Not yet recruitingPhase IIInterventional

A Phase II, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerability, pharmacokinetics, and efficacy of endosomal escape vehicle phosphorodiamidate morpholino oligomer platform products (ELEVATE-LTE)

Duchenne muscular dystrophy (DMD) is a rare genetic condition that causes muscles to weaken over time. It happens because a change in the genes stops the body from making an essential muscle-protecting protein called dystrophin. This trial is an extension study for males who have already completed an earlier trial testing either ENTR-601-44 or ENTR-601-45. These experimental medicines are designed to help the body 'skip over' the genetic error so it can produce a shorter, working version of dystrophin. There is no dummy drug (placebo) in this trial; every person will receive the active study medicine through a drip into a vein. Doctors want to monitor participants over several years to ensure the treatment remains safe and well tolerated, and to check whether it helps maintain muscle function. The study is scheduled to run between 2026 and 2032.

At a glance

Status

Not yet recruiting

Phase

Phase II

Sponsor

Entrada Therapeutics, Inc

Enrolment target

Start

28 Oct 2026

Estimated completion

18 Aug 2029

Duchenne muscular dystrophy

What is this study about?

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness. In healthy muscles, a protein called dystrophin acts like a shock absorber, protecting muscle fibres from damage during movement. In DMD, a faulty section in the genetic code prevents the body from making enough working dystrophin. Over time, this leads to muscle breakdown, making walking and everyday movements more difficult.

This research tests two experimental medicines: ENTR-601-44 and ENTR-601-45. They use an approach called 'exon skipping'. You can think of the faulty gene as a recipe book with a damaged step. Exon skipping allows the body's cells to skip past that specific damaged instruction, read the rest of the recipe, and produce a shorter but still functional version of dystrophin. This could help muscles stay stronger and work better.

This study is a 'long-term extension', meaning it is only open to people who already completed the initial trial testing these medicines. Because there is no comparison group or placebo, every participant receives the real medicine at the same dose they reached before. The main goal is to find out if the treatment is safe to use over many years and whether the benefits to muscle health continue over time.

Key takeaways

A follow-on study for males with DMD who completed an earlier trial with ENTR-601-44 or ENTR-601-45.
All participants receive the active study medicine; there is no placebo.
The treatment is given as an intravenous drip into a vein at the clinic.
The main purpose is to check long-term safety and whether the medicine helps muscle function over several years.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is only open to males of any age who have already taken part in and fully finished one of the two earlier trials: ENTR-601-44-201 or ENTR-601-45-201. If you did not participate in one of those specific studies, you will not be able to join this extension trial.

Participants must be willing to join the study and give written consent. For children and young people under the legal age of consent, a parent or legal guardian must give signed permission, and the young person will be asked for their assent. Sexually active males must also agree to use condoms to prevent any possible risks to a partner or baby.

You may not be able to join if you had significant safety issues or severe side effects during the earlier trial, or if you have any medical condition that the study doctor feels would make taking the medicine unsafe or attending visits too difficult.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

I am male.
I have fully completed either the ENTR-601-44-201 or ENTR-601-45-201 clinical trial.
The study doctor agreed it was safe for me during the previous trial.
I (or my parent/guardian) am willing to give written consent to join.
If sexually active with a female partner, I agree to use condoms.

Answer every question to see your result.

What does participation involve?

Taking part means continuing treatment with the study medicine given as a slow drip directly into a vein (an intravenous infusion). Participants will receive a set schedule of doses at the hospital clinic. During clinic visits, the medical team will carry out health checks, such as physical examinations, blood and urine tests, and muscle function assessments. These tests help doctors monitor how your body is handling the medicine. The trial is planned to run between August 2026 and March 2032, so participants may attend regular appointments over several years.

Potential risks and benefits

A potential benefit is that the medicine may help your body produce more dystrophin, which could help protect your muscle strength, although this cannot be guaranteed. Taking part also helps scientists learn more about treating DMD. However, experimental medicines carry risks of side effects. Based on earlier laboratory research and trials of similar medicines, potential risks include changes in kidney function, liver enzymes, or blood cell counts, as well as reactions to the drip. Doctors will monitor you closely at every step and can offer treatment for side effects. Taking part is entirely voluntary, and you are free to stop and leave the study at any time without giving a reason and without affecting your normal healthcare.

Locations (5)

John Radcliffe Hospital
Approximate
Oxford, England
Great Ormond Street Hospital for Children
Approximate
London, England
Royal Victoria Infirmary
Approximate
Newcastle upon Tyne, England
Alder Hey Children's Hospital
Approximate
Liverpool, England
Leeds General Infirmary
Approximate
Leeds, England

Common questions

Will anyone receive a placebo (dummy medicine)?

No. In this extension trial, there is no placebo. Everyone who takes part will receive the active study medicine.

How is the medicine given?

The medicine is given as an intravenous infusion, which means it is slowly delivered through a needle and tube directly into a vein.

Can I join if I was not in the first trial?

No. Only males who have already completed the earlier trial (ENTR-601-44-201 or ENTR-601-45-201) are eligible to take part.

Is this medicine a cure for Duchenne muscular dystrophy?

No, it is not a cure. The medicine is designed to help your body produce a working form of dystrophin to help slow down muscle weakness.

What happens if I decide to leave the trial early?

You can leave the trial at any point. Your decision will not change the standard medical care or support you receive from your regular doctors.

How to find out more

. Entrada Therapeutics Clinical Trials

clinicaltrials@entradatx.com - Official trial record

Always speak to your GP or specialist before deciding to take part in a study.