Study to Determine the Best Dose of Vesleteplirsen for Patients with Duchenne Muscular Dystrophy Suitable for Exon 51-Skipping Treatment

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What is this study about?

This clinical trial is focused on studying Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle weakness and degeneration. The treatment being tested is called Vesleteplirsen (SRP-5051), which is administered through an intravenous infusion. This means the medication is given directly into a vein. The study aims to determine the best dose of Vesleteplirsen and to evaluate its effects on the levels of a protein called dystrophin in the muscles. Dystrophin is important for muscle strength and function, and the study is particularly interested in patients whose condition can benefit from a specific type of genetic treatment known as exon 51-skipping.

The study is divided into two parts. In the first part, participants will receive different doses of Vesleteplirsen to find the highest dose that can be tolerated safely. This part will help researchers understand how the body handles the medication and its safety profile. In the second part, participants will receive the dose determined from the first part, and researchers will measure the amount of dystrophin in the muscle tissue to see how well the treatment is working. The medication is given every four weeks, and the study will monitor participants over a period of time to gather comprehensive data.

Throughout the study, researchers will also keep track of any side effects or adverse events that participants may experience. This information is crucial for understanding the safety and effectiveness of Vesleteplirsen in treating Duchenne Muscular Dystrophy. The study is designed to provide valuable insights into how this treatment can potentially improve muscle function and quality of life for those affected by this condition.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria related to Duchenne Muscular Dystrophy (DMD).

Participants may have previously received Vesleteplirsen or be new to the treatment.

2 part A: dose determination

In Part A, the focus is on determining the safe and tolerable dose of Vesleteplirsen (SRP-5051).

The medication is administered through an intravenous (IV) infusion every 4 weeks.

The goal is to find the maximum dose that can be tolerated without significant side effects.

3 monitoring and assessments in part A

Participants are monitored for any adverse events over a period of up to 75 weeks.

Blood and urine samples are collected to measure the concentration of Vesleteplirsen at various times after the infusion.

4 part B: dose expansion

In Part B, the selected dose from Part A is used to evaluate its effect on dystrophin protein levels in muscle tissue.

The medication continues to be administered IV every 4 weeks.

5 monitoring and assessments in part B

The primary focus is on changes in dystrophin protein levels at Week 28.

Participants are monitored for adverse events throughout the study, up to Week 304.

Additional assessments include changes in exon-skipping levels and dystrophin-positive fibers.

Who Can Join the Study?

  • The study is for males only.
  • Participants must have a genetic diagnosis of Duchenne Muscular Dystrophy (DMD). This is a condition that affects muscles.
  • Participants should have a specific type of genetic change called an out-of-frame deletion mutation in the DMD gene. This means a part of the gene is missing, which affects how the gene works.
  • The genetic change must be suitable for a treatment called exon 51-skipping. This is a method used to help the gene work better.
  • Participants must have been on a stable dose of oral corticosteroids for at least 12 weeks before starting the study. Corticosteroids are medicines that help reduce inflammation and improve muscle strength.
  • If participants have not been on corticosteroids, they should not have taken them for at least 12 weeks before starting the study.
  • Participants must have stable lung function, with a forced vital capacity (FVC) of at least 40% of what is expected. FVC is a measure of how much air you can exhale after taking a deep breath.
  • Participants should not need a machine to help them breathe at night.
  • Participants who have previously been treated with a medication called Vesleteplirsen in Part A of this study or in a related study are eligible.

Who Cannot Join the Study?

  • Only males can participate, so females are excluded.
  • Participants must be within a specific age range, so those outside this range cannot join.
  • Individuals with certain health conditions that make them more vulnerable may not be eligible.

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
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Universitair Ziekenhuis Gent Gent Belgium
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Uklnrzcjflvadrfbkysnq Eyxsf Amu Essen Germany
Lyptt Ubofpkvwoirx Mbmpnvj Clcxgqj (dpcxm Leiden The Netherlands
Avsemrz Ocswrkonmui Unmaqelmxorxx Cqhiosnyjxjx Dqcss Swugzl E Dhaqy Scgitnw Ds Tboazm Turin Italy
Kczgpipj dvy Upahyfpcllxz Mplhwmtk Agu Munich Germany

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

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
30.04.2019
Germany Germany
Not recruiting
30.04.2019
Italy Italy
Not recruiting
30.04.2019
Spain Spain
Not recruiting
30.04.2019
The Netherlands The Netherlands
Not recruiting
30.04.2019

Trial locations

Investigated drugs:

SRP-5051 is a medication being studied for its potential to treat patients with Duchenne Muscular Dystrophy who can benefit from exon 51-skipping treatment. It is administered intravenously every four weeks. The study aims to determine the safety and tolerability of different doses of SRP-5051 and to find the maximum dose that patients can tolerate. Additionally, the study evaluates the effect of SRP-5051 on the level of dystrophin protein in skeletal muscle tissue, which is important for muscle function.

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected individuals experiencing difficulty in walking, frequent falls, and muscle stiffness. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to increased difficulty in movement. Over time, individuals may lose the ability to walk and require assistance with daily activities. The progression of muscle weakness can also impact respiratory and cardiac muscles, affecting breathing and heart function.

Trial ID:
2023-509935-23-00
Protocol code:
5051-201
NCT ID:
NCT04004065
Trial Phase:
Therapeutic exploratory (Phase II)

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