VX-670 for Myotonic Dystrophy

(Galileo Trial)

Recruiting at 28 trial locations

Overseen By Medical Information

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Illness, Clinical condition, others

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 safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

Eligibility Criteria

This trial is for adults with Myotonic Dystrophy type 1 (DM1), who had symptoms start after their first birthday and have a genetic test confirming at least 100 CTG repeats. It's not suitable for people outside these criteria.

Inclusion Criteria

I have DM1 with onset after 1 year old and a positive genetic test showing at least 100 CTG repeats.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Participants receive a single ascending dose of VX-670 or placebo

1-2 weeks

Treatment Part B

Participants receive single and multiple ascending doses of VX-670 or placebo

4-8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• VX-670

Trial Overview The study tests VX-670, a potential new treatment for DM1. Participants will receive different doses of VX-670 or a placebo to assess the drug's safety, how well it's tolerated, its movement through the body, and its effects on the disease.

Participant Groups

Group I: Part B: Single and Multiple Ascending Dose Experimental Treatment 1 Intervention

Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A.

Group II: Part A: Single Ascending Dose Experimental Treatment 1 Intervention

Participants will be randomized to receive a single dose of different dose levels of VX-670.

Group III: Part B: Placebo Placebo Group 1 Intervention

Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.

Group IV: Part A: Placebo Placebo Group 1 Intervention

Participants will be randomized to receive single dose of placebo matched to 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