Clinical trials · Raleigh–Durham, NC
Pompe Disease clinical trials in Raleigh–Durham
We’re tracking 8 recruiting pompe disease studies with a site in or near Raleigh–Durham — including Phase 1, Phase 1/Phase 2 and Phase 2 studies, each written for real people, not researchers.
Recruiting studies near Raleigh–Durham
- 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.
Durham, North CarolinaAges 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.
Durham, North CarolinaAges Any age - NCT07354724RecruitingPhase 1
Study of DNL952 for adults with late-onset Pompe disease
This trial tests a new drug, DNL952, in adults with late-onset Pompe disease. It aims to see if the drug is safe and how it works in the body, with different groups for people currently on enzyme replacement therapy or those who haven't had it in the past year.
Durham, North CarolinaAges 18–75 - 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.
Durham, North CarolinaAges 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.
Durham, North CarolinaAges 18–65 - NCT01665326Recruiting
Pompe disease screening and follow-up study
This study follows people with Pompe disease over time and checks their “CRIM status,” which is a lab result about a key protein. It may help researchers better understand the disease and how it progresses for different patient groups.
Durham, North CarolinaAges Any age - NCT04639336Recruiting
Pompe disease study of brain and nerve effects
This study looks at how Pompe disease affects the brain and nervous system. You may be able to join if you have a confirmed diagnosis of infantile or late-onset Pompe disease and agree to participate.
Durham, North CarolinaAges birth–18 years - NCT00567073Recruiting
Register pregnant people with Pompe disease for pregnancy data
This study keeps a special record of pregnancies in people with Pompe disease to learn more about pregnancy outcomes and newborn information. It does not test a drug by itself—it collects health information to improve future care.
Durham, North CarolinaAges Any age
Hear when a new Pompe Disease trial opens near Raleigh–Durham, NC
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Pompe Disease trials in other cities
Where these studies are running near Raleigh–Durham
Institutions with a site for the recruiting pompe disease studies listed above.
- Duke University Medical Center · 7 studies
- Duke University
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 North Carolina.
Common questions
- Are there pompe disease clinical trials near Raleigh–Durham?
- Yes. We're currently tracking 8 recruiting pompe disease studies with a site near Raleigh–Durham, 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 Raleigh–Durham?
- 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 Raleigh–Durham to take part?
- Not necessarily. These studies have a site near Raleigh–Durham, 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 Raleigh–Durham regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.