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Part B: Single and Multiple Ascending Dose for Myotonic Dystrophy (Galileo Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

- 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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up part a: from baseline up to day 42; part b: from baseline and up to day 168

Awards & highlights

Galileo Trial Summary

This trial aims to find out if a drug called VX-670 is safe and well-tolerated in people with DM1. The study will also look at how the body processes and responds to different

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

Since this is an early-phase trial to determine safety and tolerability, specific side effects of VX-670 are not yet fully known but may be similar to other drugs targeting muscular disorders.

Galileo Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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It looks like the criterion is incomplete. Could you provide more details or context so that I can accurately summarize it for you?

Galileo Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ part a: from baseline up to day 42; part b: from baseline and up to day 168

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~part a: from baseline up to day 42; part b: from baseline and up to day 168

This trial's timeline: 3 weeks for screening, Varies for treatment, and part a: from baseline up to day 42; part b: from baseline and up to day 168 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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

Secondary outcome measures

Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma

Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma

Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose

+3 more

Galileo Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Part B: Single and Multiple Ascending DoseExperimental Treatment1 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 DoseExperimental Treatment1 Intervention

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

Group III: Part B: PlaceboPlacebo Group1 Intervention

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

Group IV: Part A: PlaceboPlacebo Group1 Intervention

Participants will be randomized to receive single dose of placebo matched to VX-670.

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor

243 Previous Clinical Trials

32,352 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are participants being actively sought for this ongoing research study?

"Indeed, as indicated on clinicaltrials.gov, this trial is currently in search of eligible participants. The study was originally posted on January 1st, 2024 and underwent its latest revision on January 10th, 2024."

Answered by AI

Do I meet the necessary criteria to participate in this medical study?

"- Confirmed diagnosis of DM1 based on clinical evaluation with an onset age greater than one year."

Answered by AI

What is the current number of individuals receiving treatment within this specific clinical trial?

"Indeed, the information available on clinicaltrials.gov indicates that this research endeavor is currently in the process of enrolling eligible individuals. The trial was initially posted on January 1st, 2024 and has since been updated most recently on January 10th, 2024. In order to complete the study successfully, a total of 36 participants will be admitted from one designated location."

Answered by AI

Is the clinical trial open to individuals who are below the age of 75?

"Patients aged between 18 and 64 are eligible to enroll in this clinical trial. Additionally, there are separate studies available for patients under the age of 18 (consisting of three trials) and individuals over the age of 65 (comprising six trials)."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06185764.