Clinical trials · North Carolina
Genetic Disease clinical trials in North Carolina
We’re tracking 11 recruiting genetic disease studies with a site in North Carolina — including Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in North Carolina
- NCT05432349Recruiting
Rett syndrome genetic registry for MECP2 changes
This trial is a registry that collects health and genetic information from people with Rett syndrome. It may help researchers better understand how MECP2 loss-of-function changes lead to symptoms and how to support patients.
Chapel Hill, North CarolinaAges birth–99 years - NCT07608432RecruitingPhase 3
Testing a New DMD Treatment Given Every 4 Weeks
This study tests whether a new medicine called zeleciment rostudirsen can help ambulatory (walking) males with Duchenne muscular dystrophy (DMD). The medicine is given as an intravenous infusion (through a vein) every 4 weeks and works by helping the body produce a protective muscle protein.
Hillsborough, North CarolinaAges 4–18 - NCT06057181Recruiting
Helix research network for adults
This study is a research network that collects health information from adults to better understand health and disease. It is open to most adults who are willing to follow the study procedures.
Burlington, North CarolinaAges 18 years+ - NCT06644742Recruiting
PKP2 heart condition natural history study
This study is for people with a specific genetic heart condition (PKP2-related arrhythmogenic cardiomyopathy) who already have an ICD. It does not test a new treatment but instead follows participants over time to learn more about the condition.
Durham, North CarolinaAges 12 years+ - NCT05477563RecruitingPhase 3
One-time gene therapy for severe sickle cell and transfusion anemia
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
Charlotte, North CarolinaAges 12–35 - NCT04208529Enrolling by invitationPhase 3
Long-term follow-up after receiving CTX001 infusion
This study follows people for a long time after they received CTX001 in an earlier (parent) study. It helps researchers learn about long-term safety and outcomes of the treatment.
Charlotte, North CarolinaAges 2 years+ - NCT02450851Recruiting
Find answers for undiagnosed medical conditions using genetic testing
This study helps look for causes of health problems when no one has been able to give a diagnosis. It tests and shares medical and genetic information (with privacy protections) so researchers can better match findings to possible causes.
Durham, North CarolinaAges 1 month–100 years - NCT04994015RecruitingTeam says not enrolling
Genetic registry for people with Parkinson’s
This study builds a genetics “registry” of people diagnosed with Parkinson’s disease who agree to genetic testing. Your information may help researchers understand why Parkinson’s happens and improve future treatments.
Chapel Hill, North CarolinaAges 18 years+ - NCT05589714Recruiting
Study your rare eye gene and track retinal disease over time
This study builds a registry and tracks the natural history of rare retinal dystrophies while also testing people for disease-causing gene variants. It may help researchers better understand what causes retinal degeneration and how it progresses.
Durham, North CarolinaAges 4 years+ - NCT01193088Recruiting
Study genetics to find who modifies Charcot-Marie-Tooth (CMT)
This study looks at DNA changes in families with Charcot-Marie-Tooth (CMT), especially a known gene duplication called PMP22. It aims to understand what other genetic factors may change how CMT shows up, which could help future diagnosis and care.
Chapel Hill, North CarolinaAges Any age - NCT06991114RecruitingPhase 2
AlloNK and rituximab for relapsing rheumatologic diseases
This trial tests a new natural killer (NK) cell therapy called AlloNK given with rituximab for people with certain autoimmune diseases (rheumatoid arthritis, Sjögren's disease, inflammatory myopathies, or systemic sclerosis) that have not responded well to standard treatments. The goal is to see if this combination can better control the disease.
Charlotte, North CarolinaAges 18 years+
Genetic Disease trials by city in North Carolina
Where these studies are running in North Carolina
Institutions with a site for the recruiting genetic disease studies listed above.
- University of North Carolina at Chapel Hill · 3 studies
- Levine Children's Hospital · 2 studies
- Artiva Investigational Site Charlotte
- Artiva Investigational Site Statesville
- Atrium Health
- Cone Health Cancer Center at Alamance Regional
- Duke University
- Duke University Health System
What taking part in a genetic 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.
Genetic Disease trials in other states
Common questions
- Are there genetic disease clinical trials in North Carolina?
- Yes. We're currently tracking 11 recruiting genetic disease studies with a site in North Carolina, 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 genetic disease study in North Carolina?
- 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 North Carolina to take part?
- Not necessarily. These studies have a site in North Carolina, 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 genetic 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 genetic disease studies open in North Carolina 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.