pharmaceuticals Incorporated

Enrollment

44 participants

Sites

12 locations

Start Date

April 2025

Completion

January 2029

Myotonic Dystrophy Type 1 (DM1)VX-670

Enrollment & Timeline

ENROLLING BY INVITATION

Target enrollment (estimated)

Target N

participants

Sites

locations

Duration

3.8 years

total trial span

Rate

pts/month needed

Apr 2025Month 14 of 45Jan 2029

31% of trial timeline elapsed31 months to primary endpoint

At 12 sites, each site needs to enroll approximately 0.1 patients/month to hit the enrollment target on schedule.

Enrollment target is estimated. ClinicalTrials.gov does not provide real-time enrollment progress.

View on ClinicalTrials.gov

Ask AI

Primary Outcomes

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

From Day 1 up to Week 108

Ask AI

Multi-Source Intelligencefrom PubMed · FDA FAERS · WHO ICTRP

No linked publications found in PubMed

PubMed/NCBI (daily updates) FDA FAERS (quarterly updates) WHO ICTRP (weekly updates)

Ask AI

Eligibility

Min Age

18 Years

Sex

all

Healthy Volunteers

View full 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 Sites (12)

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Wesley Research Institute

Auchenflower, Australia

Neuroscience Clinical Trials Unit, Alfred Brain

Melbourne, Australia

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology

Leuven, Belgium

Altasciences Montreal

Montreal, Canada

Montreal Neurological Institute-Hospital

Montreal, Canada

University of Ottawa

Ottawa, Canada

Universite Laval - Neurology

Québec, Canada

Maastricht UMC

Maastricht, Netherlands

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