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Study of ENTR-601-44 in Children with Duchenne Muscular Dystrophy Who Are Eligible for Exon 44 Skipping Treatment

Fast replyRegistered drugNo placebo
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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 patients with Duchenne Muscular Dystrophy, a genetic condition that causes progressive muscle weakness. The research evaluates a new medication called ENTR-601-44, which is given through intravenous infusion. The purpose is to determine if this treatment is safe and effective for patients whose disease could benefit from a process called exon 44 skipping.

The study is divided into two parts and uses both the investigational medication and sodium chloride solution as placebo. During the study, participants receive either ENTR-601-44 or placebo through an infusion into their veins. The researchers will monitor the participants' health, including physical examinations, laboratory tests, and measurements of muscle strength and function.

Throughout the study, doctors will collect various types of information about how the medication affects the body, including its presence in blood, muscle tissue, and urine. They will also measure changes in a protein called dystrophin in muscle tissue and monitor the participants' ability to perform various physical activities. The study includes regular medical check-ups to ensure participant safety.

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 and confirmation

    Your eligibility for the study will be confirmed through a genetic test that shows you have Duchenne muscular dystrophy with the possibility of exon 44 skipping

    A doctor will verify that you are a male between 4-20 years of age with clinical signs of Duchenne muscular dystrophy

    Your ability to walk and use your upper limbs will be assessed using a special test called Performance of the Upper Limb (PUL 2.0)

    A doctor will check if you have adequate muscle tissue for biopsy procedures

  2. Step 2

    Part A - Initial treatment phase

    You will receive either ENTR-601-44 or a placebo through an intravenous infusion (delivery of medicine through a vein)

    Regular health checks will be performed, including:

    - Vital signs measurements

    - Blood tests

    - Heart tests (ECG)

    - Physical examinations

    Samples will be collected to measure drug levels in your blood, muscle tissue, and urine

    A muscle biopsy will be taken to examine changes in a protein called dystrophin

  3. Step 3

    Physical ability assessments

    Throughout the study, your physical abilities will be tested through several exercises:

    - 10-meter walk/run test

    - Rising from the floor test

    - 4-stair climb test

    - Walking speed measurement

    - Upper limb function assessment

  4. Step 4

    Open-label period

    After Part A, you will continue receiving treatment

    Regular monitoring of your health and physical abilities will continue

    The study is expected to continue until January 2027

Who can join the trial?

6 criteria

  • You must have a confirmed genetic diagnosis of Duchenne muscular dystrophy with a specific gene variant that can be treated with exon 44 skipping (this will be reviewed by a genetics specialist)
  • You must be male at birth and show clinical signs of Duchenne muscular dystrophy as determined by the study doctor
  • Your age must be between 4 and 20 years old
  • You must be able to walk and perform certain arm and hand movements as measured by a special test called Performance of the Upper Limb (PUL 2.0) during the screening visit
  • You must have enough muscle tissue suitable for a biopsy (a small sample of muscle tissue taken for testing) as determined by the study doctor
  • You must meet additional study requirements that will be reviewed by the study team

Who cannot join the trial?

14 criteria

  • History of allergic reactions or severe sensitivity to any of the study medications
  • Participation in another clinical trial within the past 30 days
  • Significant heart problems or abnormal heart function tests
  • Severe liver problems or abnormal liver function tests
  • Severe kidney problems or abnormal kidney function tests
  • Current use of medications that could interfere with the study drug
  • History of blood disorders or significant bleeding problems
  • Presence of other serious medical conditions that could affect study participation
  • Unable to follow study procedures or attend scheduled visits
  • Mental health conditions that could affect ability to participate safely
  • Current drug or alcohol abuse
  • Pregnancy, breastfeeding, or planning to become pregnant during the study period
  • History of non-compliance with medical treatments
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
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Investigated drugs

ENTR-601-44 is an investigational drug being studied for the treatment of Duchenne muscular dystrophy (DMD), specifically in patients whose condition could benefit from exon 44 skipping. This medication is designed to help produce a form of dystrophin protein that is missing in these patients. It aims to potentially slow down the progression of the disease by allowing the body to produce a shortened but partially functional version of the dystrophin protein.

What is already known about the treatment

ENTR-601-44 - An investigational therapeutic compound specifically designed for treating Duchenne Muscular Dystrophy (DMD) in patients who are amenable to exon 44 skipping. This medication is currently undergoing clinical trials to evaluate its safety, tolerability, and effectiveness in treating DMD through a mechanism known as exon skipping, which aims to partially restore dystrophin protein production in muscle cells. The drug is being studied in a two-part clinical trial, with Part A focusing on safety and pharmacokinetics assessment through multiple ascending doses, while Part B evaluates its overall safety and therapeutic efficacy. While the specific form of administration is not explicitly stated in the trial data, similar exon-skipping therapies for DMD are typically administered through regular injections.

Investigated diseases

Duchenne Muscular Dystrophy - A genetic disorder characterized by progressive muscle degeneration and weakness that primarily affects males. The condition begins in early childhood, typically between ages 2 and 6, when parents may notice their child having difficulty running, jumping, or climbing stairs. It is caused by mutations in the gene that produces dystrophin, a protein essential for proper muscle function. The disease causes muscles to gradually weaken over time, affecting first the muscles close to the trunk of the body, then moving to the arms and legs. The muscle weakness affects mobility and various daily activities, and children with this condition often require mobility support as the disease progresses.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-517584-23-00Protocol codeENTR-601-44-201Estimated enrolment41 patientsSponsorEntrada Therapeutics Inc.

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