Response to Diaphragmatic Pacing in Subjects with Pompe Disease

Purpose

The purpose of this research study is to evaluate the effect of diaphragm pacing on breathing function in people with Pompe disease. You will be in this study for approximately six months. However, you will have the opportunity to participate in the extension phase of this study for up to six years.

Procedures

If you agree to participate in this study, reports from your standard of care testing and copies of images from standard of care testing will be collected and added to the study record.

The study team will follow you closely the first 6 months after DPS placement to see if your breathing function changes after the pacemaker is placed. The study team will contact you by phone or by video conference every at least twice a month to ask questions about how you are feeling and how many hours per day you are pacing and how many hours per day you are using ventilator assistance.

In addition, you will be asked to complete questionnaires about your medical history and your daily activities including a pacemaker diary and ventilator diary.

Pulmonary function testing, or special breathing tests that measure changes in your breathing pattern, will be completed by the study team during the course of the study.

Eligibility

  • Diagnosed with Pompe disease
  • 2-65 years old

Additional eligibility criteria apply. Please contact Jessica Ehrbar for more information about this study: 

Age

18 and under
18 to 65

Gender

Male
Female

Monetary compensation

Yes

Can be done from home

No

Keywords

Pompe Disease

Principal Investigator

Barbara Smith, PT, PhD

Department

Neurology

Contact Information

jehrbar@ufl.edu

(352) 273-6855


Be an Informed Participant

Before deciding to participate in a research study, take time to learn about clinical research, how it's conducted and your rights as a research participant. Following are some helpful resources from independent sources. Always remember that a clinical research study is research, not treatment.

Know Who to Contact

  • Eligibility: For questions about a specific study and who is eligible to participate, call or email the contact person listed for that study.
  • Your Rights: For questions about your rights as a research participant, contact the UF Institutional Review Boards at 352-273-9600.
  • Feedback: For general questions or feedback about study listings, email the UF Clinical and Translational Science Institute at CTSI-Studies-L@lists.ufl.edu.

Other Resources

  • HealthStreet: Health-focused services, classes and events, and opportunities to participate in research.
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