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Cipaglucosidase Alfa

Cipaglucosidase Alfa is an investigational enzyme replacement therapy being studied in clinical trials for the treatment of Pompe disease, a rare genetic disorder affecting muscle function. These trials aim to evaluate the safety, efficacy, and long-term effects of Cipaglucosidase Alfa in both children and adults with different forms of Pompe disease, including infantile-onset and late-onset variants. The studies are designed to assess various aspects of the treatment, including its impact on muscle strength, respiratory function, and overall quality of life for patients.

Table of Contents

What is Cipaglucosidase Alfa?

Cipaglucosidase alfa, also known as ATB200, is an investigational enzyme replacement therapy (ERT) being developed for the treatment of Pompe disease[1]. It is a new form of the enzyme acid alpha-glucosidase (GAA), which is deficient in people with Pompe disease. This medication is being studied as a potential improvement over existing treatments for this rare genetic disorder.

How Does It Work?

Cipaglucosidase alfa works by replacing the deficient GAA enzyme in patients with Pompe disease. This enzyme is crucial for breaking down a complex sugar called glycogen in the body’s cells. In Pompe disease, glycogen builds up in various tissues, particularly in the muscles, causing progressive weakness and other health problems[2].

The medication is typically co-administered with another drug called miglustat. This combination is designed to enhance the effectiveness of the enzyme replacement therapy[3].

What Conditions Does It Treat?

Cipaglucosidase alfa is being developed to treat Pompe disease, which has two main forms:

  • Infantile-onset Pompe disease (IOPD): This is the most severe form, typically diagnosed in infants under one year of age. It is characterized by severe muscle weakness, heart problems, and respiratory difficulties[1].
  • Late-onset Pompe disease (LOPD): This form can be diagnosed any time after the first year of life, including in adults. It generally progresses more slowly than IOPD but can still cause significant muscle weakness and respiratory problems[2].

Ongoing Clinical Trials

Several clinical trials are currently underway to evaluate the safety and efficacy of cipaglucosidase alfa in different patient populations:

  1. Study for Infantile-Onset Pompe Disease (IOPD): This trial is evaluating cipaglucosidase alfa in children with IOPD aged 0 to 18 years. It includes both patients who have never received enzyme replacement therapy (ERT-naïve) and those who have previously received treatment (ERT-experienced)[1].

  2. Study for Late-Onset Pompe Disease (LOPD) in Children: This trial is studying the use of cipaglucosidase alfa in children aged 0 to 18 years with LOPD. Like the IOPD study, it includes both ERT-naïve and ERT-experienced patients[2].

  3. Long-term Study for Adults with LOPD: This is an extension study to assess the long-term safety and efficacy of cipaglucosidase alfa in adult patients with LOPD who have completed a previous clinical trial[3].

Potential Benefits

The clinical trials are evaluating several potential benefits of cipaglucosidase alfa, including:

  • Improved muscle strength and function
  • Enhanced respiratory function
  • Increased walking distance (measured by the 6-minute walk test)
  • Better quality of life
  • Reduced levels of disease biomarkers (such as creatine kinase and Hex4)

These outcomes are being measured through various tests and assessments, including muscle strength tests, pulmonary function tests, and patient-reported outcome measures[2][3].

Safety and Side Effects

As with any medication, safety is a primary concern in the clinical trials of cipaglucosidase alfa. The studies are closely monitoring for any adverse events, particularly:

  • Infusion-associated reactions (IARs)
  • Hypersensitivity reactions
  • Changes in laboratory test results
  • Effects on vital signs and heart function (monitored by ECG)

The development of antibodies against the medication (immunogenicity) is also being closely monitored, as this can potentially affect the treatment’s effectiveness[1][2][3].

Conclusion

Cipaglucosidase alfa represents a promising new treatment option for patients with Pompe disease. While it is still in the clinical trial phase, the ongoing studies are providing valuable information about its safety and effectiveness in both children and adults with different forms of the disease. As research continues, it is hoped that this medication will offer improved outcomes for patients living with this challenging genetic disorder.

Aspect Details
Drug Name Cipaglucosidase Alfa (ATB200)
Condition Studied Pompe disease (Infantile-onset and Late-onset)
Age Groups Infants, children, and adults up to 18 years old
Administration Intravenous infusion, often co-administered with oral Miglustat
Primary Objectives Safety, tolerability, efficacy, pharmacokinetics, and immunogenicity
Key Efficacy Measures 6-minute walk test, forced vital capacity, muscle strength, motor function
Safety Assessments Adverse events, infusion-associated reactions, laboratory tests, ECG, vital signs
Study Duration Up to 104 weeks for primary treatment, with long-term extension studies
Orphan Drug Designation EU/3/18/2000

FAQ

  • What is Cipaglucosidase Alfa? Cipaglucosidase Alfa is an investigational enzyme replacement therapy designed to treat Pompe disease. It aims to replace the deficient enzyme (acid alpha-glucosidase) that causes the condition.
  • What types of Pompe disease are being studied in these clinical trials? The clinical trials are studying both infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD) in various age groups, from infants to adults under 18 years old.
  • How is Cipaglucosidase Alfa administered in these trials? Cipaglucosidase Alfa is administered intravenously (through a vein) in these clinical trials. It is often co-administered with another drug called Miglustat.
  • What are the main goals of these clinical trials? The main goals of these trials are to evaluate the safety, efficacy, pharmacokinetics (how the drug moves through the body), and immunogenicity (immune system response) of Cipaglucosidase Alfa in patients with Pompe disease.
  • How long do these clinical trials last? The duration of these clinical trials varies, with some lasting up to 104 weeks (2 years) for the primary treatment period, and others extending to long-term follow-up studies lasting several years.

Ongoing Clinical Trials on Cipaglucosidase Alfa

  • Study on Reducing Treatment Frequency of Alglucosidase Alfa for Elderly Patients with Late-Onset Pompe Disease

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease

    Recruiting

    3 1 1
    Investigated drugs:
    Denmark France Germany Italy The Netherlands

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

    Not recruiting

    3 1 1
    Investigated drugs:
    Belgium Denmark France Greece Hungary Italy +3

  • Study on the Safety and Efficacy of Cipaglucosidase Alfa and Miglustat for Children with Late-onset Pompe Disease

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Germany Italy

Glossary

  • Pompe disease: A rare genetic disorder caused by the deficiency of an enzyme called acid alpha-glucosidase (GAA), leading to the buildup of glycogen in various tissues, primarily affecting muscle function.
  • Enzyme replacement therapy (ERT): A treatment approach that involves providing a functional version of a missing or deficient enzyme to patients with certain genetic disorders.
  • Infantile-onset Pompe disease (IOPD): A severe form of Pompe disease that manifests in infants, typically characterized by muscle weakness, heart problems, and respiratory difficulties.
  • Late-onset Pompe disease (LOPD): A milder form of Pompe disease that can appear in childhood, adolescence, or adulthood, primarily affecting skeletal muscles and respiratory function.
  • Cipaglucosidase Alfa: An investigational enzyme replacement therapy designed to treat Pompe disease by replacing the deficient GAA enzyme.
  • Miglustat: A drug co-administered with Cipaglucosidase Alfa in some clinical trials, which may help stabilize the enzyme and improve its effectiveness.
  • 6-minute walk test (6MWT): A clinical test that measures the distance an individual can walk in 6 minutes, used to assess functional capacity and endurance.
  • Forced vital capacity (FVC): A measure of lung function that represents the total amount of air exhaled forcefully after taking a deep breath.
  • Infusion-associated reactions (IARs): Side effects that may occur during or shortly after receiving an intravenous infusion of a medication.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Immunogenicity: The ability of a substance to provoke an immune response in the body, which can affect the safety and efficacy of a treatment.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-cipaglucosidase-alfa-and-miglustat-for-children-with-infantile-onset-pompe-disease/
  2. http://clinicaltrials.eu/trial/study-on-the-safety-and-efficacy-of-cipaglucosidase-alfa-and-miglustat-for-children-with-late-onset-pompe-disease/
  3. http://clinicaltrials.eu/trial/study-on-long-term-safety-and-efficacy-of-cipaglucosidase-alfa-and-miglustat-for-adults-with-late-onset-pompe-disease/