Study of DYNE-251 safety and effectiveness in patients with Duchenne muscular dystrophy who are candidates for exon 51 skipping treatment

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What is this study about?

This study focuses on Duchenne Muscular Dystrophy, a genetic condition that causes progressive muscle weakness. The research evaluates a new medication called DYNE-251, which is given through intravenous infusion. Some participants will receive DYNE-251 while others will receive a placebo solution.

The main purpose of this research is to test how safe DYNE-251 is and how well patients tolerate it when given multiple doses. The study will also measure the levels of a protein called dystrophin in muscle tissue after treatment. This protein is important because people with Duchenne Muscular Dystrophy don’t make enough of it.

During the study, participants will receive regular infusions of either DYNE-251 or placebo. The treatment schedule will vary, with some participants receiving treatment every four weeks and others every eight weeks. The study includes various health checks and muscle tissue examinations to monitor how well the treatment is working. Doctors will track any changes in muscle function and strength throughout the study period.

1 Initial treatment phase

You will receive DYNE-251 or placebo (5% dextrose solution) through an intravenous infusion.

The treatment will be administered either every 4 weeks or every 8 weeks, depending on your assigned group.

Your glucocorticoid medication will continue at the same stable dose you have been taking.

2 First muscle biopsy

A sample of muscle tissue will be taken from your upper arm to measure baseline levels of dystrophin protein.

This procedure will be performed before starting the treatment.

3 Treatment monitoring at week 25

A second muscle biopsy will be taken to measure changes in dystrophin protein levels.

Blood tests will be conducted to monitor CK levels (an indicator of muscle health).

Your physical function will be assessed using specific tests that measure upper limb strength and movement.

4 Extended monitoring phase

For participants receiving treatment every 8 weeks, an additional muscle biopsy will be performed at week 49.

Regular assessments will continue up to week 145, including:

Blood tests to monitor CK levels

Tests to measure breathing function (forced vital capacity)

Physical function tests for those who can walk

Blood samples to check how the medication is processed by your body

5 Safety monitoring

Throughout the study, you will be monitored for any side effects or reactions to the treatment.

Your heart function will be regularly assessed.

Blood tests will check for antibodies against the study medication.

Who Can Join the Study?

Must be male between 4 and 16 years old at the time of providing consent
Must have a confirmed diagnosis of Duchenne muscular dystrophy with a specific genetic mutation that can be treated with exon 51 skipping
Must have an upper arm muscle group suitable for taking a tissue sample (biopsy)
Must have a Brooke Upper Extremity Scale score of 1 or 2 (this measures arm and shoulder function)
Can be either able to walk or not able to walk. If not able to walk, this must have occurred within the last 2 years
Must be taking glucocorticoids (steroid medications) at a stable dose for at least 12 weeks before starting the study
Must have good heart function, shown by:
- Heart pumping strength (ejection fraction) of 50% or higher on ultrasound, or
- Heart pumping strength of 55% or higher on MRI scan

Who Cannot Join the Study?

Male patients who are not between 4 and 17 years old
Female patients (study is only for male participants)
Patients who do not have a confirmed genetic diagnosis of Duchenne muscular dystrophy
Patients who cannot undergo multiple intravenous (IV) injections (injections into a vein)
Patients who cannot provide muscle tissue samples for dystrophin protein testing (dystrophin is a protein that helps keep muscle cells intact)
Patients who are currently participating in other clinical trials
Patients who cannot comply with study visits and procedures
Patients with any medical conditions that could interfere with study assessments
Patients who cannot provide informed consent or whose legal guardian cannot provide consent
Patients with known allergies to the study medication or its components

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
IRCCS Istituto Giannina Gaslini	Genoa	Italy
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
Children’s Health Ireland	Dublin	Ireland
Cfjhct Corqgkz Nekp	Milan	Italy
Hxqcsgkk Vocp dmernjpr	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	30.01.2023
Ireland	Not recruiting	30.01.2023
Italy	Not recruiting	30.01.2023
Spain	Not recruiting	30.01.2023

Trial locations

Investigated drugs:

Fragment Antibody Targeting Human Tfr1 Conjugated To Phosphorodiamidate Morpholino Oligomer

DYNE-251 is a medication being studied for the treatment of Duchenne Muscular Dystrophy (DMD). It is administered through intravenous (IV) injection. This medication is designed to work specifically in patients whose DMD condition could be improved through a process called exon 51 skipping. The treatment aims to help the body produce dystrophin, a protein that is essential for proper muscle function and is typically lacking in patients with DMD.

Investigated diseases:

Duchenne muscular dystrophy

Duchenne muscular dystrophy – A genetic disorder that causes progressive muscle degeneration and weakness. It primarily affects boys and typically becomes apparent in early childhood. The condition is characterized by gradual weakening of muscles, starting in the legs and pelvis, and later affecting the whole body. Children with this condition often have difficulty with basic movements like walking, running, and jumping. The muscle weakness progresses steadily over time, affecting different muscle groups throughout the body. The condition occurs due to mutations in the gene that produces dystrophin, a protein necessary for proper muscle function.

Trial ID:

2023-510351-31-00

Protocol code:

DYNE251-DMD-201

NCT ID:

NCT05524883

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study of DYNE-251 safety and effectiveness in patients with Duchenne muscular dystrophy who are candidates for exon 51 skipping treatment

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?