Recruiting Pompe Disease Studies in Farmington
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 Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)
This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygou...
About Pompe Disease Clinical Trials in Farmington
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 Farmington, CONNECTICUT. These studies are seeking a combined 81 participants. Research is being sponsored by ModernaTX, Inc., Beam Therapeutics Inc.. Clinical trial participation is free and participants receive study-related medical care at no cost.
Pompe Disease Clinical Trials in Farmington — FAQ
Are there pompe disease clinical trials in Farmington?
Yes, there are 2 pompe disease clinical trials currently recruiting in Farmington, CONNECTICUT. Browse the studies on this page to find one that fits.
How do I join a clinical trial in Farmington?
Browse the studies listed above, click one that interests you, and complete the free eligibility form. A study coordinator at the Farmington research site will contact you about next steps.
Are clinical trials in Farmington free?
Yes, clinical trial participation is always free. Study-related treatment, medical tests, and doctor visits are provided at no cost. Many Farmington studies also compensate for your time and travel.
What pompe disease treatments are being tested?
The 2 active trials in Farmington are testing new therapies including novel drugs, biologics, and treatment approaches for pompe disease.
Data updated March 2, 2026 from ClinicalTrials.gov