Clinical trials · Philadelphia, PA
Pompe Disease clinical trials in Philadelphia
We’re tracking 4 recruiting pompe disease studies with a site in or near Philadelphia — including Phase 1/Phase 2 and Phase 2 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- 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.
Philadelphia, PennsylvaniaAges Any age - 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.
Philadelphia, 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.
Philadelphia, PennsylvaniaAges 18 years+ - 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
Hear when a new Pompe Disease trial opens near Philadelphia, PA
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Pompe Disease trials in other cities
Where these studies are running near Philadelphia
Institutions with a site for the recruiting pompe disease studies listed above.
- University of Pennsylvania · 2 studies
- Children's Hospital of Philadelphia
- Cooper University Health Care
- Perelman Center for Advanced Medicine
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.
Looking wider? See all recruiting pompe disease studies in Pennsylvania.
Common questions
- Are there pompe disease clinical trials near Philadelphia?
- Yes. We're currently tracking 4 recruiting pompe disease studies with a site near Philadelphia, 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 near Philadelphia?
- 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 Philadelphia to take part?
- Not necessarily. These studies have a site near Philadelphia, 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 near Philadelphia regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.