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Study on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1

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What is this study about?

This clinical trial is focused on studying Myotonic Dystrophy Type 1, a genetic disorder that affects muscle function. The study will evaluate a new treatment called VX-670, which is administered as a solution for injection or infusion. The purpose of the study is to assess the safety and tolerability of this treatment in adults with Myotonic Dystrophy Type 1.

The study is divided into two parts. In the first part, participants will receive single doses of VX-670 to determine how well it is tolerated. In the second part, both single and multiple doses will be given to further evaluate safety and tolerability. Some participants will receive a placebo, which is a substance with no active medication, to compare the effects of VX-670.

Throughout the study, participants will be closely monitored for any side effects or changes in their health. This will include regular check-ups, laboratory tests, and assessments of heart function using standard tests like electrocardiograms (ECGs). The study aims to gather important information about how VX-670 is processed in the body and its potential impact on muscle function in individuals with Myotonic Dystrophy Type 1.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying a clinical diagnosis of myotonic dystrophy type 1 (DM1) and ensuring the participant meets specific criteria such as age, body mass index, and genetic test results.

2 part a: single dose administration

Participants receive a single dose of VX-670 solution, administered as an injection or infusion. The purpose is to evaluate safety and tolerability. Monitoring includes checking for any adverse effects, conducting laboratory tests, and performing electrocardiograms (ECGs).

3 part b: multiple dose administration

Participants may receive multiple doses of VX-670 to further assess safety and tolerability. This phase also involves monitoring for adverse effects, laboratory tests, and ECGs. Additional assessments include measuring drug concentration in plasma and muscle tissue, and evaluating changes in muscle biopsy samples.

4 ongoing monitoring

Throughout the trial, participants undergo regular monitoring to ensure safety. This includes tracking vital signs and using the Columbia Suicide Severity Rating Scale (C-SSRS) to assess mental health.

5 completion and follow-up

Upon completion of the trial, a final assessment is conducted. This includes reviewing all collected data to evaluate the overall safety and effectiveness of VX-670 in treating myotonic dystrophy type 1.

Who Can Join the Study?

  • The patient must have a body mass index (BMI) of less than 35.0 kg/m² and weigh more than 40 kg. BMI is a measure that uses height and weight to estimate body fat.
  • The patient should be between 18 and 64 years old. Both men and women can participate, including women who can have children, as long as local rules allow it.
  • The patient must have a confirmed diagnosis of myotonic dystrophy type 1 (DM1), with symptoms starting after the age of 1, and a positive genetic test showing a specific change in their DNA called a CTG repeat of at least 100.
  • For Part B of the study, the patient must be able to walk 10 meters without help, although they can use a brace or similar device if needed.
  • For Part B, the patient must show signs of myotonia, which means their muscles take longer than normal to relax after contracting. This is measured by a test called HOT, which should be 2 seconds or more.
  • For Part B, the patient must show signs of muscle weakness, measured by how strong their hand grip is compared to what is expected. This should be between 20% and 80% of the expected strength.
  • The patient must have a left ventricular ejection fraction (LVEF) greater than 55% within the last 3 months. LVEF is a measure of how well the heart pumps blood with each beat.

Who Cannot Join the Study?

  • Individuals who do not have Myotonic Dystrophy type 1 (DM1) cannot participate. This is a genetic condition that affects muscles and other body systems.
  • Participants must be within a specific age range, which is not specified here, but typically means they must be adults.
  • Both males and females are eligible, so gender is not a reason for exclusion.
  • Individuals who are considered part of a vulnerable population, such as those unable to give informed consent, are not included.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Hospital Universitario Y Politecnico La Fe Valencia Spain
University Hospital Maastricht Maastricht The Netherlands
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Friedrich Baur Institute An Der Neurologischen Klinik Und Poliklinik Munich Germany
Stichting Radboud University Medical Center Nijmegen The Netherlands
Association Institut De Myologie Paris France
Clniwd Cuqiotr Nblg Milan Italy
Uarxthehisynrvfngeddb Esdxg Adl Essen Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
15.05.2024
France France
Recruiting
15.05.2024
Germany Germany
Recruiting
15.05.2024
Italy Italy
Recruiting
15.05.2024
Spain Spain
Recruiting
15.05.2024
The Netherlands The Netherlands
Recruiting
15.05.2024

Trial locations

Investigated drugs:

VX 670 is an investigational medication being studied for its potential to treat myotonic dystrophy type 1 (DM1). The trial aims to assess how safe and tolerable this medication is when given in single and multiple doses to adults with DM1. The study also looks at how the body processes VX 670 and its effects on the body.

Investigated diseases:

Myotonic Dystrophy – Myotonic dystrophy is a genetic disorder characterized by progressive muscle wasting and weakness. It primarily affects the muscles, but it can also impact other systems in the body, such as the heart, eyes, and endocrine system. The condition is marked by prolonged muscle contractions (myotonia) and an inability to relax muscles after use. Symptoms often begin in adulthood and can vary widely in severity among individuals. Over time, muscle weakness and myotonia can lead to difficulties with movement and daily activities. The progression of the disease can be slow, with symptoms worsening gradually over many years.

Trial ID:
2023-506028-10-00
Protocol code:
VX23-670-001
Trial Phase:
Phase I and Phase II (Integrated) – First administration to humans

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