Pompe Disease Treated With Avalglucosidase Alfa in the Real-World Setting

Overview

About this study

The purpose of this study will provide important londitdinal data to charactererize the long-tem profile of avalglucosidase in alfa use in the real world setting.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria: 

  • The participant has a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as documented GAA enzyme deficiency from any tissue source and /or documentation of 2 GAA gene mutations)
  • The participant is treated with avalglucosidase alfa before enrollment of planning to start treatment with avalglucosidase alfa upon enrollment, regardless of prior experience with ERTs.
  • The participant and/or the participants’ parent/legal guardian is willing and able to provide signed informed consent, and willing to be followed up to five years after enrollment. 

Exclusion Criteria: 

  • The participant received and investigational drug within five half-lives after the most recent dose prior to signing and ICF. 
  • The participant plans to take and investigational product after signing the ICF.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 5/16/2024. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Teerin Liewluck, M.D.

Contact us for the latest status

Contact information:

Brenda Nelson CCRP, SOCRA

(507) 284-1588

Nelson.Brenda6@mayo.edu

More information

Publications

Publications are currently not available