HELP ACCELERATE RESEARCH FOR
Pompe Disease
Study
Pompe Disease At-Home Research Study
Location: Nationwide
Compensation: $150
Length: 30 MINUTES
The goal of this research is to allow researchers to enhance our understanding of Pompe Disease, in support of developing new diagnostic and treatment options for patients living with this condition.
You may quality if you:
- Are 18 to 65 years of age
- Participants who are willing to participate in a total of 3 visits according to the outlined visit schedule.
- Participants who are diagnosed with Late Onset Pompe Disease, defined as having symptom onset after 1 year of age and having no history of Pompe-related cardiac hypertrophy
- Participants who have been treated with a stable dose of Nexviazyme or Pombiliti + Opfolda administered every 2 weeks for at least 6 months at the time of screening.Participants who are willing to fast for at least 8 hours before each scheduled collection
- Live in the United States
You may not qualify if you:
- Participants who are participating in another clinical study using investigational treatment or who have taken an investigational treatment within 30 days or 5 half-lives from screening, whichever is longer
- Participants who have received an experimental cell or gene therapy at any time point.
- Participants who are invasively ventilated or require non-invasive positive pressure ventilation for more than 12 hours per day while awake.
- Participants who are wheelchair bound or unable to walk ≥ 40 m (approximately 130 feet or “half a city block,” use of assistive devices is allowed).
- Participants who are currently taking acarbose, miglitol, or voglibose or who have taken any of these medications in the past 30 days at the time of screening.
- Are pregnant or nursing
- Have a known history of HIV, hepatitis, and other infectious disease
- Experience excess blood loss including blood donation defined as 250 mL in the last two months
How It Works
It’s a simple three-step process
Step 1: Tell us about yourself
Submit a health profile by answering basic questions about your medical history.
Step 2: See if you qualify
Speak with a research coordinator to learn more about what is required to participate.
Step 3: Participate from Home
Complete study requirements from Home with the help of our mobile health staff.
Frequently Asked Questions
You can withdraw from the study or decline participation at any time for any reason.
All of our studies and staff are HIPAA compliant and follow all state and federal regulations. We keep your information confidential. Check out our privacy and protection policies to learn more.
Yes. However, to participate in most studies, you will need to provide proof of your diagnosis. Even if you are not currently seeing a physician, you can still sign an authorization for Medical Record Release from your former physician that can serve as proof of diagnosis. Previous medical records can serve as proof of diagnosis.
We may ask you to sign a Release of Medical Records so that we can provide researchers with the data they need to conduct as comprehensive of a study as possible. Your de-identified medical information will help translate laboratory results from collected samples into clinical information that may be used to develop new therapies and/or diagnostics.
No. We collect blood and other samples from donors in order to provide scientists with quality samples for their research. We do not perform diagnostic tests.