Long‑Term Safety and Efficacy Study of ENTR‑601‑45 and ENTR‑601‑44 in Participants with Duchenne Muscular Dystrophy

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What is this study about?

The study focuses on Duchenne Muscular Dystrophy, a rare genetic condition that causes progressive muscle weakness. Participants receive an investigational medicine called ENTR-601-45 or ENTR-601-44, which are given by intravenous infusion. These drugs belong to a special class designed to help the body skip over faulty sections of the gene (a process known as exon skipping) and use a delivery system referred to as an endosomal escape vehicle phosphorodiamidate morpholino oligomer platform to reach muscle cells.

The main goal of the trial is to evaluate the long‑term safety and tolerability of the study drug in people with the condition. After an initial screening, participants receive the medication at regular intervals for an extended period, during which doctors monitor vital signs, blood tests, heart recordings, and physical examinations. Simple walking and climbing tests are performed at the start and at later visits to see how the treatment may affect mobility.

Throughout the study, blood samples are taken to measure how much of the medicine remains in the body and to check for any immune response. All observations are recorded to help determine whether the therapy is safe for continued use over time.

1 initial enrollment and baseline assessments

upon joining the study, baseline measurements are taken to record current health status. these include vital signs such as blood pressure and heart rate, basic laboratory tests, an electrocardiogram (ecg) to check heart rhythm, and a physical examination.

the purpose of these assessments is to provide reference points for later comparison during the study.

2 first intravenous infusion of entr-601-45

the participant receives a solution for infusion called entr-601-45. the dose is 20 mg/kg, meaning 20 milligrams of medication for each kilogram of body weight.

the medication is administered through an intravenous infusion, which involves delivering the drug directly into a vein over a short period of time.

3 first intravenous infusion of entr-601-44

the participant receives a second solution for infusion called entr-601-44. the dose is 18 mg/kg, meaning 18 milligrams of medication for each kilogram of body weight.

this medication is also given by intravenous infusion, using a vein as the route of delivery.

4 regular safety monitoring visits

at each scheduled visit, the study team checks for any new or worsening health problems, known as treatment‑emergent adverse events (teaes).

measurements include vital signs, repeat laboratory tests, another ecg, and a physical examination to track safety over time.

5 efficacy assessments

the participant performs simple functional tests to evaluate muscle strength and mobility. these tests include a 10‑meter walk/run, timed rise from floor, timed 4‑stair climb, stride velocity at the 95th percentile, the north star ambulatory assessment, and the performance of the upper limb version 2.0.

each test is done in a standard way so that changes can be compared to the baseline measurements.

6 blood sampling for drug levels and antibody testing

blood samples are taken to measure the concentration of the study drugs in the plasma and to detect any anti‑drug or anti‑dystrophin antibodies, which are proteins that the body might produce against the medication.

7 continuation of treatment until study end

the participant continues to receive the specified doses of entr-601-45 and entr-601-44 by intravenous infusion according to the study schedule for the duration of the long‑term extension period.

ongoing safety checks and efficacy assessments are performed throughout this time to gather long‑term data.

Who Can Join the Study?

  • Must be a male (boy) who has Duchenne muscular dystrophy.
  • Must be between the ages of 2 and 3 years.
  • Must be able to give consent (agree to join the study if he is an adult) or assent (agree to join the study if he is a child).
  • Must have already finished the earlier clinical trial called ENTR‑601‑44‑201 or ENTR‑601‑45‑201.
  • If he is sexually active with a female partner who could become pregnant, he must agree to use condoms during sexual intercourse.

Who Cannot Join the Study?

  • If there has been any change to the rules used in the earlier related study (parent study), such as a health problem that occurred during that study (safety events), and the doctor in charge of the trial (investigator) together with the safety‑watching doctor (medical monitor) or the company’s representative (sponsor designee) decides that it would not be safe (precludes safe use) for you to take the study drug, you cannot join the trial.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Katholieke Universiteit te Leuven Leuven Belgium
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy

Other Sites

Site Name City Country Status
Ospedale San Raffaele S.r.l. Milan Italy
Stichting Radboud University Medical Center Nijmegen The Netherlands
Universitair Ziekenhuis Gent Gent Belgium
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Centre Hospitalier Regional De La Citadelle Liege Belgium
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Hqvvrwum Vucg dkbikrxb Barcelona Spain
Cflmfj Ccmbato Nisl Milan Italy
Lqnvd Ufradusvsmch Mwvmnvf Ctkysyj (wwzfr Leiden The Netherlands

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

Trial status

Country Status Recruitment Start
Belgium Belgium
Not yet recruiting
26.08.2026
Italy Italy
Not yet recruiting
26.08.2026
Spain Spain
Not yet recruiting
26.08.2026
The Netherlands The Netherlands
Not yet recruiting
26.08.2026

Trial locations

ENTR-601-45 is an experimental medicine that is given through an intravenous (IV) infusion. It belongs to a group of drugs called phosphorodiamidate morpholino oligomers, which are designed to “skip” a specific part of the genetic code (an exon) that is faulty in Duchenne muscular dystrophy. By skipping this exon, the drug aims to help the body produce a healthier version of the protein dystrophin, which is missing or broken in people with this disease. In the study, the medicine is being evaluated for how safe it is to use over a long period, how well patients tolerate it, how it moves through the body, and whether it improves muscle function.

ENTR-601-44 is another experimental medicine tested in the same way, also given by IV infusion. Like ENTR-601-45, it is a phosphorodiamidate morpholino oligomer that works by skipping a different exon that can also help restore the production of functional dystrophin in Duchenne muscular dystrophy. The trial is looking at its long‑term safety, how patients feel while taking it, how the drug is processed in the body, and whether it can help maintain or improve muscle strength and overall health.

Duchenne muscular dystrophy – Duchenne muscular dystrophy is a genetic disorder that causes progressive weakening of the muscles. It appears in early childhood, often before age five, and the muscle weakness starts in the legs and pelvis. As the disease advances, the arms, neck, and respiratory muscles become affected. Children may have difficulty walking, climbing stairs, and raising from the floor. Over time, the ability to move independently decreases, and the condition can affect posture and coordination.

Trial ID:
2025-525124-10-00
Protocol code:
ENTR-601-DMD-202
Trial Phase:
Therapeutic exploratory (Phase II)

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