Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,762 recruiting studies in this area right now.
By phase
- Not applicable2,682
- Phase 2280
- Phase 1239
- Phase 3209
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease133
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy36
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)29
Leading sponsors
- Assistance Publique - Hôpitaux de Paris57
- Mayo Clinic39
- Boston Children's Hospital38
- National Institute of Allergy and Infectious Diseases (NIAID)38
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Children's Hospital Medical Center, Cincinnati31
- Hospices Civils de Lyon29
- Children's Hospital of Philadelphia23
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
Where studies are running
- California324
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York84
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT07434037Recruiting
Trust and the brain in Down syndrome and Fragile X
This study explores how people with Down syndrome or Fragile X syndrome learn to trust others. It uses simple tasks and a brain scan (MRI) to compare their responses with those of typically developing children and young adults.
BronAges 3–29 - NCT07431632Recruiting
Nerve freezing vs. epidural for pain after pectus excavatum repair
This study compares two ways to manage pain after pectus excavatum surgery: freezing the nerves during surgery (cryoanalgesia) versus an epidural block. The goal is to see which method provides better pain relief with fewer side effects.
São Paulo, São PauloAges 13 years+ - NCT07432490RecruitingPhase 2
Fucose supplement trial for GLUT1 deficiency
This trial tests whether taking fucose (a type of sugar) can help people with GLUT1 deficiency syndrome, a rare genetic condition that affects how the brain gets energy. If you have ataxia (trouble with balance or coordination) and meet other criteria, this study may be an option for you.
Portland, OregonAges 18 years+ - NCT06813443Recruiting
Studying cardiomyopathy to find who needs a heart transplant
This study is looking at people with cardiomyopathy to understand which patients are most in need of a heart transplant. By joining, you will help doctors learn more about critical cases and improve care for heart muscle disease.
Bologna, Emilia-Romagna/BolognaAges 12 years+ - NCT06815159Recruiting
Tissue markers for early AV fistula narrowing
This study looks at tissue samples from people getting or fixing an AV fistula for dialysis. The goal is to find markers that might predict early narrowing (stenosis) of the fistula, so doctors can prevent problems in the future.
BolognaAges 18 years+ - NCT06815380Recruiting
Study on lebrikizumab for people with atopic dermatitis
This study looks at how lebrikizumab (Ebglyss) affects daily life and skin symptoms in adults with moderate-to-severe eczema. You may join if your doctor has prescribed this medication as part of your regular care.
Aarhus, DenmarkAges 18 years+ - NCT05739253Recruiting
Study how the aorta changes after a valve procedure
This trial looks at how the ascending aorta (the main artery near the heart) changes over time after transcatheter aortic valve replacement. It may help doctors predict who is at risk of aorta enlargement after this less-invasive valve procedure.
Beijing, Beijing MunicipalityAges Any age - NCT06574282Recruiting
Better diagnosis for hypophosphatasia in rheumatology patients
This study aims to find better ways to diagnose hypophosphatasia (HPP) in adults who see a rheumatologist. If your doctor suspects HPP and you have a low ALP blood test, you may be able to help researchers develop a screening tool.
Bonn, North Rhine-WestphaliAges 18 years+ - NCT07422870Recruiting
Registry for granulomatous mastitis
This study is creating a registry (a database) for people with a rare inflammatory breast condition called granulomatous mastitis. It aims to learn more about the disease and how it affects people, without testing any new treatments.
Düsseldorf, North Rhine-WestphaliaAges 18 years+ - NCT07417111Recruiting
CPAP breathing support for very premature babies
This study looks at whether a careful CPAP breathing support plan helps very premature babies breathe better. It may be an option for babies who are stable on low oxygen settings and have few breathing pauses.
Birmingham, AlabamaAges Up to 7 months - NCT07421297RecruitingPhase 3
Study of SYH2053 for high cholesterol in Chinese adults
This trial tests a new medication called SYH2053 for people with high cholesterol or mixed high cholesterol and triglycerides. If you have high LDL cholesterol and are not taking strong cholesterol medicines, you may be able to join. The study will see if this drug is safe and works well.
Beijing, Beijing MunicipalityAges 18 years+ - NCT07419334RecruitingPhase 3
Study of a new drug for Stargardt disease
This trial tests a new drug, ALK-001, to see if it can slow down vision loss from Stargardt disease. It is for people who have a confirmed genetic diagnosis and are between 8 and 45 years old.
Phoenix, ArizonaAges 8–45 - NCT07416526RecruitingPhase 3
Testing NXT007 for hemophilia A without inhibitors
This study tests a new medicine called NXT007 for people with hemophilia A. It compares NXT007 to standard factor VIII treatment to see if it works better at preventing bleeding episodes.
Aurora, ColoradoAges 12 years+ - NCT07420907Recruiting
Tracking heart health over time
This study uses a wearable patch to monitor heart-related signs in people with heart conditions. It aims to learn more about how these conditions progress, which could help improve future care.
Chicago, IllinoisAges 18 years+ - NCT07416604RecruitingPhase 3
Study of NXT007 vs emicizumab for hemophilia A
This trial tests if a new medicine, NXT007, is better than emicizumab at preventing bleeding episodes in people with hemophilia A. It may be an option for those with or without inhibitors who are willing to switch to a preventive treatment.
Orange, CaliforniaAges 12 years+ - NCT07425171Recruiting
Testing a drug-coated balloon for long leg artery blockages
This trial is testing a special balloon coated with the drug paclitaxel to open up long blockages in the leg artery (femoropopliteal). The goal is to see if it is safe and effective for improving blood flow and symptoms like pain or ulcers.
Suwon, Gyeonggi-doAges 19–85 - NCT07417566RecruitingPhase 1
DC6001 Tablet Study in Healthy Chinese Adults
This trial tests a new tablet called DC6001 in healthy volunteers. It helps find out how the body processes the drug and if it is safe. You may join if you are a healthy adult between 18 and 55 years old.
Beijing, Beijing MunicipalityAges 18–55 - NCT06817148Recruiting
TAVR vs surgery for bicuspid aortic valve disease
This study compares two treatments for severe aortic stenosis in people with a bicuspid aortic valve: transcatheter aortic valve replacement (TAVR, a less invasive procedure) and surgical aortic valve replacement (SAVR, open-heart surgery). The goal is to see which approach works better for this specific condition.
Los Angeles, CaliforniaAges 50 years+ - NCT06816056Recruiting
Foot therapy for hemophilia ankle arthritis
This study tests if manual therapy can help patients with hemophilia who have arthritis in their ankle. It seeks to improve joint health and reduce pain.
MurciaAges 18–65 - NCT07417137Recruiting
Long-term study of Angelman syndrome
This study follows people with Angelman syndrome over time to learn more about the condition. It does not test any new treatments, but you need a parent or caregiver to come with you to all visits.
Lexington, MassachusettsAges 1 year+ - NCT07421323Recruiting
Hand cleaning study for premature babies in the NICU
This study tests if a special hand-cleaning method using chlorhexidine and alcohol can help prevent infections in premature babies (born before 37 weeks) in the newborn intensive care unit (NICU). It aims to see if this cleaning approach is better than the usual method for keeping babies safe from hospital-acquired infections.
AlexandriaAges 6 hours–1 day - NCT07423013Recruiting
Teprotumumab treatment for active thyroid eye disease
This trial tests a new version of an antibody treatment (teprotumumab) for people with active thyroid eye disease. It aims to reduce bulging and inflammation of the eyes, and you must have had symptoms for less than 9 months to join.
BeijingAges 18–80 - NCT07423026Recruiting
Remote study using technology to track DMD in young boys
This study uses remote technology, like apps or wearable devices, to monitor motor skills and development in boys with Duchenne muscular dystrophy (DMD). It aims to find better ways to measure how the disease progresses over time without requiring frequent clinic visits.
OxfordAges 1–3 - NCT07420400Recruiting
Detecting heart shunts without needles
This trial uses a specialized ultrasound test (TEE with bubble study) to find a small opening in the heart called a PFO. It may help people who need this test to check for causes of certain health problems.
La Jolla, CaliforniaAges 18 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,762 recruiting medical genetics studies from the U.S. registry right now, out of 5,482 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.