Share Your Thoughts to Shape the Future of Clinical Trials
The APBD Research Foundation (APBDRF) is conducting a survey to understand what matters to patients and their families when it comes to participating in clinical trials for new treatments -- referred to as “clinical trial readiness.”
As clinical treatment trials for adult-onset GSD IV (also known as “APBD”) appear on the horizon, it becomes increasingly important to understand the many elements that may impact the success of future clinical trials in the GSD IV community.
The Foundation wants to understand how to make a clinical trial successful for all GSD IV patients, including APBD and early-onset GSD IV (also known as “Andersen Disease”). Your voice is essential in shaping the future of these studies! We want to understand what makes participating in clinical trials easier or more difficult for patients and their families.
TAKE THE SURVEY!
Follow one of the links below to share your voice:
NOTE: The surveys will remain open until October 1, 2026.
For questions regarding your rights as a participant in this research or IRB approval, contact Dr. Lindsay Gill, APBDRF Research Manager, by email at lindsay@apbdrf.org.
For technical questions or assistance with completing the survey, contact our Study Staff by email at intern@apbdrf.org.
Clinical Trial Readiness Survey Study Details
What is the purpose of the study?
To understand what and how certain factors may impact a patient’s decision to participate in a clinical treatment trial.
Who is eligible to participate?
Adult-onset GSD IV (APBD) patients
Caregivers of patients with an APBD diagnosis
Caregivers of patients with an early-onset GSD IV diagnosis
You must be 18 years or older to participate.
What is involved in participation?
Patients and caregivers are asked to complete a 10-15 minute, anonymous survey that includes questions regarding patient current health status, willingness to participate in clinical treatment trials, and what conditions could make or break interest in participating in a trial. Participation in the survey is voluntary.
Are there potential risks or benefits to participating?
No risks or discomforts beyond what would be experienced in everyday life are anticipated. There are no direct or medical benefits associated with participating.
Will I be compensated for my participation?
No, participants will not be compensated for taking the survey. There is also no cost to the participant to participate in the study.