Clinical trials · North Carolina
Down Syndrome clinical trials in North Carolina
We’re tracking 116 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+ - 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+ - 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 - 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 - NCT07519395RecruitingPhase 2
Camlipixant for IBS with diarrhea or mixed symptoms
This study tests an investigational drug, camlipixant, to see if it can reduce belly pain and diarrhea or mixed bowel symptoms in adults with irritable bowel syndrome (IBS). If you've tried other IBS treatments without enough relief, this might be an option.
Charlotte, North CarolinaAges 18–80 - 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+ - 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.
Durham, 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.
Durham, 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.
Durham, 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.
Durham, North CarolinaAges 18 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+ - 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 - 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.
Charlotte, North CarolinaAges Up to 22 years - 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 - NCT07782827RecruitingPhase 3
Angel syndrome treatment study for children and adults
This study tests an experimental medicine for Angelman syndrome (AS), a genetic condition affecting the nervous system. The medicine is given by lumbar puncture and aims to improve symptoms.
Carrboro, North CarolinaAges 2–50 - 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+ - NCT07625579Recruiting
Collaboration for Down Syndrome Progress Research Study
This research study aims to better understand Down syndrome by collecting health information, family history, and sometimes brain imaging or sleep data from children and adults with Down syndrome and their families. The goal is to advance knowledge that could improve health and development for people with Down syndrome.
Chapel Hill, North CarolinaAges Any age - NCT07611435RecruitingPhase 2
Testing a Safe Control for Alpha-Gal Syndrome Food Challenges
This study helps researchers understand the best way to safely test whether people with alpha-gal syndrome (an allergy to red meat) can tolerate pork. You'll undergo a controlled food challenge with medical supervision and access to emergency medications.
Chapel Hill, North CarolinaAges 12–50 - 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 - NCT07531745RecruitingPhase 1/Phase 2
ION337 gene therapy for children with Dravet syndrome
This study tests a new gene therapy called ION337 designed to treat Dravet syndrome, a severe childhood epilepsy caused by a SCN1A gene mutation. The therapy is given as a spinal fluid injection and aims to reduce seizures in children ages 2–12.
Durham, North CarolinaAges 2–12 - NCT07450664Enrolling by invitation
Observational study of VYKAT XR for Prader-Willi syndrome
This study follows people with Prader-Willi syndrome who are already taking or starting VYKAT XR, including those switching from an earlier version of the drug (DCCR). It aims to understand how the treatment works in everyday life.
Chapel Hill, North CarolinaAges 4 years+ - 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+
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 · 7 studies
- Duke Lung Transplant Clinic - Clinic 2F/2G - PPDS · 4 studies
- Durham VA Medical Center · 4 studies
- Wake Forest University Health Sciences · 4 studies
- UNC Lineberger Comprehensive Cancer Center · 3 studies
- Duke University Medical Center · 2 studies
- Carolinas Medical Center/Levine Cancer Institute
- Duke University Health Systems
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 116 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.