Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,858 recruiting studies in this area right now.
By phase
- Not applicable2,771
- Phase 2284
- Phase 1235
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome55
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease38
- Hemophilia A38
- Cystic Fibrosis (CF)37
- Cardiac Amyloidosis35
- Huntington Disease32
- Lynch Syndrome32
- Heart Failure31
- Retinitis Pigmentosa31
- Bronchopulmonary Dysplasia30
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)27
Leading sponsors
- Assistance Publique - Hôpitaux de Paris62
- Boston Children's Hospital42
- National Institute of Allergy and Infectious Diseases (NIAID)38
- Mayo Clinic37
- Children's Hospital Medical Center, Cincinnati33
- Hospices Civils de Lyon32
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Riphah International University26
- Massachusetts General Hospital23
- National Cancer Institute (NCI)23
Where studies are running
- California341
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona78
- Texas72
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT06466304Recruiting
Breathing muscle training for children with beta thalassemia major
This study tests whether special breathing exercises can help children with beta thalassemia major. It aims to strengthen the muscles used for breathing, which may improve overall health.
GizaAges 8–12 - NCT06467344RecruitingPhase 1/Phase 2
Testing ACDN-01 for ABCA4 Stargardt eye disease
This trial tests an experimental medicine called ACDN-01 for people with Stargardt retinopathy caused by ABCA4 gene mutations. The goal is to see if it can slow or stop vision loss in an eye with already damaged central vision.
San Francisco, CaliforniaAges 12 years+ - NCT06468774Recruiting
Test for gut ischemia in chronic belly artery disease
This study tests a blood marker for chronic mesenteric ischemia (poor blood flow to the gut). If it works, it could help doctors diagnose the condition faster and more easily.
Oslo, OsloAges 18 years+ - NCT06469580Recruiting
Lung ultrasound to guide breathing tube removal in newborns
This study tests whether using lung ultrasound helps doctors decide the best time to remove a breathing tube from preterm and full-term newborns. It may reduce the chance of needing to put the tube back in.
Graz, StyriaAges 1 minute–6 months - NCT06469918Recruiting
Support community for congenital heart defects
This trial is for people affected by congenital heart defects. It offers a support community (Co-Op) to help you connect, share, and get resources.
Rochester, MinnesotaAges birth+ - NCT06475651Recruiting
DNA patterns in rare prenatal diseases
This study looks at DNA patterns in fetuses or children with rare genetic conditions. It aims to better understand how these patterns relate to diseases that start before birth, which could help with diagnosis and future care.
ParisAges birth–18 years - NCT06475976Recruiting
Identifying lung types in severe BPD in preemies
This study looks at babies born very early who have severe lung disease (grade 3 BPD) and still need a breathing machine. Researchers will use tests to understand their lung problems better, which may help guide their care.
Philadelphia, PennsylvaniaAges 1 month–1 year - NCT06475898Recruiting
Tracking Huntington's disease burden over time
This study follows people with Huntington's disease (or those who carry the gene) over time using online surveys. It aims to better understand how the disease progresses, which could help improve future care.
Rochester, New YorkAges 18 years+ - NCT06478238RecruitingEarly Phase 1
Calcium folinate for hereditary spastic paraplegia type 56
This trial tests a drug called calcium folinate (a form of vitamin B9) to see if it can help people with a rare genetic type of spastic paraplegia (SPG56). It aims to improve leg stiffness and weakness caused by a specific gene change.
Shanghai, Shanghai MunicipalityAges Any age - NCT06479343Recruiting
Liquid embolic for brain vessel malformations
This trial tests a new liquid material (called a liquid embolic system) that is injected into the abnormal blood vessels in the brain to block them off. It is for people who have a cerebrovascular malformation and can be treated with a catheter from inside the blood vessel.
Guangzhou, GuangdongAges 18–80 - NCT06479616Recruiting
Long-term follow-up for CS-101 recipients
This study checks on participants who previously received the experimental treatment CS-101. It aims to track their health over time to see how safe and effective the treatment is in the long run.
Shanghai, Shanghai MunicipalityAges 3–17 - 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.
New Haven, ConnecticutAges 18 years+ - NCT06808477RecruitingPhase 1
BBT001 for moderate to severe eczema
This trial tests an experimental drug called BBT001 for people with moderate to severe eczema (atopic dermatitis) that hasn't been helped enough by creams. The study is in early phases, so it will check how safe the drug is and how well it might work.
Irvine, CaliforniaAges 18–72 - NCT07692256RecruitingPhase 2
Empagliflozin for heart failure in children
This trial tests if empagliflozin, a diabetes medicine that also helps the heart, can improve symptoms in children with heart failure. It may help kids with severe symptoms or high levels of a heart stress marker called BNP.
Cairo, Cairo GovernorateAges 1 month–18 years - NCT06420336Recruiting
Pain relief after cleft palate surgery
This study compares two ways to manage pain after cleft palate repair surgery that uses a bone graft from the hip. The goal is to see which method provides better pain control for children and teens.
Charleston, South CarolinaAges 6–18 - NCT07692061Enrolling by invitation
Speech therapy study for Cantonese-speaking kids with cleft palate
This study tests a new speech therapy program for children and teens (ages 4–17) who had cleft palate repair before age 2 and have certain speech sound errors. It aims to improve their speech clarity and communication.
Shatin, New TerritoriesAges 4–17 - NCT07062588RecruitingPhase 2
AGA2115 for osteogenesis imperfecta in adults with COL1A1/COL1A2 variations
This trial tests a new drug called AGA2115 for people with osteogenesis imperfecta (also known as brittle bone disease) who have a confirmed genetic change in the COL1A1 or COL1A2 genes. The study aims to see if the drug can help strengthen bones and reduce fracture risk.
Phoenix, ArizonaAges 18–75 - NCT06880016Recruiting
Lung ultrasound after surfactant in preterm babies
This study uses lung ultrasound to see how different ways of giving surfactant (a medicine for breathing problems) affect preterm babies born before 32 weeks. It may help doctors choose the best method for your baby.
Konya, KaratayAges 1 day–5 days - NCT06487624RecruitingPhase 1
Study of HCB301 for advanced solid tumors and Hodgkin lymphoma
This trial tests a new drug called HCB301, which targets proteins that help cancer hide from the immune system. It is for people with advanced solid tumors or classical Hodgkin lymphoma who have tried other treatments without success.
Greenville, South CarolinaAges 18 years+ - NCT06488274Recruiting
Study of a new infant formula for digestive issues in babies
This trial is testing a new infant formula to see if it helps with common digestive problems like spitting up, colic, or constipation in babies up to 4 months old. It aims to improve symptoms and comfort for infants who are formula-fed.
NiceAges Up to 4 months - NCT06488313RecruitingPhase 2
Study of ARCT-810 for people with OTC deficiency
This trial tests an experimental medicine called ARCT-810 for people with Ornithine Transcarbamylase (OTC) deficiency, a condition that affects the liver's ability to remove ammonia from the body. The study aims to see if ARCT-810 can help lower ammonia levels safely.
Chevy Chase, MarylandAges 12 years+ - NCT06488742Recruiting
Kanuka oil vs. hydrocortisone for eczema
This trial tests if a cream made from kanuka oil works better than a mild steroid cream (hydrocortisone) to treat eczema. It looks at how much the itching and skin rash improve.
Boynton Beach, FloridaAges 12–75 - NCT06488924RecruitingPhase 1/Phase 2
Study of JR-446 for Sanfilippo syndrome type B
This trial tests an experimental drug, JR-446, for children with Sanfilippo syndrome type B (MPS IIIB). The goal is to see if it is safe and can help with symptoms.
HiroshimaAges Up to 17 years - NCT06490627RecruitingPhase 2
thalidomide for transfusion-dependent beta thalassemia
This trial tests different doses of thalidomide to see if it can reduce or eliminate the need for blood transfusions in people with beta thalassemia major or intermedia.
Karachi, SindhAges 8–35
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,858 recruiting medical genetics studies from the U.S. registry right now, out of 5,764 we cover in this area in total.
- What does “Genetic & congenital” cover here?
- Inherited and congenital conditions present from birth or written into the genes. We group each study into genetic & congenital based on the conditions it studies, so you can scan the whole therapeutic area at once and drill into a specific condition.
- How do I find a genetic & congenital trial near me?
- Use the search to filter genetic & congenital studies by location and eligibility. Every Clin2 trial page explains the criteria in plain English and offers a short, optional pre-screen for a fit read — the study team makes the final decision.
Other specialties
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov; specialty groupings are derived from each study’s conditions. Talk with your doctor about whether a specific trial is right for you.