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 Disease84
- 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
- California325
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York85
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT07491237Recruiting
Heart rhythm and function study
This study looks at how heart rhythm problems affect heart function. It may help doctors better understand and treat these conditions.
RomaAges 18 years+ - NCT07493096Recruiting
Intensive therapy program for children with brain-based conditions
This trial tests an intensive therapy program designed to help children with brain-based conditions like cerebral palsy or autism improve their skills in movement, thinking, and daily activities. The program runs for 2 weeks with daily sessions, and researchers will check for changes in your child's abilities.
The Woodlands, TexasAges 4–12 - NCT07495410Recruiting
Exercise program for people with Fabry disease
This study tests a structured exercise program designed to improve fitness in people with Fabry disease. It aims to see whether regular exercise is a safe and helpful way to support your health.
PragueAges 18 years+ - NCT07491172RecruitingPhase 1
Trial of CTX310 for stubborn high cholesterol and fats
This trial tests a new medicine, CTX310, for people with persistently high levels of fats (triglycerides) and cholesterol that have not improved with standard treatments. It aims to see if the drug is safe and tolerable.
Hialeah, FloridaAges 18–75 - NCT05702476Recruiting
Non-invasive 3D scan study for Marfan face features
This study uses a non-invasive 3D scan to better measure facial features in people with Marfan syndrome. It may help researchers describe face differences more accurately, without needles or surgery.
San Donato Milanese, LombardyAges Any age - NCT06227182Recruiting
Comparing MRI and ultrasound for muscle health
This study compares two imaging methods, MRI and ultrasound, to see how well they measure muscle health. It may help find better ways to monitor muscle conditions without invasive tests.
Nijmegen, GelderlandAges 18–70 - NCT06381713Recruiting
Speech therapy for children with cleft palate
This study compares two types of speech therapy for children with cleft palate. It looks at how well each type works and how much it costs, to help find the best way to improve your child's speech.
GhentAges 4–12 - NCT06688448Recruiting
New biologic aortic valve replacement study
This study is testing a new generation of biologic (tissue) replacement valves for the aortic valve in adults aged 18 to 65. It aims to see how well these newer devices work compared to standard options.
São Paulo, São PauloAges 18–65 - NCT06641154RecruitingPhase 1/Phase 2
Gene therapy for severe Crigler-Najjar syndrome
This trial tests a gene therapy for children with severe Crigler-Najjar syndrome type I. It aims to reduce the need for daily phototherapy by fixing the faulty gene.
MoscowAges 3 months–10 years - NCT06770218Recruiting
Pelvic taping for stability in Down syndrome
This trial tests whether taping the pelvic area can help children with Down syndrome feel more stable. It may improve their movement and balance.
Lahore, Punjab ProvinceAges 4–10 - NCT06773988Recruiting
Long-term monitoring of breathing support at home for Duchenne patients
This study follows adults with Duchenne muscular dystrophy who use a breathing machine (non-invasive ventilation or NIV) at home. It aims to track their health over time to improve care.
Leuven, Vlaams-BrabantAges 18 years+ - NCT06773546Recruiting
Immune system disorders study for children and adults
This study looks at people with known or suspected inborn errors of immunity (problems with the immune system that you are born with). The goal is to improve how these conditions are diagnosed and managed. If you have immune system issues that started before age 50, you may be able to join.
Bologna, BolgonaAges Up to 50 years - NCT06773650Recruiting
Managing lactose intolerance with diet and enzyme supplements
This trial tests whether diet tracking and lactase enzyme supplements help people with lactose intolerance manage their symptoms. It uses an online survey to gather information.
MilanAges 18 years+ - NCT07486934RecruitingPhase 3
Study of DYNE-101 for Myotonic Dystrophy Type 1
This trial tests an experimental drug called DYNE-101 to see if it can help people with myotonic dystrophy type 1 (DM1). The study aims to improve muscle function and daily life. It is for adults who can walk and stand up from a chair without help.
La Jolla, CaliforniaAges 16 years+ - NCT07489209RecruitingPhase 2
Study of EDP167 for homozygous familial hypercholesterolemia
This trial tests a new drug, EDP167, for people with homozygous familial hypercholesterolemia (HoFH), a genetic condition that causes very high cholesterol. The goal is to see if EDP167 can lower 'bad' cholesterol (LDL-C).
BeijingAges 18 years+ - NCT07488065RecruitingPhase 1
New drug for moderate to severe eczema
This trial tests a new injectable drug called SKB575 for people with moderate to severe atopic dermatitis (eczema). It aims to see how safe and effective the drug is, especially for those who haven't had success with strong creams or pills.
ShanghaiAges 18–70 - NCT06773715Recruiting
Virtual reality for pain from sickle cell disease
This study tests if using virtual reality (VR) can help children and teens with sickle cell disease feel better during a severe pain episode. You would use VR while in the hospital to see if it reduces your pain.
New Orleans, LouisianaAges 8–21 - NCT06772766RecruitingPhase 1
Study of 9MW3011 in adults with beta-thalassemia without regular transfusions
This trial tests a new drug, 9MW3011, in people with a type of beta-thalassemia that does not require regular blood transfusions. The goal is to see if the drug can safely help manage iron overload and improve hemoglobin levels.
Nanning, GuangxiAges 18–65 - NCT06019637Recruiting
Long-term safety study of Zolgensma for spinal muscular atrophy
This study follows the long-term safety of Zolgensma gene therapy in Brazilian children with spinal muscular atrophy (SMA). It helps doctors understand how safe the treatment is over time.
Curitiba, ParanáAges birth–100 years - NCT06582914Recruiting
Lynch syndrome genetics and health study
This study looks at the genetics and health of people with Lynch syndrome, a condition that raises the risk for certain cancers. Researchers want to learn more about how different genetic changes affect health over time.
Aurora, ColoradoAges 18 years+ - NCT07485543RecruitingEarly Phase 1
Inhaled genetic therapy for cystic fibrosis patients
This trial tests an inhaled medicine that delivers a corrective genetic message (mRNA) to lung cells in people with cystic fibrosis. It aims to see if the treatment is safe and can improve lung function over time.
Beijing, Beijing MunicipalityAges 12 years+ - NCT07487831RecruitingPhase 4
Salt intake and skin sodium in inflammatory skin disease
This study tests how salt in your diet affects sodium levels in your skin and its link to inflammatory skin conditions like eczema. It may help explain why some people's skin flares up after eating salty foods.
San Francisco, CaliforniaAges 18 years+ - NCT07483424Enrolling by invitation
Growth restriction in full-term babies: body composition study
This study looks at how body composition differs in full-term babies who had intrauterine growth restriction. It may help doctors better understand and care for these infants.
LisbonAges 9 months–9 months - NCT07484607Recruiting
Study on infections and treatments in cystic fibrosis exacerbations
This study looks at how lung infections and inflammation affect people with cystic fibrosis during severe flare-ups. It aims to understand which antibiotics work best and why, so your care team can choose the right treatment for you.
CardiffAges 16 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 5,456 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.