Clinical trials · North Carolina
Down Syndrome clinical trials in North Carolina
We’re tracking 114 recruiting down syndrome studies with a site in North Carolina — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in North Carolina
- NCT06872125RecruitingPhase 3
Testing zorevunersen for Dravet syndrome
This study tests an experimental drug called zorevunersen for people with Dravet syndrome. It aims to see if the drug can reduce seizures and improve safety.
Chapel Hill, North CarolinaAges 2–17 - NCT07742735RecruitingPhase 3
Study of apraglutide for short bowel syndrome with intestinal failure
This study tests a new medicine called apraglutide, given as a shot, to see if it can help adults with short bowel syndrome and intestinal failure reduce their need for intravenous nutrition (parenteral support). The study compares apraglutide to a placebo (a shot with no medicine) to check if it is safe and effective.
Durham, North CarolinaAges 18 years+ - NCT06577441RecruitingPhase 2
Adding enasidenib to usual care for higher-risk MDS with IDH2 mutation
This trial tests whether adding enasidenib (a targeted pill) to the standard chemotherapy (cedazuridine-decitabine) works better than the chemotherapy alone for people with higher-risk myelodysplastic syndrome (MDS) whose cells have a specific change in the IDH2 gene.
Durham, North CarolinaAges 18 years+ - NCT05677100Recruiting
Aortix device for acute heart failure
This trial is testing a small pump placed in the aorta to help the kidneys remove extra fluid when standard IV diuretics aren't working well enough for severe heart failure. It may help you if you're hospitalized and still very congested (swollen, fluid buildup) despite strong water pills.
Charlotte, North CarolinaAges 21 years+ - NCT07605429RecruitingPhase 3
Rugonersen treatment for Angelman syndrome
This study tests a new medicine called rugonersen for people with Angelman syndrome. The medicine is given as an injection into the fluid around the spinal cord and may help improve symptoms by targeting the genetic cause of the condition.
Chapel Hill, North CarolinaAges 1–50 - NCT06371417RecruitingPhase 1
Testing RAY121 for several immune diseases (RAINBOW trial)
This trial tests a new medicine called RAY121 (an antibody) for people with certain autoimmune diseases. It aims to see if RAY121 is safe and how the body handles it. If you have one of these conditions and meet the basic health requirements, you might be able to join.
Chapel Hill, North CarolinaAges 18–85 - NCT07422480RecruitingPhase 3
Comparing elritercept to epoetin alfa for anemia in MDS needing transfusions
This trial tests a new drug called elritercept against a standard treatment, epoetin alfa, for people with a low-risk form of MDS who need regular blood transfusions for anemia. The goal is to see if elritercept works better to reduce the need for transfusions.
Winston-Salem, North CarolinaAges 18 years+ - NCT07277582RecruitingPhase 2/Phase 3
Study of THRV-1268 for long QT syndrome type 2
This trial tests an investigational drug called THRV-1268 in people with a specific genetic form of long QT syndrome (type 2). The goal is to see if it can safely help control the heart's electrical activity and reduce the risk of dangerous heart rhythms.
Wilmington, North CarolinaAges 15 years+ - NCT04278404Recruiting
Drug safety study for children already on certain medicines
This study measures how certain medicines move through the body and checks their safety in children. It may help doctors better understand dosing and side effects in kids who are already receiving these treatments, or in some kids who recently tested positive for COVID-19.
Chapel Hill, North CarolinaAges birth–20 years - NCT04507997Recruiting
Study of the natural course of Angelman syndrome
This study follows people with Angelman syndrome to understand how their symptoms and development change over time. It may help researchers better describe the condition and support future treatments.
Carrboro, North CarolinaAges Any age - NCT05052606Recruiting
Turner syndrome patient registry study
This is a registry study that collects health information from people who have Turner syndrome. It helps researchers better understand the condition and guide future care.
Chapel Hill, North CarolinaAges Any age - NCT05127226RecruitingPhase 1/Phase 2
Test drug safety for Angelman syndrome in young children
This early-phase study tests a new experimental medicine called ION582 to see if it is safe, tolerable, and how the body processes it in people with Angelman syndrome. It may also look at early signs of how the medicine affects the brain and body, which could guide future treatments.
Carrboro, North CarolinaAges birth–50 years - NCT05143996RecruitingPhase 1
First study drug for relapsed AML or MDS
This Phase 1 trial tests CLN-049 in adults whose acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) has come back or did not respond to treatment. The goal is to find out how well the drug can work and how safe it is in people with limited options.
Durham, North CarolinaAges 18 years+ - NCT05308264RecruitingPhase 1/Phase 2
R289 for lower-risk MDS that still needs blood support
This Phase 1/2 study tests R289 in adults with lower-risk myelodysplastic syndromes (MDS) whose disease keeps causing anemia and/or ongoing need for red blood cell transfusions. It may help reduce blood problems and treat MDS when standard options haven’t worked well enough or aren’t suitable.
Durham, North CarolinaAges 18 years+ - NCT05354141RecruitingPhase 3
Tested treatment with tiny lung particles for moderate to severe ARDS
This Phase 3 trial studies an “extracellular vesicle” treatment in people with moderate to severe ARDS, a serious lung breathing problem. The goal is to see whether the treatment can improve outcomes compared with standard care.
Durham, North CarolinaAges 18–75 - NCT05889559Recruiting
Testing a fluid drainage method to detect leg pressure problems
This study looks at a special drainage measurement (“tissue ultrafiltration”) to better detect and treat acute compartment syndrome, a dangerous increase in pressure in the leg after major injury. It may help doctors identify who needs urgent treatment and improve outcomes after severe leg fractures or crush injuries.
Charlotte, North CarolinaAges 18–60 - NCT06366464RecruitingPhase 3
Study of a drug for sleepiness in Prader-Willi syndrome
This trial tests a medication called pitolisant to see if it helps reduce excessive daytime sleepiness in people with Prader-Willi syndrome. You or your loved one may be able to join if they have a confirmed diagnosis and a caregiver who can regularly help with study tasks.
Morrisville, North CarolinaAges 6 years+ - NCT05419492RecruitingPhase 1/Phase 2
ETX101 for children with SCN1A Dravet syndrome
This early-phase study tests how safe ETX101 is and whether it can help reduce seizures in young children with Dravet syndrome caused by an SCN1A gene change. It mainly enrolls infants and children with a specific type of seizure history and genetic result.
Durham, North CarolinaAges 6 months–17 years - NCT06499285RecruitingPhase 3
Study of elritercept for anemia in low-risk MDS needing transfusions
This trial tests an investigational drug called elritercept for adults with a specific type of low-risk MDS (myelodysplastic syndromes) who have anemia (low red blood cells) and need regular blood transfusions. The goal is to see if elritercept can reduce or eliminate the need for transfusions.
Winston-Salem, North CarolinaAges 18 years+ - NCT06668805RecruitingPhase 2
Testing vosoritide for children with Noonan syndrome and slow growth
This study tests a medication called vosoritide for children with Noonan syndrome who are still growing slowly despite using human growth hormone. The goal is to see if vosoritide can help them grow taller.
Charlotte, North CarolinaAges 3–11 - NCT06747949RecruitingPhase 3
Long term extension study of dazodalibep for Sjogren's syndrome
This study gives continued access to the study drug dazodalibep for people with Sjogren's syndrome who completed a prior phase 3 study. The goal is to see how safe and effective the medication is over a longer period.
Charlotte, North CarolinaAges 18 years+ - NCT07135167RecruitingPhase 3
Epi-ON corneal crosslinking study for people with Down syndrome
This study tests a special light treatment for cornea problems in people with Down syndrome. It aims to strengthen the front of the eye and may help prevent vision loss.
Asheville, North CarolinaAges 8 years+ - NCT07157254RecruitingPhase 2
Study of GTX-102 for People with Angelman Syndrome
This study tests an experimental drug called GTX-102 for people with Angelman syndrome. It aims to see if the drug is safe and can help with symptoms. The trial includes different age groups and genetic types of Angelman syndrome.
Hillsborough, North CarolinaAges 1–64 - NCT07219602RecruitingPhase 3
Study of obicetrapib and ezetimibe for diabetes and metabolic syndrome
This trial tests a combination medication (obicetrapib plus ezetimibe) taken daily to lower cholesterol and triglycerides in people with type 2 diabetes or metabolic syndrome who are already on standard cholesterol treatment. It may help reduce heart risks.
Durham, North CarolinaAges 18 years+
Down Syndrome trials by city in North Carolina
Where these studies are running in North Carolina
Institutions with a site for the recruiting down syndrome studies listed above.
- Duke University Medical Center · 3 studies
- University of North Carolina at Chapel Hill · 3 studies
- Duke University Health System · 2 studies
- Novant Health · 2 studies
- Arthritis and Osteoporosis Consultants of the Carolinas
- Atrium Health Musculoskeletal Institute
- Carolina Institute for Developmental Disabilities
- Carolinas Medical Center/Levine Cancer Institute
What taking part in a down syndrome 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.
Down Syndrome trials in other states
Common questions
- Are there down syndrome clinical trials in North Carolina?
- Yes. We're currently tracking 114 recruiting down syndrome 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 down syndrome 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 down syndrome 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 down syndrome 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.