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 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in North Carolina
- NCT03836300Enrolling by invitation
Parent-infant coaching for families with young diagnoses
This study tests a parent-and-infant coaching program for babies and caregivers. It aims to see whether the program helps after an early diagnosis when English is used at home.
Research Triangle Park, North CarolinaAges Up to 99 years - NCT03047369Recruiting
Myelin brain disorder research biobank for samples and data
This study collects brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
Winston-Salem, North CarolinaAges Any age - NCT06740162Recruiting
Physical activity program for adults with intellectual disabilities
This study tests a program to help adults with intellectual disabilities become more physically active. You and your caregiver will work with a coach to increase your daily movement, with the goal of improving health and community connection.
Chapel Hill, North CarolinaAges 18 years+ - NCT05702645Recruiting
Down syndrome long-term study after leukemia treatment
This study follows children and young adults with Down syndrome who have survived acute leukemia (ALL or AML) to learn about their longer-term health after treatment ends. It may help families and doctors understand what health problems to watch for and how to provide better follow-up care.
Chapel Hill, North CarolinaAges 6–39 - NCT06484062RecruitingPhase 1
Testing cirtuvivint alone and with ASTX727 for leukemia and MDS
This trial tests a new drug called cirtuvivint, given alone or with another drug (ASTX727), for people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that has come back or not responded to treatment, and for people with high-risk MDS who have not been treated before. The goal is to see if these combinations work better than current options.
Winston-Salem, North CarolinaAges 18 years+ - 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 - NCT06789913RecruitingPhase 2
Testing RLY-2608 for growth disorders linked to PIK3CA mutations
This trial tests a new medicine, RLY-2608, that targets the specific genetic change (PIK3CA mutation) causing overgrowth conditions. It aims to see if the drug can slow or stop the growth of affected tissues.
Chapel Hill, North CarolinaAges 2 years+ - NCT07075445Recruiting
Living with long QT syndrome types 2 and 3
This study looks at how long QT syndrome types 2 and 3 affect your daily life and health. It asks you to wear a smartwatch and answer questions to help researchers understand the challenges you face.
Greenville, North CarolinaAges 18 years+ - NCT06703073RecruitingPhase 2
Testing new treatments for acute respiratory distress syndrome (ARDS)
This trial is testing new treatments for people with ARDS, a serious lung condition. The goal is to find therapies that help patients breathe better and recover faster. If you or a loved one has ARDS, this study may offer access to experimental treatments.
Chapel Hill, North CarolinaAges 18 years+ - NCT06701656RecruitingPhase 2
Testing bevacizumab for breathing problems from ARDS
This trial tests if bevacizumab can help people with ARDS (a serious lung condition) breathe better. It is for patients with mild, moderate, or severe ARDS who are already in the hospital.
Chapel Hill, North CarolinaAges 18 years+ - NCT06701682RecruitingPhase 2
Study of vilobelimab for moderate to severe ARDS
This trial tests a drug called vilobelimab for people with moderate or severe ARDS (a serious lung condition). It aims to find if this treatment can help improve breathing and outcomes.
Chapel Hill, North CarolinaAges 18 years+ - NCT06701669RecruitingPhase 2
Study of Paridiprubart for moderate to severe ARDS
This trial tests a new medicine called paridiprubart to see if it can help people with moderate or severe acute respiratory distress syndrome (ARDS), a serious lung condition. It may be an option if you have low oxygen levels and no known allergy to the drug.
Chapel Hill, North CarolinaAges 18 years+ - NCT06168058Recruiting
Pelvic vein embolization for chronic pelvic pain
This trial tests if blocking enlarged veins in the pelvis (called embolization) can reduce long-term pelvic pain that hasn't improved with other treatments. It may help women whose pain is worse when standing or walking and better when lying down.
Chapel Hill, North CarolinaAges 18–75 - NCT04571658Recruiting
NEPTUNE Match study linking you to possible kidney research trials
This study is an add-on process that uses information from the NEPTUNE kidney study to see whether you might be able to join other kidney research trials. It may help connect eligible people with appropriate new treatment studies when they become available.
Chapel Hill, North CarolinaAges 1–80 - NCT04726241RecruitingPhase 1/Phase 2
Testing blood and bone marrow samples in young leukemia patients
This trial looks at blood and bone marrow samples from children and teens whose leukemia has come back or is hard to treat. The goal is to better understand the disease and guide future options for these patients.
Asheville, North CarolinaAges Up to 22 years - NCT06465953RecruitingPhase 3
Ivosidenib vs azacitidine for IDH1-mutated MDS
This trial tests if the drug ivosidenib alone or azacitidine alone can help people with a specific type of MDS that has an IDH1 mutation. It is for people who have not had strong MDS treatments yet.
Chapel Hill, North CarolinaAges 18 years+ - NCT07519395RecruitingPhase 2
Camlipixant for abdominal symptoms in IBS-D and IBS-M
This study tests whether a new drug called camlipixant can reduce belly pain and other uncomfortable symptoms in people with irritable bowel syndrome that causes diarrhea or mixed symptoms. The trial compares camlipixant to a placebo (inactive pill) to see if it works better.
Greenville, North CarolinaAges 18–80 - NCT06914609RecruitingPhase 3
Study of ION582 for people with Angelman syndrome
This trial tests a new medicine called ION582 for people with Angelman syndrome. It aims to see if it can help with symptoms and is given as a lumbar puncture (spinal tap). You may be able to join if you have a genetic diagnosis of Angelman syndrome and are between 2 and 50 years old.
Carrboro, North CarolinaAges 2–50 - NCT05320198RecruitingPhase 1/Phase 2
Testing DISC-0974 for anemia in myelofibrosis or MDS
This trial tests an investigational drug called DISC-0974 for people with myelofibrosis or certain types of MDS who have low red blood cell counts (anemia). The goal is to see if this treatment can help improve anemia and reduce the need for blood transfusions.
Winston-Salem, North CarolinaAges 18 years+ - NCT06196203RecruitingPhase 2
Study of AK117 with Azacitidine for High-Risk MDS
This trial tests a new drug, AK117, combined with azacitidine for people newly diagnosed with high-risk myelodysplastic syndromes (MDS), a blood disorder. The goal is to see if the combination can help improve outcomes.
Chapel Hill, North CarolinaAges 18 years+ - NCT05090345Recruiting
Registry for young people getting bone marrow transplants
This trial is a registry that follows children and young adults up to age 26 who are having a bone marrow transplant, to better understand complications related to blood vessel and organ injury. It may help future patients by improving how doctors predict and prevent these problems.
Durham, North CarolinaAges birth–26 years - NCT06679855RecruitingPhase 3
Milrinone to prevent heart problems after PDA closure
This trial tests if giving milrinone before surgery to close a common heart vessel (PDA) can prevent heart issues in premature babies. It may help very premature infants who need surgery for a large PDA.
Durham, North CarolinaAges Up to 3 months - 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+
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.
- University of North Carolina at Chapel Hill · 9 studies
- Duke Cancer Institute · 4 studies
- Durham VA Medical Center · 4 studies
- UNC Lineberger Comprehensive Cancer Center · 4 studies
- Wake Forest University Health Sciences · 3 studies
- Atrium Health Wake Forest Baptist Medical Center (Wake Forest Baptist) · 2 studies
- Duke University · 2 studies
- Duke University Medical Center · 2 studies
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.