Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,756 recruiting studies in this area right now.
By phase
- Not applicable2,677
- Phase 2277
- Phase 1238
- Phase 3210
- Phase 1/Phase 2208
- Phase 497
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease134
- Atopic Dermatitis125
- Congenital Heart Disease84
- Cystic Fibrosis76
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy37
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)28
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
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
- Massachusetts General Hospital22
Where studies are running
- California325
- Maryland161
- Alabama133
- Massachusetts107
- Arizona86
- New York85
- Texas70
- Minnesota68
- Pennsylvania62
- Florida59
Recruiting studies3,756 total
- NCT07743671RecruitingPhase 3
Engasertib for frequent nosebleeds from HHT
This trial tests an experimental pill, engasertib, to reduce frequent nosebleeds in adults with a rare blood vessel condition called hereditary hemorrhagic telangiectasia (HHT). Participation may help if you have moderate to severe nosebleeds and need iron or blood transfusions.
Chicago, IllinoisAges 18 years+ - NCT07745712Recruiting
Testing a robotic exoskeleton for children with walking difficulties
This trial tests a robotic exoskeleton called ABLE Kids to help children with walking difficulties due to neurological or muscle conditions. It checks if the device is safe and works well. Children who meet certain size and health requirements may be eligible to participate.
Girona, GironaAges Up to 17 years - NCT06607471Recruiting
Study on non-ischemic heart muscle diseases
This study looks at different types of heart muscle diseases that are not caused by blocked arteries (non-ischemic cardiomyopathies). It aims to better understand how to diagnose, predict outcomes, and treat these conditions by gathering information from many patients over time.
Milan, MilanoAges 18 years+ - NCT06056635Recruiting
Study of special telescopes for fetal surgery
This study tests two types of small cameras (fetoscopes) to see how well they work for surgery on a baby before birth. Doctors want to learn if these tools can safely perform fetal procedures.
Boston, MassachusettsAges 18–45 - NCT07742046Enrolling by invitation
Heart blood flow scan for congenital heart disease
This study tests a new MRI technique to measure blood flow in the heart without invasive procedures. It may help doctors better understand and manage congenital heart conditions.
LausanneAges 16 years+ - NCT07745920Recruiting
An online program for depression in people with adpkd
This study tests an internet-based program to help manage depression symptoms in people with ADPKD. The program may be a convenient way to get support for your mood without traveling to a clinic.
Rochester, MinnesotaAges 18–70 - NCT07747220Recruiting
Abdominal massage for newborn jaundice
This study tests if gentle belly massage can help reduce jaundice in healthy full-term newborns. It may help lower bilirubin levels and support your baby's recovery without extra medication.
Taichung, TaiwanAges birth–1 day - NCT07746427Recruiting
Mindfulness program on a phone or tablet for teens with eczema
This study tests a digital mindfulness program to help teens manage their eczema. It looks at whether this approach can reduce symptoms and improve quality of life.
New York, New YorkAges 13–17 - NCT07745218Recruiting
Testing a new companion therapy for LAMA2 muscular dystrophy
This study looks at a new protein that might help people with LAMA2-related muscular dystrophy. It uses stored samples to see if the protein could be a useful addition to future treatments.
MilanAges Any age - NCT03131726RecruitingPhase 3
Simvastatin for Graves eye disease in mild-to-moderate cases
This trial tests whether simvastatin (a cholesterol medicine) can improve eye symptoms from Graves’ eye disease. It is aimed at people with active mild-to-moderate disease and will also check safety with blood tests, heart/skin tolerance, and stomach health.
MalmöAges 18–70 - NCT06611787Recruiting
Registry study for congenital heart disease
This study collects information from people with congenital heart disease (a heart problem you're born with) over their entire life to better understand the condition and improve future care.
Beijing, Beijing MunicipalityAges Any age - NCT06028646Recruiting
Using CT scans to diagnose airway collapse in children
This trial tests if a special CT scan can help diagnose tracheomalacia (a condition where the airway collapses during breathing) in children who have already had a bronchoscopy.
New York, New YorkAges birth–18 years - NCT06616545Recruiting
French study on type 3 Glycogen storage disease
This study tracks the health of people with type 3 Glycogen storage disease. It may help doctors understand the disease better and plan better care.
ClamartAges Any age - NCT06452199Recruiting
Bifidobacterium infantis for newborns study (BEGIN)
This study gives healthy newborns a probiotic supplement called Bifidobacterium infantis to see if it helps with growth, immune health, and reducing inflammation. It may be a safe way to support your baby's early development.
Herning, Central JutlandAges 1 day–1 week - NCT07739914RecruitingPhase 3
Switching anti-rejection medicine after a liver transplant
This trial tests whether switching from a standard anti-rejection medicine to a different one (everolimus) about a year after a liver transplant can help protect your new liver with fewer side effects. If you qualify, you will be monitored closely for a few years to see how well the new medicine works for you.
Heidelberg, Baden-WurttembergAges 18–80 - NCT07738874Recruiting
Eye imaging study for premature infants at risk of retinopathy
This study looks at the tiny blood vessels in the back of the eye (retina) of premature infants who are at risk for retinopathy of prematurity, a condition that can harm vision. It aims to improve care and monitoring by using advanced eye imaging to see more detail than current methods.
Durham, North CarolinaAges birth–9 months - NCT07734090Recruiting
Understanding MADD over time
This study follows people with MADD over time to understand how the condition changes, with the goal of improving future care and treatment. It does not test a new medication – it collects information through visits and tests.
Aurora, ColoradoAges Any age - NCT07738107RecruitingPhase 1/Phase 2
Study of ATR 1072 for PRKAG2 syndrome
This trial tests a new medicine called ATR 1072 for people with PRKAG2 syndrome, a genetic heart condition. It aims to see if the drug is safe and can help manage symptoms.
Kansas City, MissouriAges 18–65 - NCT07734064RecruitingPhase 1
Safety of one eye injection for Stargardt vision loss
This study tests a single injection of ASP2020 in the eye to see if it is safe and if it can help improve vision for people with Stargardt disease or similar inherited eye conditions.
Phoenix, ArizonaAges 6 years+ - NCT07734415Enrolling by invitationPhase 2
Long-term safety of IMG-007 for moderate-to-severe eczema
This study checks the long-term safety and effectiveness of IMG-007 in people with moderate-to-severe atopic dermatitis (eczema) who have already finished a previous IMG-007 trial.
Lancaster, CaliforniaAges 18–75 - NCT05012475Recruiting
Test whether video games help arm movement in Rett syndrome
This trial looks at whether computer gaming can improve how well you can use your arms to control game actions in Rett syndrome. It checks whether the gaming tasks can be done safely based on your arm movement range.
Washington D.C., District of ColumbiaAges 4–60 - NCT06366815Recruiting
A study of Duchenne muscular dystrophy in boys who no longer walk
This study is for boys with Duchenne muscular dystrophy who can no longer walk. It aims to describe how the condition changes over time once walking is lost, without testing any new treatments.
Bosisio Parini, LcAges 8–35 - NCT07727538RecruitingPhase 3
Olezarsen for familial chylomicronemia syndrome in children and teens
This study tests a new medicine called olezarsen to lower very high triglycerides (fats) in the blood of children and teenagers with familial chylomicronemia syndrome (FCS), a rare genetic condition. The goal is to reduce the risk of pancreatitis and other complications.
Birmingham, AlabamaAges 2–17 - NCT07729137RecruitingPhase 2
Eye cell therapy for corneal swelling
This trial tests an investigational cell therapy called EO2002 for people with corneal edema (swelling of the clear front part of the eye) caused by endothelial dysfunction, which can happen with Fuchs dystrophy or after cataract surgery. The treatment aims to restore the inner layer of the cornea to reduce swelling and improve vision.
Torrance, CaliforniaAges 21 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,756 recruiting medical genetics studies from the U.S. registry right now, out of 5,439 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.