Recruiting Pompe Disease Studies in Gainesville
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric sub...
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experience...
About Pompe Disease Clinical Trials in Gainesville
Pompe disease is a rare genetic disorder caused by accumulation of glycogen in cells due to deficiency of the enzyme acid alpha-glucosidase. It can affect the heart, skeletal muscles, and respiratory system. Treatment includes enzyme replacement therapy.
There are currently 2 pompe disease clinical trials recruiting participants in Gainesville, FLORIDA. These studies are seeking a combined 58 participants. Research is being sponsored by Amicus Therapeutics. Clinical trial participation is free and participants receive study-related medical care at no cost.
Pompe Disease Clinical Trials in Gainesville — FAQ
Are there pompe disease clinical trials in Gainesville?
Yes, there are 2 pompe disease clinical trials currently recruiting in Gainesville, FLORIDA. Browse the studies on this page to find one that fits.
How do I join a clinical trial in Gainesville?
Browse the studies listed above, click one that interests you, and complete the free eligibility form. A study coordinator at the Gainesville research site will contact you about next steps.
Are clinical trials in Gainesville free?
Yes, clinical trial participation is always free. Study-related treatment, medical tests, and doctor visits are provided at no cost. Many Gainesville studies also compensate for your time and travel.
What pompe disease treatments are being tested?
The 2 active trials in Gainesville are testing new therapies including novel drugs, biologics, and treatment approaches for pompe disease.
Data updated March 2, 2026 from ClinicalTrials.gov