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Study on Long-term Safety and Efficacy of Cipaglucosidase Alfa and Miglustat for Adults with Late-onset Pompe Disease

Verified siteInvestigationalNo placebo
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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 the long-term safety and effectiveness of a treatment for Pompe disease, specifically in adults with the late-onset form of the disease. Pompe disease is a rare genetic disorder that affects muscle function due to the buildup of a complex sugar called glycogen in the body's cells. The treatment being studied involves two medications: ATB200 and AT2221. ATB200 is administered through an intravenous (IV) injection, which means it is given directly into a vein, while AT2221 is taken orally in the form of a capsule. The purpose of this study is to assess how safe and tolerable these medications are when used together over a long period.

Participants in the study will receive the treatment and be monitored for any side effects or changes in their health. The study will track various health indicators, such as the distance a participant can walk in six minutes, muscle strength, and overall physical function. These measures help researchers understand how the treatment affects the participants' ability to perform daily activities and their overall quality of life. The study will also look at any potential immune reactions to the treatment, as well as changes in certain blood and urine markers that are related to Pompe disease.

The study is designed to last for several years, allowing researchers to gather comprehensive data on the long-term effects of the treatment. Participants will have regular check-ups and assessments to ensure their safety and to evaluate the treatment's impact on their condition. This research aims to provide valuable insights into the management of Pompe disease and potentially improve treatment options for those affected by this condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant must have completed a previous study, ATB200-03. If the participant had to leave the previous study due to reasons unrelated to the treatment, such as an accident or emergency surgery, they may still be eligible to join this study.

    Participants must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of the study drug.

  2. Step 2

    Treatment administration

    The study involves the administration of two medications: ATB200 and AT2221.

    ATB200 is given as an intravenous infusion. This means it is administered directly into a vein.

    AT2221 is taken orally in the form of a hard gelatin capsule.

  3. Step 3

    Monitoring and assessments

    Throughout the study, the participant's health and response to the treatment will be closely monitored.

    Assessments will include checking for any side effects, changes in muscle strength, and overall physical well-being.

    Specific tests will measure walking distance, muscle function, and breathing effort, among other health indicators.

  4. Step 4

    Study duration

    The study is expected to continue until December 31, 2027.

    Participants will be involved in regular assessments to evaluate the long-term safety and effectiveness of the treatment.

Who can join the trial?

3 criteria

  • The person must provide signed informed consent before any study-related procedures are done. If the person is under 20 years old, they must provide written informed consent.
  • The person must have completed Study ATB200-03. If they had to leave Study ATB200-03 for reasons not related to the effectiveness or safety of the study drugs (like being hospitalized for a car accident, the COVID-19 pandemic, or emergency surgery) and missed several doses, they might still be able to join this study with approval from the Amicus medical monitor.
  • Female participants who can have children and male participants must agree to use medically accepted methods of birth control during the study and for 90 days after the last dose of the study drug.

Who cannot join the trial?

4 criteria

  • Patients who are not adults cannot participate. This means only people who are 18 years or older can join.
  • Patients who do not have **Late Onset Pompe Disease (LOPD)** cannot participate. LOPD is a condition that affects muscles and movement.
  • Patients who are not able to safely take the study medications, **ATB200/AT2221**, cannot participate. This is to ensure the safety of all participants.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate. This is to make sure they are not put at risk.
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Investigated drugs

  • ATB200

    is a medication given through an intravenous infusion. It is being studied for its potential to help people with late-onset Pompe disease, a condition that affects muscle strength and function. The medication aims to improve the breakdown of certain substances in the body that can build up and cause muscle damage.

  • AT2221

    is an oral medication that is taken by mouth. It is used in combination with ATB200 to enhance its effects. The goal of AT2221 is to help improve the overall treatment outcomes for individuals with late-onset Pompe disease by supporting the action of ATB200.

What is already known about the treatment

  • ATB200

    This medication is administered intravenously and is currently being studied in clinical trials for its long-term safety and efficacy in treating adult subjects with Late-Onset Pompe Disease (LOPD). It is not yet widely available in medical practice, as it is still under investigation. ATB200 is designed to replace the deficient enzyme in patients with Pompe disease, helping to break down glycogen accumulation in cells. It belongs to the class of enzyme replacement therapies.

  • AT2221

    This medication is taken orally and is used in combination with ATB200 in clinical trials to enhance the treatment of Late-Onset Pompe Disease (LOPD) in adults. It is still in the research phase and not yet approved for general medical use. AT2221 works by stabilizing the enzyme provided by ATB200, improving its effectiveness in breaking down glycogen. It is classified as a pharmacological chaperone.

Investigated diseases

Pompe Disease, Late Onset – This is a genetic disorder characterized by the buildup of glycogen in the body's cells, leading to muscle weakness and respiratory issues. It typically manifests in adulthood, with symptoms gradually worsening over time. Individuals may experience difficulty walking, climbing stairs, and performing other physical activities due to muscle weakness. Respiratory muscles can also be affected, leading to breathing difficulties. The progression of the disease varies among individuals, with some experiencing a slower decline in muscle function.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-505170-15-00Protocol codeATB200-07Estimated enrolment119 patientsSponsorAmicus Therapeutics Inc.

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