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

This article discusses the clinical trials of ATL1102, a drug being studied for the treatment of Duchenne Muscular Dystrophy (DMD) in non-ambulatory patients. ATL1102 is a 2′-O-(2-Methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA. The trials aim to evaluate the drug’s efficacy, safety, and pharmacokinetic profile in improving upper limb muscle function and other aspects of DMD patients’ health.

Table of Contents

What is ATL1102?

ATL1102 is a new medication being studied for the treatment of Duchenne Muscular Dystrophy (DMD). Its full scientific name is 2′-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA. This long name describes its chemical structure and how it works in the body.[1]

How Does ATL1102 Work?

ATL1102 is a type of drug called an antisense oligonucleotide. This means it’s designed to interfere with specific genetic messages in the body. In this case, it targets a protein called CD49d, which is involved in inflammation. By reducing CD49d, ATL1102 may help slow down the progression of muscle damage in DMD.[1]

Target Condition: Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a genetic disorder that causes progressive muscle weakness and loss of muscle mass. It primarily affects boys and typically becomes apparent in early childhood. DMD is caused by a mutation in the gene that produces dystrophin, a protein essential for maintaining muscle integrity.[1]

Clinical Trial Overview

A clinical trial is currently underway to test ATL1102 in patients with DMD. This trial is designed to evaluate how well the drug works and how safe it is. Here are some key points about the study:[1]

  • It’s a Phase II trial, which means it’s testing the drug in a larger group of patients after initial safety studies.
  • The study is randomized (patients are assigned to different groups by chance) and double-blind (neither the patients nor the doctors know who is getting the real drug or a placebo).
  • It includes an open-label extension phase where all participants receive the active drug.
  • The drug is given by subcutaneous injection (an injection under the skin) once a week.

Eligibility Criteria

To participate in this study, patients must meet certain criteria. Some key requirements include:[1]

  • Male patients aged 10 to less than 18 years
  • Confirmed diagnosis of DMD through genetic testing
  • Non-ambulatory (unable to walk 10 meters without assistance)
  • Body weight of at least 25 kg
  • Adequate heart and lung function

There are also several exclusion criteria, such as recent use of certain medications or participation in other clinical trials.

Study Objectives

The main goals of this study are:[1]

  1. To evaluate how ATL1102 affects upper limb muscle function in non-ambulant DMD patients
  2. To assess the safety and tolerability of ATL1102
  3. To study how the drug moves through the body (pharmacokinetics)
  4. To measure effects on muscle strength, respiratory function, and quality of life

Potential Benefits

If successful, ATL1102 could potentially:[1]

  • Slow down the progression of muscle weakness in DMD patients
  • Improve or maintain upper limb function
  • Enhance quality of life for patients with DMD

Safety Considerations

As with any clinical trial, patient safety is a top priority. The study includes several measures to monitor safety:[1]

  • Regular medical check-ups and laboratory tests
  • Monitoring of heart and lung function
  • A safety monitoring plan and stopping rules to quickly address any concerns
  • Careful tracking of any side effects or adverse events

It’s important to note that while ATL1102 shows promise, it is still an experimental treatment. More research is needed to fully understand its effectiveness and safety profile in treating Duchenne Muscular Dystrophy.

Aspect Details
Drug Name ATL1102 (2′-O-(2-Methoxyethyl) Phosphorothioate Antisense Oligonucleotide Targeting CD49d RNA)
Condition Duchenne Muscular Dystrophy (DMD)
Trial Phase Phase II
Study Design Multicentre, randomised, double-blind, placebo-controlled with open label extension
Primary Objectives Evaluate effect on upper limb muscle function, safety, and tolerability
Key Measurements Performance of Upper Limb Module (PUL 2.0), muscle strength, respiratory function, quality of life
Administration Subcutaneous injection, once weekly
Participant Age 10 to less than 18 years
Key Inclusion Criteria Non-ambulatory DMD patients, minimum 25 kg body weight, stable corticosteroid therapy
Duration 49 weeks (25 weeks blinded phase + 24 weeks open label extension)

FAQ

  • What is ATL1102 and how does it work? ATL1102 is a 2'-O-(2-Methoxyethyl) phosphorothioate antisense oligonucleotide that targets CD49d RNA. It is designed to potentially improve muscle function in patients with Duchenne Muscular Dystrophy by affecting the expression of certain proteins involved in the disease process.
  • Who is eligible for these clinical trials? The trials are designed for non-ambulatory male patients aged 10 to less than 18 years with a confirmed diagnosis of Duchenne Muscular Dystrophy. Participants must meet specific criteria, including a minimum body weight of 25 kg and the ability to perform certain upper limb functions.
  • What are the main objectives of the clinical trials? The main objectives are to evaluate the effect of ATL1102 on upper limb muscle function, assess its safety and tolerability, and study its pharmacokinetic profile. The trials also aim to measure impacts on muscle strength, respiratory function, and quality of life.
  • How is the drug administered in the trials? ATL1102 is administered by subcutaneous injection once weekly. The trials are testing two different dose levels of the drug.
  • What are the key measurements used in these trials? The primary measurement is the change in the Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) score. Other measurements include muscle strength tests (MyoGrip and MyoPinch), respiratory function tests, and quality of life assessments using the PedsQL Duchenne Muscular Dystrophy Module.

Ongoing Clinical Trials on 2′-O-(2-Methoxyethyl) Phosphorothioate Antisense Oligonucleotide Targeting Cd49D Rna

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

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Bulgaria

Glossary

  • Duchenne Muscular Dystrophy (DMD): A genetic disorder characterized by progressive muscle degeneration and weakness, primarily affecting boys.
  • Antisense Oligonucleotide: A synthetic strand of nucleic acid that binds to specific RNA molecules, potentially altering gene expression.
  • CD49d: A protein involved in cell adhesion and signaling, which may play a role in the progression of Duchenne Muscular Dystrophy.
  • Non-ambulatory: Unable to walk without assistance, a stage in DMD progression where patients lose the ability to walk independently.
  • Performance of Upper Limb Module (PUL 2.0): A standardized test used to assess upper limb function in patients with Duchenne Muscular Dystrophy.
  • Pharmacokinetic (PK) profile: The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Subcutaneous injection: A method of administering medication by injecting it into the layer of tissue between the skin and muscle.
  • Open Label Extension (OLE): A phase of a clinical trial where all participants receive the active treatment, often following a blinded phase.
  • MyoGrip and MyoPinch: Specialized tests used to measure muscle strength in the hands and fingers of patients with neuromuscular disorders.
  • Forced Vital Capacity (FVC): A measure of lung function that represents the total amount of air exhaled forcefully after a deep breath.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effects-of-atl1102-for-nonambulatory-patients-with-duchenne-muscular-dystrophy/