VX-670 for Myotonic Dystrophy

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Illness, Clinical condition, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

36 Participants Needed

Trial Summary

What is the purpose of this trial?

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).

Eligibility Criteria

This trial is specifically for individuals who have Myotonic Dystrophy Type I and have completed treatment in the parent study VX23-670-001. It's designed to assess long-term outcomes of using VX-670.

Inclusion Criteria

I completed treatment in the VX23-670-001 study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of VX-670

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive VX-670 to evaluate long-term safety, tolerability, and efficacy

Treatment Details

Interventions

• VX-670

Trial Overview The focus of this study is on the drug VX-670, which is being evaluated for its long-term safety, effectiveness, and how it's processed by the body (pharmacokinetics) in patients with Myotonic Dystrophy Type I.

Participant Groups

Group I: VX-670 Experimental Treatment 1 Intervention

Participants will receive multiple doses of VX-670.

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead Sponsor

Trials

267

Recruited

36,100+

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology