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
- NCT07307612RecruitingPhase 1/Phase 2
High-energy breast milk diets for extremely preterm infants
This trial tests whether feeding extremely premature babies (born at 28 weeks or earlier) a higher-energy breast milk diet in the first two weeks of life can reduce the risk of bronchopulmonary dysplasia (BPD), a serious lung condition. It aims to find a safe and effective way to support lung development in these tiny infants.
Birmingham, AlabamaAges 1 day–3 days - NCT07308704Recruiting
Using AI to spot heart muscle disease on echocardiograms
This study at Mayo Clinic is testing an artificial intelligence (AI) tool that analyzes echocardiograms (ultrasound of the heart) to find early signs of four types of heart muscle disease. If the AI flags a high-risk pattern, doctors can learn whether acting on that alert helps catch the condition sooner.
Rochester, MinnesotaAges 18 years+ - NCT07312734Recruiting
Testing new ways to find lung germs in CF
This study tests a new method to detect bacteria in the lungs of people with cystic fibrosis (CF) by analyzing mucus samples. It aims to improve how we diagnose and treat lung infections.
La Jolla, CaliforniaAges 16 years+ - NCT07314983Recruiting
Long-term blood thinning in severe hemophilia A
This study looks at using long-term blood thinners (anticoagulants) in adults with severe hemophilia A, a bleeding disorder. It aims to understand if this treatment can be safe and effective for patients who may need it.
StrasbourgAges 18 years+ - NCT07307664Recruiting
Increasing genetic testing for cancer patients
This trial aims to increase the use of genetic testing (germline testing) among cancer patients by working with healthcare providers like oncologists and nurses. If you are an oncology care team member treating adults with breast, pancreatic, or colorectal cancer, this study may be for you.
Los Angeles, CaliforniaAges Any age - NCT06875245RecruitingPhase 4
Comparing 8-Week vs 12-Week Faricimab for Wet AMD
This trial compares two different schedules (8 weeks vs 12 weeks) for follow-up injections of faricimab in people who are starting treatment for wet AMD. It aims to see if a longer gap between doses works just as well as the shorter interval.
PragueAges Any age - NCT02798705Recruiting
Testing heart tiny blood vessel function in amyloidosis
This study measures how well the heart’s tiny blood vessels work in people with cardiac amyloidosis, using special heart testing. It may help doctors understand the disease better and improve how patients are evaluated over time.
SeoulAges 18–85 - NCT06178887Recruiting
Cardiac MRI for heart and blood vessel issues in Turner syndrome
This study uses cardiac MRI to look for heart and blood vessel differences in adults with Turner syndrome. It may help find problems early so you can get the right care.
ModenaAges 18 years+ - NCT07301463Recruiting
Study of a new drug for children with achondroplasia
This trial tests a new medicine for children with achondroplasia (a common form of dwarfism). It aims to see if the drug can improve growth. The study is for children aged 2.5 to under 11 years old who have not started puberty and can walk without help.
Beijing, Beijing MunicipalityAges 2.5 years–11 years - NCT07303296RecruitingPhase 2
Gene therapy study for vision loss from LHON
This trial tests a gene therapy called GS010 given as an injection into the eye for people with vision loss from LHON. It aims to see if it can help improve vision in both eyes.
ParisAges 15 years+ - NCT07298044RecruitingPhase 4
Switching from tafamidis to acoramidis for heart amyloidosis
This study will see how switching from tafamidis to a newer medicine called acoramidis changes the level of a protein (called transthyretin) in your blood. The goal is to see if acoramidis might work better for people with this type of amyloidosis that affects the heart.
Sankt Pölten, Lower AustriaAges 18–90 - NCT07298395RecruitingPhase 2
Study of ENV-294 for moderate-to-severe eczema
This trial tests an experimental cream or pill called ENV-294 in adults whose eczema is moderate to severe and hasn't gotten better with standard creams. It aims to see if ENV-294 can help control symptoms like itching and skin redness.
Birmingham, AlabamaAges 18–75 - NCT07298447RecruitingPhase 3
Donidalorsen for children with hereditary angioedema
This study tests a new medicine, donidalorsen, to prevent swelling attacks in children ages 2 to 11 with hereditary angioedema (HAE). If your child has HAE type 1 or 2, this trial may help find a treatment option.
Santa Monica, CaliforniaAges 2–11 - NCT07297602RecruitingPhase 2
Oral roflumilast for moderate to severe eczema in teens and adults
This study tests an oral medicine called roflumilast to see if it helps treat moderate to severe eczema in people aged 12 and up who haven't used other systemic treatments recently. It's a pilot study to check both safety and how well it works.
Al Mansurah, Dakahlia GovernorateAges 12 years+ - NCT07301918Recruiting
Comparing two expanders for crossbite in children
This trial compares two types of expanders (Leaf Expander vs. Hyrax Expander) to fix crossbites in children who still have some baby teeth. The goal is to see which device works better and is more comfortable.
CairoAges 6–12 - NCT07305168Recruiting
Breathing after tonsil surgery in children with sleep apnea
This study looks at breathing problems that can happen after tonsil or adenoid surgery in children who have sleep apnea. Researchers want to learn more so they can make surgery safer for kids.
Edegem, AntwerpAges 1–18 - NCT07304193Enrolling by invitation
Study of sex chromosome disorders in fetuses
This study looks at the genetics of fetuses with sex chromosome disorders. It compares them to fetuses without these conditions to understand more about how these disorders affect development.
AarhusAges 3 months–5 months - NCT07303621Recruiting
Study of Trikafta in children with cystic fibrosis
This study looks at how the drug Trikafta (also called Kaftrio) works in children aged 2 to 17 with cystic fibrosis. The goal is to learn how the body processes the medicine so doctors can find the best dose.
BronAges 2–17 - NCT07303140Recruiting
Liver stiffness in young people with glycogen storage disease type III
This study looks at liver stiffness (a sign of scarring, called fibrosis) in children and young adults with type III glycogen storage disease. Researchers want to see how common liver fibrosis is in this group and how it changes over time, using a non-invasive ultrasound-like test called elastography.
StrasbourgAges 1 month–21 years - NCT07298564Recruiting
Magnesium and levocarnitine for PCOS symptoms
This trial tests whether taking magnesium and levocarnitine (a natural substance that helps cells make energy) can improve metabolism and reduce symptoms like irregular periods or weight gain in women with polycystic ovary syndrome (PCOS). If you have PCOS, you may be able to join.
Tehran, Tehran ProvinceAges 19–65 - NCT07301216Recruiting
Copper in urine after stopping Wilson disease therapy
This study is looking at what happens to copper levels in the urine when people with Wilson disease briefly stop their medicine. It may help doctors understand how well your body controls copper without treatment.
New Haven, ConnecticutAges 18 years+ - NCT06876857Recruiting
UCF Cerebrospinal Fluid Biobank for Adults
This is a research biobank at the University of Central Florida that collects and stores cerebrospinal fluid samples for future studies. It is open to most adults who can give their consent.
Orlando, FloridaAges 18 years+ - NCT05499741Recruiting
Heat-based therapy to improve sleep in adults with Tourette’s
This trial tests a forehead temperature-regulating therapy to help improve insomnia symptoms in adults who have Tourette’s (and significant tics). It may help you sleep better by testing whether changing forehead temperature can improve sleep quality and efficiency.
Los Angeles, CaliforniaAges 18–50 - 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+
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.