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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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 trial?

6 criteria

  • 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 trial?

12 criteria

  • 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
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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.

What is already known about the treatment

ENTR-601-45 - An investigational therapeutic agent designed for treating Duchenne Muscular Dystrophy (DMD) in patients who are amenable to exon 45 skipping. This medication is currently being studied in clinical trials to assess its safety, tolerability, and effectiveness in DMD treatment. It belongs to the class of exon-skipping therapies, which work by allowing the body's cellular machinery to 'skip over' faulty sections of genetic code, potentially enabling the production of a partially functional dystrophin protein. The medication is being evaluated through a two-part clinical trial process, with Part A focusing on safety assessment and dose determination, while Part B aims to establish its therapeutic efficacy in DMD patients.

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-517499-39-00Protocol codeENTR-601-45-201Estimated enrolment38 patientsSponsorEntrada Therapeutics Inc.

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