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Study on the Safety and Effects of SQY51 for Children and Adults with Duchenne Muscular Dystrophy

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What is this trial about?

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

This clinical trial is focused on studying Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The trial will evaluate a new treatment called SQY51, which is a type of medication known as a palmitoyl-conjugated tricyclo-DNA antisense oligonucleotide. This treatment is administered as a solution for injection and is designed to target specific genetic mutations associated with DMD.

The purpose of the study is to gather information on the safety and tolerability of SQY51 in both children and adults who have a genetically confirmed diagnosis of DMD. The study is divided into two phases: a 13-week Phase 1, where multiple doses of the treatment will be given to understand how the body processes the drug, and a 32-week Phase 2a, which will continue to monitor the effects of the treatment over a longer period. Participants will receive the treatment through intravenous use, meaning it will be delivered directly into the bloodstream.

Throughout the study, various health assessments will be conducted to monitor the participants' response to the treatment. These assessments will include checking for any adverse events, conducting physical examinations, and performing tests such as electrocardiograms (ECGs) to evaluate heart function. Additionally, the study will measure motor function, respiratory function, and muscle strength to understand the impact of SQY51 on the symptoms of DMD. The study will also analyze biomarkers in the blood and muscle tissue to gain further insights into the treatment's effects. The trial is expected to conclude in 2025.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Beginning of the trial
Upon joining the study, you will begin the Phase 1 of the trial, which lasts for 13 weeks. This phase involves multiple doses of the study medication, SQY51, which is administered as a solution for injection through a vein (intravenous use).
During this phase, the dosage of SQY51 may be gradually increased to determine the safest and most effective dose for you.
Step 2
Phase 1 activities
Throughout Phase 1, you will have regular visits to the study site. These visits will include physical examinations, blood tests, and other assessments to monitor your health and how your body responds to the medication.
You will also undergo tests to check your heart function using electrocardiograms (ECGs) and other safety laboratory tests.
Step 3
Transition to phase 2a
If you complete Phase 1 successfully, you will move on to Phase 2a of the trial, which lasts for 32 weeks. This phase continues to evaluate the safety and effects of SQY51.
The dosage and frequency of the medication will be based on the findings from Phase 1.
Step 4
Phase 2a activities
During Phase 2a, you will continue to receive SQY51 through intravenous injections. Regular visits to the study site will be necessary for ongoing monitoring.
Assessments will include tests of motor function, respiratory function, and cardiac function, as well as measurements of muscle strength and quality of life evaluations.
Step 5
End of the trial
At the end of Phase 2a, the trial will conclude. Final assessments will be conducted to gather comprehensive data on your health and the effects of the medication.
The study is expected to end by July 2025, and you will be informed about the results and any further steps if necessary.

Who can join the trial?

20 criteria

Boys who are at least 6 years old and weigh at least 16 kg.
Must be affiliated with a Social Security system.
An informed consent form must be signed by the patient or, if the patient is a minor, by their legal guardian(s).
For the phase 2a study: Must have completed Phase 1 of the study.
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Can be either able to walk or not able to walk:
- Ambulatory stage: Able to stand up from the floor and walk 10 meters without help (at least 4 patients needed).
- Early non-ambulatory stage: Unable to walk 10 meters without help, including human assistance. Loss of ability to walk (LoA) for 5 years or less before joining the study (up to 8 patients).
- Late non-ambulatory stage: LoA for more than 5 years before joining the study (up to 3 patients).
- Patients and, if minor, their legal guardians must be willing and able to attend scheduled visits, follow the treatment plan, and complete tests and other study procedures.
- Must have a diagnosis of Duchenne muscular dystrophy (DMD), confirmed by genetic testing that shows mutations suitable for exon-51 skipping.
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  Must have stable liver and kidney function:
  - GGT (a liver enzyme) less than 1.5 times the upper limit of normal (ULN).
  - AST (a liver enzyme) less than 25 times ULN.
  - ALT (a liver enzyme) less than 15 times ULN.
  - Total bilirubin less than 1.5 times ULN.
  - Alkaline phosphatase less than 1.5 times ULN.
  - Estimated glomerular filtration rate (a measure of kidney function) at least 90 mL/min/1.73m².
  - Proteinuria and microalbuminuria (protein in urine) within normal limits.
  - Echocardiography (heart ultrasound) showing left ventricular ejection fraction (LVEF) of at least 40% at screening.
  - Must be on regular treatment with corticosteroids (such as Prednisone or Deflazacort) for at least three months before joining the study. The treatment should remain stable during the study, but adjustments can be made based on weight changes.
  - If needed, must be on approved treatments for DMD according to care guidelines, such as medications for blood pressure, heart health, cholesterol, thyroid, vitamins, minerals, stomach protection, and nutritional supplements. These treatments must be stable for at least one month before joining the study and can be adjusted as needed during the study.
  - Use of non-invasive mechanical ventilation at night is allowed if it is used for less than 16 hours per day.

Who cannot join the trial?

4 criteria

Patients who do not have Duchenne muscular dystrophy cannot participate.
Only male patients are eligible; female patients cannot participate.
Patients who are not within the specified age range cannot participate. The age range for this study is not specified in the provided data.
Patients who are considered part of a vulnerable population may not be eligible. This typically refers to groups who may have difficulty giving informed consent or are at higher risk of harm.

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Raymond-Poincare Hospital
Garches, France

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Investigated drugs

SQY51 is a medication being studied for its potential to help people with Duchenne Muscular Dystrophy, a genetic disorder that affects muscles. This medication is being tested to see if it is safe and how it behaves in the body. The study aims to understand if SQY51 can be tolerated by patients and if it might help in managing the symptoms of this muscle condition.

Sqy51

What is already known about the treatment

SQY51 – SQY51 is administered as an injection and is currently being studied in clinical trials for its safety and effectiveness in treating Duchenne muscular dystrophy, a genetic disorder that causes muscle weakness. The medication is in the early stages of research, specifically in Phase 1/2a trials, which aim to gather information on its safety and how the body processes it. SQY51 is designed to help improve muscle function by targeting specific genetic components involved in the disease. It falls under the category of experimental drugs, as it is still being tested and is not yet approved for general medical use.

Investigated diseases

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. The disease typically begins in early childhood, with symptoms such as difficulty walking, frequent falls, and challenges in climbing stairs. As the condition progresses, muscle weakness spreads to the arms, neck, and other areas, leading to loss of mobility. Over time, respiratory and cardiac muscles may also be affected, impacting breathing and heart function. The progression of muscle weakness varies among individuals, but it generally leads to significant physical disability.