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, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in North Carolina
- 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+ - NCT07277582RecruitingPhase 2/Phase 3
Study of a new medicine for long QT syndrome types 2 and 3
This study tests a new medicine called THRV-1268 to see if it can safely improve heart rhythm in people with Long QT syndrome types 2 and 3. It may help reduce the risk of dangerous heart rhythm problems.
Wilmington, North CarolinaAges 15 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 - NCT07219485Enrolling by invitationPhase 3
Pitolisant for Prader-Willi syndrome in ongoing study
This study tests an oral medication, pitolisant, in people with Prader-Willi syndrome who have already been in a pitolisant study. It aims to see if continuing treatment is safe and effective.
Morrisville, North CarolinaAges 7 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+ - 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.
Durham, North CarolinaAges 2–17 - NCT06846606RecruitingPhase 1
Study of AUTX-703 for advanced blood cancers
This trial tests an experimental drug, AUTX-703, for people with certain blood cancers (AML or MDS) that have returned or not improved after other treatments. The goal is to see if the drug is safe and helps control the disease.
Chapel Hill, North CarolinaAges 18 years+ - 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+ - 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 - NCT06641414RecruitingPhase 3
Lisaftoclax with azacytidine for higher-risk myelodysplastic syndrome
This trial tests a new drug called lisaftoclax combined with a standard treatment, azacytidine, for people newly diagnosed with higher-risk myelodysplastic syndrome (MDS). The goal is to see if the combination helps control the disease better than what's currently available.
Charlotte, 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+ - 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 - NCT06499285RecruitingPhase 3
Study of a new drug to treat anemia in adults with low to intermediate risk MDS who need transfusions
This study tests a new drug called elritercept for people with myelodysplastic syndromes (MDS) who have low blood counts and need regular blood transfusions. The drug aims to boost red blood cell production and reduce or eliminate the need for transfusions, which can improve energy and quality of life.
Winston-Salem, North CarolinaAges 18 years+ - NCT01522183Recruiting
Registry for people with atypical hemolytic uremic syndrome
This is a registry that collects information from people diagnosed with atypical hemolytic uremic syndrome (aHUS). It helps researchers better understand the condition across different genetic causes and treatment needs.
Winston-Salem, North CarolinaAges Any age - 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+ - 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 - NCT06066502RecruitingPhase 3
Precision ventilation for ARDS
This study tests whether a customized breathing machine approach ("precision ventilation") is better than usual care for patients with ARDS. The goal is to improve oxygen levels and recovery while avoiding lung damage.
Winston-Salem, 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.
Wilmington, North CarolinaAges 21 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 - 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+ - 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 - 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+ - 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+ - 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
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 · 8 studies
- Science 37 · 2 studies
- University of North Carolina at Chapel Hill · 2 studies
- Wake Forest University Health Sciences · 2 studies
- Arthritis and Osteoporosis Consultants of The Carolinas - Charlotte - 1918 Randolph Rd
- Ascentage Investigative Site
- Atrium Health Musculoskeletal Institute
- Atrium Health Sanger Heart and Vascular 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 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.