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...
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...
Frequently Asked Questions
What clinical trials are available for Pompe Disease?
There are currently 3 actively recruiting clinical trials for pompe disease. These studies are testing new treatments, therapies, and interventions at research sites across 19 cities.
How do I join a Pompe Disease clinical trial?
To join a pompe disease clinical trial: 1) Browse the available studies above, 2) Click on a study that interests you, 3) Complete the eligibility form, and 4) A study coordinator will contact you.
Are clinical trials free for patients?
Yes, clinical trial participation is free. In most studies, the treatment, medical tests, and doctor visits related to the trial are provided at no cost. Some studies may also compensate for time and travel.
Can I leave a clinical trial if I change my mind?
Yes, participation in any clinical trial is completely voluntary. You can withdraw at any time, for any reason, without affecting your regular medical care.