Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,767 recruiting studies in this area right now.
By phase
- Not applicable2,684
- Phase 2280
- Phase 1241
- Phase 3210
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease134
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)38
- 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
- Massachusetts108
- Arizona87
- New York85
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,767 total
- NCT04040452RecruitingPhase 4
Continuous versus intermittent ketorolac for kids’ heart surgery pain
This study compares two ways of giving ketorolac (a pain medicine) to young children after heart surgery—continuous drip versus doses at set times. It aims to find which approach controls pain better with fewer side effects.
Phoenix, ArizonaAges 3 months–4.9 years - NCT04119908Recruiting
Test video microscopy to predict bleeding risk
This study uses special video imaging to try to predict when bleeding may happen in certain inherited bleeding disorders. It may help doctors better anticipate bleeding and guide safer care.
Lille, NordAges 18 years+ - NCT03325946Recruiting
Neuromotor research clinic for people with movement problems
This study is looking at people who have difficulties with movement or muscle control (neuromotor impairment). It may help researchers better understand these problems and improve future care.
Roanoke, VirginiaAges 3 months–18 years - NCT03952637RecruitingPhase 1/Phase 2
Gene therapy for GM1 disease with IV delivery
This early-stage study tests an IV gene therapy that delivers a working copy of an enzyme gene to help the body make beta-galactosidase in GM1 gangliosidosis. It may help slow or improve disease symptoms and looks closely at safety and how well the treatment works.
Bethesda, MarylandAges 6 months–12 years - NCT03937817Recruiting
Donate samples to study globin gene variants
This study collects common body samples to learn how different globin gene variants work and how they relate to health. Your samples may also be used later for new research, including genetic testing.
Bethesda, MarylandAges 18–70 - NCT04023019Recruiting
Hemophilia A trial for people with FVIII inhibitors
This trial studies a treatment approach for people with hemophilia A who develop antibodies (inhibitors) that block clotting factor FVIII. It may help those whose usual FVIII therapy is not working because of these inhibitors.
Atlanta, GeorgiaAges Any age - NCT03921060RecruitingPhase 4
Check osteoporosis markers in people with cystic fibrosis
This study looks for signs (markers) of bone thinning—called osteoporosis—in people with cystic fibrosis. Your participation may help researchers understand how osteoporosis develops in CF and whether a bone-strengthening medicine like denosumab could help, since some participants may need that medication.
Dallas, TexasAges 18–64 - NCT03882827Recruiting
Natural history study of Duchenne muscular dystrophy in young boys
This study follows boys with Duchenne muscular dystrophy over time to better understand how the condition changes and how standard treatments affect health and function. It does not appear to test a new medicine, but it may help doctors plan better future care and studies.
BrusselsAges 4–9 - NCT03981575Recruiting
Biomarker and health marker study in myotonic dystrophy type 1
This study measures physical and lab “health markers” in people with myotonic dystrophy type 1 (DM1) to better understand how the disease changes over time. Some participants may also have a muscle biopsy to study tissue differences.
La Jolla, CaliforniaAges 18–70 - NCT03893643Recruiting
Skin and mouth symptoms of NF2 in children under 15
This study looks at skin and mouth problems that children with NF2 may have. It helps doctors learn more about NF2 symptoms in kids, which may lead to better care.
NiceAges birth–15 years - NCT04003363Recruiting
Myotonic dystrophy registry to help track your condition
This is a national registry that collects information from people who have (or may soon have) myotonic dystrophy. By joining, you help researchers better understand the disease and may make future studies easier to access.
Newcastle upon TyneAges Any age - NCT03914664Enrolling by invitation
Brain and sensation study in adults with Tourette syndrome
This study looks at brain activity and how sensory experiences feel in adults with Tourette syndrome. It compares you to carefully matched adults without neurologic or psychiatric conditions to understand what is different in the brain.
Nashville, TennesseeAges 18 years+ - NCT03976622Enrolling by invitation
Inflammation study in vitiligo and other skin conditions
This study looks at inflammation in people with vitiligo, psoriasis, atopic dermatitis, or alopecia areata. It aims to understand how these conditions compare, which may lead to better treatments.
Bordeaux, BordeauxAges 18–75 - NCT04001582Recruiting
UK facial and shoulder muscle disease patient registry
This registry aims to collect health information from people in the UK who have facioscapulohumeral muscular dystrophy (FSHD). It helps researchers better understand the disease and plan future studies.
Newcastle upon TyneAges Any age - NCT03901521Enrolling by invitation
Biobank for people with autosomal dominant polycystic kidney disease
This study collects samples and health information from adults with autosomal dominant polycystic kidney disease (ADPKD) who are having their own kidney removed. It helps researchers learn more about ADPKD and its genetic changes to support future treatments.
New York, New YorkAges 18–100 - NCT03931707Recruiting
Genetic study of newborns in China
This study collects genetic information from very young newborns to better understand early-life health. Your child’s information may help researchers learn about genetics and future research.
Shanghai, Shanghai MunicipalityAges Up to 4 weeks - NCT03980717Recruiting
Balloon procedure for severe congenital diaphragmatic hernia in pregnancy
This trial tests whether placing a tiny balloon in the fetus’s windpipe can help lungs develop and improve severe pulmonary (lung) blood pressure in babies with severe congenital diaphragmatic hernia (CDH). It may lower the chance of breathing and circulation problems after birth, but it requires a specialized procedure and close follow-up in Houston.
Houston, TexasAges 18–45 - NCT04012411Recruiting
Testing brain fluid biomarkers in Huntington’s disease
This study looks at specific “biomarkers” (measurable signs) found in cerebrospinal fluid, to better understand Huntington’s disease. The results may help researchers track the disease and improve future tests or treatments.
MontpellierAges 18 years+ - NCT03937661RecruitingPhase 2/Phase 3
Platelet-rich plasma injected into ovaries for poor egg responders
This trial studies whether an injection of your own “platelet-rich” plasma into the ovaries can improve egg production in people who respond poorly to fertility treatment. It targets women with low egg reserve and tries to see if this approach helps them get better results from stimulation.
AthensAges 35–47 - NCT04002882Recruiting
Study how body fat pattern affects lung decline in cystic fibrosis
This study looks at how where your body stores fat may be linked to changes in metabolism and lung function over time in people with cystic fibrosis. It also compares results with healthy people similar in age, sex, and body size to understand the differences better.
Birmingham, AlabamaAges 16 years+ - NCT02879071Recruiting
Follow-up after blocking abnormal brain blood vessels
This study checks people long-term after a procedure called embolization, which blocks abnormal blood vessel connections in the brain (arteriovenous malformations). It may help doctors learn how well the treatment lasts over time and what outcomes to expect.
ParisAges 18 years+ - NCT03805919Recruiting
Study for men with high inherited risk of prostate cancer
This study is for men who have a known inherited gene change that raises their prostate cancer risk. It aims to help prevent or detect prostate cancer earlier, and you may be asked to undergo MRI and other study steps.
Bethesda, MarylandAges 30–75 - NCT03835767RecruitingPhase 2
Predicting food allergy from blood IgE levels in eczema patients
This study looks for blood test patterns that may predict whether a child with eczema (atopic dermatitis) is likely to have a food allergy. If you join, you may do supervised oral food challenges with a placebo and the suspected foods to see what triggers symptoms.
Bethesda, MarylandAges 3–21 - NCT03745924Enrolling by invitation
Study of Rebinyn for boys and men with Hemophilia B
This study follows males with Hemophilia B who are already on preventive (prophylaxis) treatment with Rebinyn/Refixia (a factor replacement medicine). It mainly looks at safety and how treatment is used over time, especially in people starting or continuing this prophylaxis plan.
ViennaAges Any age
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,767 recruiting medical genetics studies from the U.S. registry right now, out of 5,497 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.