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
- NCT07435168Recruiting
Chest pain and hidden cardiac amyloidosis study
This study looks for signs of a condition called cardiac amyloidosis in people who have chest pain but no blocked heart arteries. It aims to find hidden cases that might be missed with standard tests.
CopenhagenAges 65 years+ - NCT07438691Recruiting
Comparing surgical methods for children with anorectal malformations
This study compares different surgical techniques for children with a specific type of anorectal malformation (perineal or vestibular fistula). It aims to see which surgery leads to better outcomes.
Florence, FlorenceAges birth–18 years - NCT07442669Recruiting
Lung ultrasound for surfactant decision in premature babies
This trial uses a lung ultrasound score, a type of ultrasound, to decide when to give surfactant (a medicine that helps lungs work) to premature babies born between 27 and 35 weeks of pregnancy. It aims to reduce the need for more breathing support and avoid side effects.
Hackensack, New JerseyAges 6 months–8 months - NCT07436312Recruiting
Effect of ultra-processed foods on cancer risk in high-risk people
This trial looks at whether eating less ultra-processed foods can lower cancer risk in people who are already at high risk for cancer. It is part of a larger program called Interception that helps monitor and reduce cancer risk.
AvignonAges 18 years+ - NCT07444333Recruiting
Heart function and fatigue in Friedreich's ataxia
This study looks at how the heart works during exercise and how that relates to fatigue in people with Friedreich's ataxia. You'll ride a stationary bike, and we'll measure your heart's output to understand fatigue better.
New York, New YorkAges Any age - NCT07442682Enrolling by invitation
Studying cystic fibrosis in adults at a Belgian center
This trial gathers information about adults with cystic fibrosis at the Erasme Hospital in Belgium. Your participation helps doctors better understand the condition and how it affects quality of life.
BrusselsAges 18 years+ - NCT07442851Recruiting
SickleFit exercise and nutrition study for sickle cell
This study looks at whether a personalized exercise and nutrition program can help people with sickle cell disease feel better and have fewer pain crises. It is for adults 35 and older with any type of sickle cell disease.
Durham, North CarolinaAges 35 years+ - NCT07436351Recruiting
ACT With CF Self-Help Toolkit
This trial tests an online self-help program based on acceptance and commitment therapy (ACT) for adults with cystic fibrosis who are feeling depressed or anxious. It aims to help you manage these feelings and improve your well-being.
Philadelphia, PennsylvaniaAges 18 years+ - NCT05773638Recruiting
Study blood clots and heart disease in adults with Von Willebrand
This study looks at cardiovascular problems and blood-clot events (like deep vein clots or pulmonary embolism) in adults with Von Willebrand disease in western France. It may help doctors better understand who is at risk and how to manage these complications.
AngersAges 18 years+ - NCT06228924RecruitingPhase 1
Gene therapy for ARVC with PKP2 mutation
This trial tests a new gene therapy called TN-401 for people with ARVC caused by a specific PKP2 gene mutation. The goal is to see if the treatment is safe and can help reduce dangerous heart rhythms.
San Francisco, CaliforniaAges 18–65 - NCT06568471RecruitingPhase 3
Study of HST101 for high cholesterol in Chinese patients
This study tests a new injectable medication called HST101 for lowering cholesterol in people who are already taking cholesterol pills but still have high levels. It may help if you have heart disease or are at high risk.
Beijing, Beijing MunicipalityAges 18 years+ - NCT07431112RecruitingPhase 1
Study of AIR-001 for adults with alpha-1 antitrypsin deficiency
This study tests an experimental RNA-based medicine (AIR-001) for people with a genetic form of alpha-1 antitrypsin deficiency (AATD). It aims to see if the drug can help boost your body's own protective protein levels and may slow lung damage.
MelbourneAges 18–74 - NCT07431437Enrolling by invitation
GENOSS paclitaxel balloon for leg artery disease
This trial tests a special balloon coated with paclitaxel (a drug that helps keep arteries open) in people with narrowed or blocked leg arteries. It aims to see how well the balloon works over one year.
Seoul, SeoulAges 19–85 - NCT07429240RecruitingPhase 1/Phase 2
Gene editing trial for Duchenne muscular dystrophy (ages 2-7)
This trial tests a new gene editing treatment called PBGENE-DMD for boys with Duchenne muscular dystrophy (DMD) whose genetic mutation falls within a specific range of the dystrophin gene (exons 45-55). The goal is to see if it is safe and can help improve muscle function.
Little Rock, ArkansasAges 2–7 - NCT07432880Recruiting
Study of children with severe MMA linked to the MMUT gene
This study is for children up to 16 years old with a severe form of methylmalonic acidemia (MMA) caused by a specific gene change (MMUT). It aims to better understand the disease and may lead to future treatments. Both children who have had a liver transplant and those who have not can join, as long as certain criteria are met.
Philadelphia, PennsylvaniaAges Up to 16 years - NCT07428499RecruitingPhase 3
Long-term safety of ADX-324 for hereditary angioedema
This study is for people who already finished a previous trial of the drug ADX-324 for hereditary angioedema. It tests the long-term safety and effectiveness of continuing the medication.
Litchfield Park, ArizonaAges 18 years+ - NCT07426419RecruitingPhase 1/Phase 2
Gene therapy for heart failure caused by BAG3 mutation
This trial tests an experimental gene therapy (AFTX-201) designed to address the genetic cause of heart failure in people with a specific BAG3 gene mutation. It aims to improve heart function and reduce symptoms.
Miami, FloridaAges 18–70 - NCT02097758Recruiting
3D heart ultrasound to guide a device closure for ASD
This trial looks at whether a special 3D ultrasound picture helps doctors safely place a small device to close an atrial septal defect (ASD), a hole in the heart’s upper chambers. If you need ASD closure and are suitable for a device, the results could help improve guidance and outcomes.
SeoulAges 18–80 - NCT07432867RecruitingPhase 1/Phase 2
Gene therapy for severe sickle cell disease in teens and adults
This trial tests a new gene therapy for sickle cell disease. It uses your own blood stem cells (collected from bone marrow) that are modified in a lab to help produce healthy red blood cells, then given back to you after a short chemotherapy. The goal is to reduce or stop severe complications like pain crises and lung problems.
Paris, Île-de-France RegionAges 12–35 - 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+ - NCT07425769Recruiting
Adapting a program for adults with Down syndrome and caregivers
This study tests a program to help adults with Down syndrome and their caregivers become more active. It aims to improve health and well-being for both.
Kansas City, KansasAges Any age - 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+ - NCT07428902Recruiting
French method with or without brace for clubfoot
This study compares two ways to treat clubfoot in babies: one using the French method alone, and the other adding a brace. It aims to see which approach helps better without surgery.
Lahore, Punjab ProvinceAges 1 day–1 year - NCT07435116Enrolling by invitation
Duchenne Muscular Dystrophy and Upper Limb Muscle Stiffness Study
This study looks at how Duchenne Muscular Dystrophy (DMD) affects the stiffness of muscles in the upper limbs (arms and shoulders). The goal is to better understand muscle changes and help improve care.
Gaziantep, GaziantepAges 5–18
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.