A Post-Authorization Safety Study (Pass) to Assess Long-Term Safety in Patients with Pompe Disease Treated with Avalglucosidase Alfa in the Real-World Setting: the Savant (Safety Assessment of Avalglucosidase Alfa as a Novel Treatment) Study
Investigating Long-Term Safety of an Investigational Treatment for Pompe Disease, a Rare Glycogen Storage Disorder
Melissa Lah, MD
Primary Investigator
Brief description of study
This is an observational study with the aim of gathering safety information on avalglucosidase alfa treatment for Pompe disease. We will collect long term safety data and evaluate the important identified risk of infusion associated reactions (IARs) including hypersensitivity and anaphylactic reactions and the important potential risk of medication errors in participants treated with avalgucosidase alfa in both clinic/hospital and/or home settings as per standard of care (SoC) determined be their treating physician. In addition, to collect and evaluate immunogenicity of avalgucosidase alfa treatment in the long-term.
Detailed description of study
To collect long term safety data and evaluate the important identified risk of infusion associated reactions (IARs) including hypersensitivity and anaphylactic reactions and the important potential risk of medication errors in participants treated with avalglucosidase alfa in both clinic/hospital and/or home settings as per standard of care (SoC) determined by their treating physician. In addition, to collect and evaluate the immunogenicity of avalglucosidase alfa treatment in the long-term.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Pompe disease
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Age: 18 years - 100 years
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Gender: All
Inclusion criteria:
- Pompe disease
This study investigates the long-term safety of an investigational treatment for Pompe disease, a rare disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The purpose is to gather information on potential risks associated with the treatment, including infusion-related reactions and medication errors.
Participants will be observed in real-world settings, such as clinics or hospitals, as well as at home, following their usual care routines. This observational study will also monitor the body's immune response to the treatment over an extended period.
- Who can participate: This study is by invitation only. Participants are identified by their physicians and must already be receiving the investigational treatment or have it prescribed independently of the study.
- Study details: Participants will continue their usual treatment routine while data is collected on safety and immune responses. This study does not involve any changes to their existing care plan.
