VX-670 for Myotonic Dystrophy

(Galileo Trial)

Recruiting at 31 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called VX-670, a potential drug for individuals with myotonic dystrophy type 1 (DM1), a condition causing muscle weakness and other symptoms. The study aims to assess the safety and tolerability of VX-670 at various dose levels and how the body processes it. Participants will receive either the actual treatment or a placebo (a harmless pill with no active drug) for comparison. The trial seeks participants with a confirmed DM1 diagnosis, a genetic test showing at least 100 CTG repeats, and symptoms that began after their first birthday. As a Phase 1 trial, this research focuses on understanding how VX-670 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

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

Is there any evidence suggesting that VX-670 is likely to be safe for humans?

Research is examining the safety of VX-670 for individuals with Myotonic Dystrophy Type 1. This treatment aims to address a cellular issue caused by the disease.

So far, VX-670 appears well-tolerated, with most participants not experiencing serious side effects. However, as testing continues, a complete safety profile is not yet available. Participants in these studies have received varying doses to determine the safest and most effective amount.

VX-670 is not yet approved for any condition. The current testing phase primarily focuses on ensuring its safety for humans. Researchers are closely monitoring for adverse reactions.

While early results are promising, more information is needed to fully understand the safety of VX-670 for everyone. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for myotonic dystrophy?

Unlike the standard treatments for myotonic dystrophy, which often focus on managing symptoms with medications like mexiletine or physical therapy, VX-670 is designed to target the underlying cause of the disease. VX-670 is unique because it introduces a new mechanism of action that aims to directly influence the genetic mutations responsible for myotonic dystrophy. Researchers are excited about VX-670 because it has the potential to provide more targeted and effective relief than existing options, which are more palliative in nature and do not address the root cause. This could mean significant improvements in muscle function and quality of life for patients.

What evidence suggests that VX-670 might be an effective treatment for Myotonic Dystrophy?

Research has shown that VX-670 aims to help people with Myotonic Dystrophy Type 1 by fixing a problem called mis-splicing. Mis-splicing occurs when genetic instructions become mixed up, leading to symptoms of the disease. VX-670 is designed to correct this issue and address the root cause of the disease, potentially improving muscle function. Participants in this trial will receive either single or multiple doses of VX-670, or a placebo. Although researchers are still studying this treatment, early results suggest it can effectively reach the muscle tissues where needed. This could lead to better outcomes for patients with this condition. ^{2 3 5 6 7}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06926621

A Study of Long-term Safety and Efficacy of VX-670 in ... 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 ...

2. vrtx.com vrtx.com/our-science/pipeline/myotonic-dystrophy-type-1/

R&D Pipeline | Myotonic Dystrophy Type 1 Through this mechanism, VX-670 aims to correct mis-splicing, and is being investigated to address the underlying cause of disease. Whereas delivery into muscle ...

3. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/453495

A Phase 1/2 Study of VX-670 in Adult Participants With ... The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and ...

4. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-of-vx-670-for-adults-with-myotonic-dystrophy-type-1/

Study on the Safety of VX-670 for Adults with Myotonic ... This clinical trial evaluates the safety and tolerability of VX-670, a potential treatment for adults with Myotonic Dystrophy Type 1, a genetic disorder ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06185764

NCT06185764 | A Phase 1/2 Study of VX-670 in Adult ... The DMC can recommend to the sponsor that the trial be stopped if it is not effective, is harming participants, or is unlikely to serve its scientific purpose.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06926621

A Study of Long-term Safety and Efficacy of VX-670 in ... 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 ...

7. clinicaltrial.be clinicaltrial.be/en/details/421612?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

A Phase 1/2 Study of VX-670 in Adult Participants With My... The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in ...