Recruiting Pompe Disease Studies in Cincinnati
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a....
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...
About Pompe Disease Clinical Trials in Cincinnati
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 Cincinnati, OH. These studies are seeking a combined 81 participants. Research is being sponsored by ModernaTX, Inc., Amicus Therapeutics. Clinical trial participation is free and participants receive study-related medical care at no cost.
Pompe Disease Clinical Trials in Cincinnati — FAQ
Are there pompe disease clinical trials in Cincinnati?
Yes, there are 2 pompe disease clinical trials currently recruiting in Cincinnati, OH. Browse the studies on this page to find one that fits.
How do I join a clinical trial in Cincinnati?
Browse the studies listed above, click one that interests you, and complete the free eligibility form. A study coordinator at the Cincinnati research site will contact you about next steps.
Are clinical trials in Cincinnati free?
Yes, clinical trial participation is always free. Study-related treatment, medical tests, and doctor visits are provided at no cost. Many Cincinnati studies also compensate for your time and travel.
What pompe disease treatments are being tested?
The 2 active trials in Cincinnati are testing new therapies including novel drugs, biologics, and treatment approaches for pompe disease.
Data updated March 2, 2026 from ClinicalTrials.gov