Study of ENTR-601-45 Safety and Effectiveness in Patients with Duchenne Muscular Dystrophy Who Can Skip Exon 45

2 1 1

What is this study about?

This study focuses on Duchenne Muscular Dystrophy, a genetic condition that causes progressive muscle weakness and primarily affects males. The study will test a new medication called ENTR-601-45, which is given through intravenous infusion (delivered directly into a vein). Some participants will receive the study medication while others will receive a placebo (inactive substance).

The purpose of this research is to determine if ENTR-601-45 is safe and well-tolerated in people with Duchenne Muscular Dystrophy who have a specific genetic change that can be treated by skipping a part of the gene called exon 45. The study is divided into two parts, with the first part testing different dose levels of the medication to understand how it works in the body and how safe it is.

During the study, participants will receive regular infusions of either ENTR-601-45 or placebo. The research team will monitor the participants’ health through various tests, including muscle tissue samples to see how the treatment affects the muscles. Neither the participants nor the study doctors will know who is receiving the actual medication or the placebo during the study period.

1 Initial screening

A genetic test confirms if you have Duchenne muscular dystrophy that can be treated with exon 45 skipping

Medical evaluation confirms clinical signs of the condition

Assessment of upper limb function using a specific test called Performance of the Upper Limb v2.0

Evaluation of muscle tissue suitability for biopsy

2 Treatment administration

You will receive either ENTR-601-45 or a placebo through an intravenous infusion

The treatment assignment is random and neither you nor the doctor will know which treatment you receive

The treatment will be given as a solution directly into your vein

3 Monitoring and tests

Regular blood samples to measure medication levels in your plasma

Collection of urine samples to track medication levels

Muscle tissue samples (biopsies) will be taken to measure: dystrophin protein levels, exon 45 skipping, and medication concentration

Blood tests to check for antibodies against the medication and dystrophin

4 Final evaluation

Comparison of muscle biopsy results from the start and end of the study

Final safety assessment

The study is expected to continue until October 30, 2028

Who Can Join the Study?

  • Must have a confirmed genetic test showing Duchenne muscular dystrophy with a specific gene change that can be treated with exon 45 skipping therapy (this will be reviewed by a genetics expert)
  • Must be born male and show typical signs of Duchenne muscular dystrophy as determined by the study doctor
  • Must be between 4 and 20 years old for Part A of the study
  • Must be able to walk independently and achieve a specific score on the Performance of Upper Limb test (a test that measures arm and hand function)
  • Must have enough muscle tissue suitable for taking a small sample (biopsy) as determined by the study doctor
  • Must be able to meet other study requirements that will be explained by the study team

Who Cannot Join the Study?

  • History of allergic reactions or severe sensitivity to any of the study medications or similar compounds
  • Presence of any significant medical condition that could interfere with the study, including severe heart, liver, or kidney problems
  • Current participation in other clinical trials or use of investigational drugs within 30 days before starting this study
  • Any major surgery in the past 3 months
  • Inability to follow study procedures or attend scheduled visits
  • Previous gene therapy treatment for Duchenne Muscular Dystrophy
  • Serious mental health conditions that could affect the ability to provide informed consent or comply with study procedures
  • Active infections or other serious medical conditions that could affect safety
  • Use of medications that could interact with the study drug
  • Abnormal laboratory test results that indicate significant health issues
  • History of substance abuse within the past year
  • Pregnancy, breastfeeding, or planning to become pregnant during the study period

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
Centre Hospitalier Regional De La Citadelle Liege Belgium
Ospedale San Raffaele S.r.l. Milan Italy
Stichting Radboud University Medical Center Nijmegen The Netherlands
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Lfcaa Urfqjquyxmja Mdyzvtn Csrqbgo (zcvzn Leiden The Netherlands
Hawbcqoo Vrqp dkqglisk Barcelona Spain

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

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
30.06.2025
Italy Italy
Recruiting
30.06.2025
Spain Spain
Recruiting
30.06.2025
The Netherlands The Netherlands
Recruiting
30.06.2025

Trial locations

Investigated drugs:

ENTR-601-45 is an investigational medication designed for people with Duchenne muscular dystrophy (DMD) who may benefit from exon 45 skipping. This medication is being studied to see if it can help treat DMD by modifying how the body processes genetic information related to muscle function. The treatment aims to help produce a form of dystrophin protein that is needed for proper muscle function in patients with DMD.

Investigated diseases:

Duchenne Muscular Dystrophy – A genetic disorder characterized by progressive muscle degeneration and weakness that primarily affects male children. The condition begins in early childhood, typically between ages 2 and 3, when children start showing difficulty in walking, running, and climbing stairs. It is caused by mutations in the gene that produces dystrophin, a protein necessary for proper muscle function. The muscle weakness begins in the legs and pelvis, then gradually moves to the arms, neck, and other areas. The muscles become increasingly weaker over time as muscle tissue is gradually replaced by fat and scar tissue.

Trial ID:
2024-517499-39-00
Protocol code:
ENTR-601-45-201
Trial Phase:
Phase I and Phase II (Integrated) – First administration to humans

Other Trials to Consider

  • Study of SAT-3247 Compared to Placebo for Patients with Duchenne Muscular Dystrophy Who Can Walk

    Recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Belgium Poland Spain
  • Study of ENTR-601-44 in Children with Duchenne Muscular Dystrophy Who Are Eligible for Exon 44 Skipping Treatment

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Italy Spain