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16-Base Single Stranded Rna Targeting Mir-23B Linked To Oleic Acid

A new drug called ATX-01 is being studied in clinical trials for the treatment of Myotonic Dystrophy Type 1 (DM1), a genetic disorder affecting muscle function. This article explores the ongoing Phase 1/2a clinical trial investigating the safety, effectiveness, and potential benefits of ATX-01 for adults with DM1. The study aims to evaluate how well patients tolerate the drug and its effects on various aspects of the condition, including muscle strength and daily activities.

Table of Contents

What is ATX-01?

ATX-01 is a new medication being developed to treat Myotonic Dystrophy Type 1 (DM1). It’s classified as a novel synthetic antisense oligonucleotide, which is a type of drug that targets specific genetic sequences[1]. ATX-01 is also known by its scientific name: 16-base single stranded RNA targeting miR-23b linked to oleic acid[1].

Target Condition: Myotonic Dystrophy Type 1 (DM1)

Myotonic Dystrophy Type 1 (DM1) is a genetic disorder that affects multiple body systems. It’s characterized by muscle weakness, difficulty relaxing muscles (myotonia), and various other symptoms. DM1 is caused by a specific genetic mutation in the DMPK gene[1].

How ATX-01 Works

ATX-01 is designed to target a specific genetic sequence related to DM1. It aims to modify the expression of certain genes involved in the disease process. While the exact mechanism isn’t fully explained in the available information, antisense oligonucleotides typically work by binding to specific RNA molecules, potentially altering how genes are expressed or proteins are produced[1].

Current Research on ATX-01

ATX-01 is currently being studied in a Phase 1/2a clinical trial. This type of study is designed to assess the safety, tolerability, and early signs of effectiveness of the drug. The study is divided into two parts[1]:

  1. Single Ascending Dose (SAD) part: Participants receive a single dose of ATX-01 or placebo.
  2. Multiple Ascending Dose (MAD) part: Participants receive multiple doses of ATX-01 or placebo over time.

Potential Benefits of ATX-01

While it’s important to note that the effectiveness of ATX-01 is still being studied, researchers are investigating its potential to improve several aspects of DM1, including[1]:

  • Reducing myotonia (difficulty relaxing muscles)
  • Improving ankle dorsiflexion strength (ability to lift the foot upwards)
  • Enhancing activities of daily living, including mobility, upper extremity function, breathing, stamina, and gastrointestinal symptoms

Who Can Participate in the ATX-01 Study?

The study is looking for adults aged 18 to 64 with a confirmed diagnosis of DM1. Some key eligibility criteria include[1]:

  • Ability to walk without assistive devices (except ankle-foot orthoses)
  • Presence of grip myotonia (difficulty relaxing hand muscles)
  • Body Mass Index (BMI) less than 35 kg/m²
  • No severe heart conditions or other significant health issues

There are additional specific criteria that doctors use to determine if someone is eligible for the study.

Study Details

The ATX-01 study is conducted in two parts[1]:

  1. Single Ascending Dose (SAD) part: Participants receive a single dose of ATX-01 or placebo. The dose starts at 16 mg and may increase up to 128 mg in different groups.
  2. Multiple Ascending Dose (MAD) part: Participants receive three doses of ATX-01 or placebo, given once every two weeks. The starting dose is planned to be 32 mg, potentially increasing to 128 mg in later groups.

Both parts of the study are “double-blind,” meaning neither the participants nor the doctors know who is receiving ATX-01 or placebo[1].

Safety Monitoring

Participant safety is a top priority in this study. The researchers will closely monitor participants for any side effects or adverse reactions. Some of the safety measures include[1]:

  • Regular check-ups and laboratory tests
  • Monitoring of heart function through ECGs and other tests
  • Assessment of kidney function
  • Evaluation of mental health status

A special committee called the Data Safety Monitoring Board (DSMB) will review the safety data regularly to ensure participant well-being[1].

Aspect Details
Study Drug ATX-01 (16-base single stranded RNA targeting miR-23B linked to oleic acid)
Condition Studied Myotonic Dystrophy Type 1 (DM1)
Study Design Phase 1/2a, double-blind, placebo-controlled, single and multiple ascending dose study
Main Objectives Evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical efficacy
Participant Age Range 18 to 64 years
Administration Method Intravenous injection/infusion
Study Duration Approximately 13 weeks (SAD) and 17 weeks (MAD) per participant
Key Assessments Safety monitoring, muscle biopsies, strength tests, daily activity questionnaires

FAQ

  • What is ATX-01? ATX-01 is a new drug being tested for the treatment of Myotonic Dystrophy Type 1 (DM1). It is a synthetic antisense oligonucleotide, which is a type of molecule designed to target specific genetic material in the body.
  • What is the main goal of this clinical trial? The main goal of this clinical trial is to evaluate the safety and tolerability of ATX-01 in adult participants with DM1. The study also aims to assess how the drug works in the body and its potential effectiveness in treating DM1 symptoms.
  • Who can participate in this clinical trial? The trial is open to adults aged 18 to 64 with a confirmed diagnosis of DM1. Participants must be able to walk without assistance and have certain levels of muscle function. There are specific health criteria that participants must meet to be eligible for the study.
  • How is ATX-01 administered in the trial? ATX-01 is administered intravenously (through a vein) in the trial. The study includes both single-dose and multiple-dose parts, with different dosage levels being tested.
  • What kind of tests and procedures are involved in the trial? The trial involves various tests and procedures, including blood tests, urine tests, heart monitoring (ECGs), muscle strength tests, and muscle biopsies. Participants will also complete questionnaires about their daily activities and symptoms.

Ongoing Clinical Trials on 16-Base Single Stranded Rna Targeting Mir-23B Linked To Oleic Acid

  • Study on ATX-01 for Adults Aged 18-64 with Myotonic Dystrophy Type 1 (DM1)

    Recruiting

    1
    Investigated diseases:
    Investigated drugs:
    France Italy The Netherlands Spain

Glossary

  • Myotonic Dystrophy Type 1 (DM1): A genetic disorder that affects muscle function, causing muscle weakness and difficulty relaxing muscles after use.
  • Clinical Trial: A research study that tests how well new medical approaches work in people, such as new ways to prevent, detect, or treat diseases.
  • Phase 1/2a Trial: An early stage of clinical research that focuses on testing a drug's safety, determining the right dose, and looking for early signs of effectiveness.
  • Intravenous (IV): A method of giving medications or fluids directly into a vein.
  • Placebo: A substance that looks like the study drug but contains no active ingredient, used to compare the effects of the real drug.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including how it's absorbed, distributed, metabolized, and eliminated.
  • Pharmacodynamics (PD): The study of how a drug affects the body, including its mechanism of action and the relationship between drug concentration and effect.
  • Muscle Biopsy: A medical procedure where a small sample of muscle tissue is removed for examination.
  • Myotonia: A symptom of DM1 where muscles have difficulty relaxing after contraction.
  • Adverse Event (AE): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.

References

  1. http://clinicaltrials.eu/trial/study-on-atx-01-for-adults-aged-18-64-with-myotonic-dystrophy-type-1-dm1/