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ADS-019

Clinical trials investigating ADS-019 are studying its safety, tolerability, and how it works in adults with type 1 myotonic dystrophy. The trials also look at whether repeated doses may help, especially in people aged 18 to 65 years.

Table of contents

Trial overview

The source data includes one clinical trial for ADS-019, identified as NCT06138743, which is a Phase 1/2 interventional study in adults with type 1 myotonic dystrophy (DM1).[1] The trial is authorised and plans to enroll 90 people.[1]

The study title says it is a dose-escalating trial, which means the treatment amount is increased step by step so researchers can learn more about safety and body response.[1] The brief summary says the study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics, and it will also look at efficacy in one cohort only.[1]

Who can join the study

This trial is for subjects with type 1 myotonic dystrophy who are at least 18 years old and no older than 65 years.[1] In patient-friendly terms, the study is focused on adults within a specific age range, not children or older adults outside that range.[1]

The trial data does not list more detailed entry rules, so the available information only confirms the disease and age range.[1]

What is being measured

The main outcome is the incidence, frequency, and severity of treatment-emergent adverse events and treatment-related adverse events through the end of study.[1] In simple language, this means the researchers are tracking how often health problems happen, how serious they are, and whether they may be linked to the study treatment.[1]

The study also aims to evaluate pharmacokinetics and pharmacodynamics.[1] Pharmacokinetics means what the body does to the treatment, and pharmacodynamics means what the treatment does in the body.[1]

According to the brief summary, the study will also assess the efficacy of multiple doses in Cohort 5 only.[1] Efficacy means whether the treatment shows signs of helping with the condition being studied.[1]

Study design and treatment groups

The trial is interventional, which means researchers assign the study treatment rather than only observing what happens.[1] The interventions listed are placebo, given as sodium chloride 9 mg/ml (0.9%) solution for injection, and ADS-019 given by intravenous infusion.[1]

The presence of a placebo group helps researchers compare results fairly, because the placebo looks like treatment but does not contain the active study drug.[1] The study uses escalating single and multiple doses, which means different groups may receive different dose levels over time.[1]

What the phase means

A Phase 1/2 study is an early clinical trial stage.[1] Phase 1 usually focuses on safety and tolerability, while Phase 2 begins to explore whether the treatment may help the condition.[1]

For patients, this means the trial is mainly designed to learn how ADS-019 behaves in people with DM1 and whether it can be studied further in larger trials.[1] The available data does not provide results, so the article is based on the study plan rather than final findings.[1]

Trial ID Phase Condition studied Status Enrollment
NCT06138743 Phase 1/2 Type 1 Myotonic Dystrophy Authorised 90

FAQ

  • What is being studied in ADS-019 clinical trials? The trials are studying safety, tolerability, pharmacokinetics, and pharmacodynamics. In simple terms, they check how the study treatment behaves in the body and what effects it has.
  • Who can take part in these trials? The trial shown here includes adults with type 1 myotonic dystrophy who are 18 to 65 years old. Participation depends on the study rules.
  • What phase is the ADS-019 trial? This is a Phase 1/2 study. That means it is an early trial that looks first at safety and also explores early signs of benefit.
  • What condition is the trial focused on? The trial is focused on type 1 myotonic dystrophy, also called DM1. This is the main condition being studied in the available trial data.
  • What outcomes are measured in the trial? The main outcome is the number, type, and seriousness of treatment-emergent side effects. The study also looks at whether repeated doses may show benefit in one cohort.

Ongoing Clinical Trials on ADS-019

  • A study testing the safety and effects of ADS-019 in adults aged 18 to 65 years with type 1 myotonic dystrophy

    Not yet recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Belgium Germany Italy Spain

Glossary

  • Type 1 myotonic dystrophy (DM1): A condition being studied in this trial. The study includes adults with DM1, which is the main disease focus of the research.
  • Phase 1/2: An early stage of clinical research. Phase 1 mainly checks safety, and Phase 2 also starts to look for signs that the treatment may help.
  • Safety: Whether the study treatment causes harmful effects or serious problems.
  • Tolerability: How well people can take the study treatment without major problems.
  • Pharmacokinetics (PK): How the body absorbs, moves, and removes the study treatment over time.
  • Pharmacodynamics (PD): What the study treatment does in the body and what effects it has.
  • Dose-escalating study: A study where the amount of treatment is increased step by step to learn more about safety and effects.
  • Placebo: A look-alike treatment with no active study drug. It helps researchers compare results fairly.
  • Treatment-emergent adverse events (TEAEs): Health problems that start or get worse after treatment begins.
  • Cohort: A smaller group of participants within a study, often used for a specific dose or schedule.

References