Study on the Effects of ATL1102 for Nonambulatory Patients with Duchenne Muscular Dystrophy

What is this study about?

This clinical trial is focused on studying a condition called Duchenne Muscular Dystrophy (DMD), which is a genetic disorder characterized by progressive muscle weakness. The study is testing a treatment called ATL1102, which is a special type of medication known as an antisense oligonucleotide. This medication is designed to target a specific part of the genetic material in the body to help improve muscle function. The treatment is given as a solution for injection under the skin.

The purpose of the study is to evaluate how well ATL1102 works in improving upper limb muscle function in participants who are unable to walk due to DMD. The study will compare the effects of the medication to a placebo. Participants will receive the treatment once a week, and the study will monitor their muscle function over time. The study will also assess the safety and tolerability of the medication to ensure it is safe for use.

The study is divided into two phases: a blinded phase, where neither the participants nor the researchers know who is receiving the actual medication or the placebo, and an open-label extension phase, where all participants will receive ATL1102. The study will last for several weeks, and participants will be regularly assessed to track changes in their muscle function and overall health. The goal is to determine if ATL1102 can help improve the quality of life for those living with Duchenne Muscular Dystrophy.

1 Joining the Study

Upon joining the study, the participant will be randomly assigned to receive either the study medication, ATL1102, or a placebo. This process is double-blind, meaning neither the participant nor the researchers know which treatment is being administered.

The study medication is administered as a solution for injection under the skin (subcutaneous use).

2 Blinded Phase

During the blinded phase, the participant will receive the assigned treatment once a week for 25 weeks.

The main goal is to evaluate the effect of ATL1102 on upper limb muscle function, measured by changes in the Performance of Upper Limb Module for Duchenne Muscular Dystrophy (PUL 2.0) score.

3 Open Label Extension (OLE) Phase

After completing the blinded phase, participants may enter the open label extension phase, where all participants receive ATL1102 once a week for an additional 24 weeks.

This phase aims to assess the continued safety and tolerability of the medication.

4 Assessments and Monitoring

Throughout the study, various assessments will be conducted, including physical examinations, laboratory tests, and evaluations of cardiac and respiratory function.

Participants’ muscle strength and quality of life will also be monitored using specific tools and questionnaires.

5 Completion of the Study

The study is expected to conclude in March 2025. Upon completion, the data collected will be analyzed to determine the efficacy and safety of ATL1102 in treating non-ambulatory participants with Duchenne Muscular Dystrophy.

Who Can Join the Study?

The participant must have a parent or guardian who understands the study’s purpose and risks and can provide written consent for the participant to join the study.
The participant must have good heart function, with a left ventricular ejection fraction (LVEF) of 45% or higher, as shown by an echocardiogram. If taking heart medication, the participant must have been on a stable dose for at least 3 months before starting the study.
If the participant is post-pubertal and sexually active, they must agree to use approved birth control methods, like condoms or abstinence, during the study and for 4 months after the last dose of the study medication. If the participant has a female partner, she must also use a medically accepted form of birth control.
The participant and their parent or guardian must be willing and able to attend scheduled visits, take the study medication, and follow study procedures.
If required by law or if the participant is mature enough to understand the study, their consent will be obtained. Otherwise, assent will be documented.
The participant must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) through genetic testing, which checks for changes in the dystrophin gene.
The participant must be non-ambulatory, meaning they cannot walk 10 meters without help, at the time of screening.
The participant must be a male aged between 10 and less than 18 years at the time of screening and consent.
The participant must weigh at least 25 kg at the time of screening.
If the participant is taking corticosteroid therapy, it must have started at least six months before the study begins, with a stable daily dose for at least 3 months before the study starts. There should be no plan to change the dose during the study, except for adjustments based on body weight.
At the start of the study, the participant must have a Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A score of 2 or higher.
The participant must be able to perform a breathing test called spirometry and have enough lung function, with a forced vital capacity (FVC) of 50% or higher. The FVC results should be consistent, with the best of three attempts at the start of the study being within 20% of the best of three attempts at screening.

Who Cannot Join the Study?

Participants who are able to walk on their own are not eligible.
Females cannot participate in this study.
Participants who have any other serious health conditions that might interfere with the study cannot join.
Participants who are unable to follow the study procedures or instructions will be excluded.
Participants who have taken certain medications that might affect the study results cannot participate.
Participants who have had a recent surgery or are planning to have surgery during the study period are not eligible.
Participants who have a history of allergic reactions to the study medication or similar medications cannot join.
Participants who are currently participating in another clinical trial are not eligible.
Participants who have a history of drug or alcohol abuse may be excluded.
Participants who have any condition that the study doctors believe would make it unsafe for them to participate will be excluded.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Diagnostic-consultative center “Aleksandrovska” EOOD	Sofia	Bulgaria

Trial status

Country	Status	Recruitment Start
Bulgaria	Not recruiting	09.11.2023

Trial locations

Investigated drugs:

2'-O-(2-Methoxyethyl) Phosphorothioate Antisense Oligonucleotide Targeting Cd49D Rna

ATL1102 is a medication being studied for its potential to improve muscle function in individuals with Duchenne Muscular Dystrophy (DMD) who are unable to walk. It is administered through a subcutaneous injection, which means it is injected under the skin. The study aims to determine how effective ATL1102 is in enhancing the function of the upper limbs in these patients. Additionally, the study will assess the safety and tolerability of ATL1102 when given once a week over an extended period.

Investigated diseases:

Duchenne muscular dystrophy

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy (DMD) 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. Symptoms typically begin in early childhood, with affected individuals experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to loss of mobility and independence. Over time, complications can arise in the heart and respiratory muscles, further impacting the individual’s health. The progression of DMD varies, but it generally leads to significant physical disability.

Trial ID:

2024-512265-13-00

Protocol code:

1102-DMD-Pre-CT03

NCT ID:

NCT05938023

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effects of ATL1102 for Nonambulatory Patients with Duchenne Muscular Dystrophy

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