Clinical trials · Pennsylvania
Pompe Disease clinical trials in Pennsylvania
We’re tracking 4 recruiting pompe disease studies with a site in Pennsylvania — including Phase 1/Phase 2 and Phase 2 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT07282847RecruitingPhase 1/Phase 2
Gene therapy for adults with Pompe disease
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
Philadelphia, PennsylvaniaAges 18–65 - NCT06121011Recruiting
Pompe disease patient registry study
This is a registry study that collects information over time from people with Pompe disease. It helps doctors and researchers better understand the condition and how it affects daily life, without testing any new treatments.
Pittsburgh, PennsylvaniaAges Any age - NCT07123155RecruitingPhase 2
Testing a new add-on treatment for late-onset Pompe disease
This trial tests a new medication, S-606001, given alongside your current enzyme replacement therapy (ERT) for late-onset Pompe disease. It may help improve your breathing and walking ability.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT00231400Recruiting
Pompe disease registry for people with confirmed diagnosis
This trial is a registry, meaning it collects health information from people with confirmed Pompe disease. It may help researchers better understand the disease and improve future treatments.
Hershey, PennsylvaniaAges Any age
Pompe Disease trials by city in Pennsylvania
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting pompe disease studies listed above.
- University of Pennsylvania · 2 studies
- University of Pittsburgh · 2 studies
- UPMC Children's Hospital of Pittsburgh · 2 studies
- Children's Hospital of Philadelphia
- Penn State Milton S Hershey Medical Center
- Perelman Center for Advanced Medicine
- University of Pittsburgh Medical Center
What taking part in a pompe disease study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Pompe Disease trials in other states
Common questions
- Are there pompe disease clinical trials in Pennsylvania?
- Yes. We're currently tracking 4 recruiting pompe disease studies with a site in Pennsylvania, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a pompe disease study in Pennsylvania?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Pennsylvania to take part?
- Not necessarily. These studies have a site in Pennsylvania, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a pompe disease trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New pompe disease studies open in Pennsylvania regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.