Clinical trials · Raleigh–Durham, NC
Down Syndrome clinical trials in Raleigh–Durham
We’re tracking 88 recruiting down syndrome studies with a site in or near Raleigh–Durham — including Early Phase 1, 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 near Raleigh–Durham
- 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.
Durham, North CarolinaAges Up to 22 years - NCT05003986RecruitingPhase 2
Sparsentan for children with protein leaking kidney diseases
This Phase 2 study tests sparsentan in children with certain kidney diseases that cause high amounts of protein in the urine. It aims to see if the medicine can reduce kidney damage signals and protein leakage compared with standard care.
Durham, North CarolinaAges 1–17 - NCT07444567RecruitingPhase 3
Continuing benralizumab treatment after a previous study
This trial lets people who completed an earlier benralizumab study and are benefiting from the medicine keep receiving it. The goal is to provide ongoing access while we continue to monitor safety and effectiveness.
Durham, North CarolinaAges 6 years+ - 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.
Durham, North CarolinaAges 4 years+ - NCT03233646Recruiting
Retina scans in memory and nerve disorders
This study uses pictures of the back of your eye (retinal imaging) to look for differences in people with neurodegenerative diseases and compares them with people without these conditions. It may help researchers understand eye changes that could connect to brain or nerve health.
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+ - NCT03850574RecruitingPhase 1/Phase 2
Tuspetinib for relapsed or hard-to-treat AML
This early-phase study tests tuspetinib in people with AML that has come back or did not respond to treatment. The goal is to learn whether it is safe and tolerable, how the drug behaves in the body, and whether it shows signs of benefit.
Durham, North CarolinaAges 18 years+ - NCT05219617RecruitingPhase 3
Carisbamate for frequent drop seizures in Lennox-Gastaut
This Phase 3 study tests whether carisbamate can reduce drop seizures in people with Lennox-Gastaut syndrome (LGS). It also checks whether the treatment is safe when added to existing seizure medicines.
Durham, North CarolinaAges 4–55 - NCT04988555RecruitingPhase 1/Phase 2
Study of an investigational drug for high-risk acute leukemia
This Phase 1/2 study tests enzomenib (DSP-5336) in people with relapsed or hard-to-treat acute leukemia (and, in some places, certain other blood cancers). The goal is to see how the drug works and how safe it is, especially for leukemia cases with specific genetic changes (KMT2A/MLL or NPM1).
Durham, North CarolinaAges 12 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 - NCT04987775RecruitingEarly Phase 1
Study of NAC for Gulf War illness in veterans
This early-stage study tests the treatment called NAC to understand how it may work in veterans with Gulf War illness–related symptoms like fatigue and brain/mood changes. It also looks at “mechanisms,” meaning what’s happening in the body and brain while you’re in the study.
Durham, North CarolinaAges 47–70 - 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.
Durham, North CarolinaAges 18 years+ - NCT07038200RecruitingPhase 3
Study of Del-brax (AOC 1020) for People with FSHD
This study tests an experimental medicine called Del-brax (AOC 1020) for people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to see if it can help improve muscle strength and slow the disease. You may be able to join if you have a confirmed FSHD diagnosis and can walk at least 10 meters on your own.
Durham, North CarolinaAges 16–70 - 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.
Durham, North CarolinaAges 21 years+ - 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.
Durham, North CarolinaAges 18 years+ - NCT05564390RecruitingPhase 2
MyeloMATCH screening to match you to AML or MDS treatment
This study screens people with newly suspected acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) to see which treatment approach fits best—either a specific study treatment or standard care. It may help by directing you to the right option and by sharing your blood/bone marrow samples for research.
Durham, North CarolinaAges 18 years+ - NCT06154252RecruitingPhase 2/Phase 3
Study of CABA-201 for active myositis in adults and children
This trial tests a new treatment called CABA-201, which uses your own immune cells modified to attack the overactive cells causing myositis. It is for people with active disease that hasn't improved enough with standard therapies.
Durham, North CarolinaAges 6–75 - NCT06639984RecruitingPhase 2
Psyllium for kids with IBS
This study tests if psyllium, a fiber supplement, helps reduce belly pain and other symptoms in children with irritable bowel syndrome (IBS). It may be an option if standard treatments haven't worked well.
Durham, North CarolinaAges 12–17 - NCT05329935Recruiting
Registry for children with congenital thymus missing
This registry follows children born with congenital athymia (a missing or non-working thymus). It collects information around treatment with RETHYMIC, which may help researchers better understand how care and outcomes look over time.
Durham, North CarolinaAges birth–21 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+ - NCT05267821RecruitingPhase 2/Phase 3
Testing anakinra to reduce inflammation in pediatric sepsis MODS
This study tests whether a medicine called anakinra can “turn down” harmful inflammation in children with severe illness from sepsis. It aims to improve organ function when multiple organs are starting to fail.
Durham, North CarolinaAges 1 day–17 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 - 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+ - 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+
Hear when a new Down Syndrome trial opens near Raleigh–Durham, NC
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Down Syndrome trials in other cities
Where these studies are running near Raleigh–Durham
Institutions with a site for the recruiting down syndrome studies listed above.
- Duke University Medical Center · 12 studies
- University of North Carolina at Chapel Hill · 3 studies
- Duke Lung Transplant Clinic - Clinic 2F/2G - PPDS · 2 studies
- Durham VA Medical Center · 2 studies
- UNC Lineberger Comprehensive Cancer Center · 2 studies
- Ascentage Investigative Site
- Direct Biologics Investigational Site
- Duke Children's Hospital & Health Center
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.
Looking wider? See all recruiting down syndrome studies in North Carolina.
Common questions
- Are there down syndrome clinical trials near Raleigh–Durham?
- Yes. We're currently tracking 88 recruiting down syndrome studies with a site near Raleigh–Durham, 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 near Raleigh–Durham?
- 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 Raleigh–Durham to take part?
- Not necessarily. These studies have a site near Raleigh–Durham, 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 near Raleigh–Durham regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.