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
- NCT07852390Recruiting
Pacing after heart surgery in children
This study tests whether using a pacemaker after heart surgery helps children's hearts recover and pump more efficiently. It is for children under 12 years old who are having heart surgery at Lurie Children's Hospital.
Chicago, IllinoisAges Up to 12 years - NCT07850934Recruiting
Melatonin for high blood pressure in the lungs of newborns
This trial tests whether giving melatonin to full-term newborns with high blood pressure in their lungs (pulmonary hypertension) can help improve their breathing and oxygen levels. It may offer a new treatment option for babies with this condition.
Cairo, AbbassiaAges Any age - NCT05740579Recruiting
Storing eggs in teens with Turner syndrome variants
This study looks at freezing and storing eggs (cryopreservation) in children and teens with Turner syndrome or related genetic variants. It may help preserve the chance of having biological children later, if you are able to undergo the study procedures safely.
AarhusAges 2–17 - NCT05661318Recruiting
Study of heart health in Ballana adults
This study is looking at heart health in adults living in Ballana. Your involvement could help researchers understand local heart risks and needs in the community.
AswānAges 18 years+ - NCT06371937Recruiting
Heart and blood vessel disease biobank study
This study creates a collection of skin cells (turned into stem cells in a lab) from people with various heart and blood vessel conditions. It aims to find new ways to understand and treat these diseases.
London, OntarioAges 18–80 - NCT06107400RecruitingEarly Phase 1
RM-004 cell therapy for Hemoglobin H-Constant Spring disease
This early-phase trial tests a new cell therapy called RM-004 for people with Hemoglobin H-Constant Spring disease, a form of alpha thalassemia that requires regular blood transfusions. The goal is to see if the treatment is safe and can reduce or eliminate the need for transfusions.
Nanning, GuangxiAges 12–35 - NCT06437158RecruitingPhase 2
Bleomycin sclerotherapy for lymphatic malformations in children
This trial tests a medicine called bleomycin, injected into lymphatic malformations (abnormal fluid-filled growths) to shrink them. It is for children under 14 whose malformations are growing quickly, causing symptoms or problems, and cannot be removed by surgery alone.
Chengdu, SichuanAges Up to 14 years - NCT04286360Recruiting
Study blood findings in children with genetic rasopathies
This study looks at blood and other hematologic (blood-related) findings in children who are newly diagnosed with certain genetic conditions called rasopathies. It may help doctors understand what blood changes are common early on and how to monitor them.
AngersAges Up to 15 years - NCT07844876Recruiting
Improving pregnancy problem prediction for African mothers
This study looks at improving how doctors predict pregnancy problems in African women. It will follow first-time mothers early in pregnancy to find better ways to keep you and your baby safe.
KampalaAges 18 years+ - NCT07846345Recruiting
Case management support for ear reconstruction in microtia
This study looks at whether extra support and coordination (case management) can help people going through ear reconstruction for microtia. If you're planning this surgery, the study may help you understand and manage the process better.
ShanghaiAges 6–40 - NCT07844694RecruitingPhase 1
Low-dose sirolimus for patients with RUNX1 platelet disorder
This trial tests whether a low dose of sirolimus, a drug that calms the immune system, can help improve blood counts and reduce bleeding risk in people with a genetic condition called RUNX1 familial platelet disorder. If it works, it might lower the chance of developing serious blood problems later.
Portland, OregonAges 18 years+ - NCT03283852Recruiting
Genetic study for certain childhood hormone and development disorders
This study looks for new genetic causes of certain “development disorders” that involve hormone problems and how the body develops at puberty. It may help families understand the root cause, which can guide future care and testing.
ParisAges Any age - NCT05534568Recruiting
Parent support program for families affected by pregnancy substance use
This program aims to help mothers and young children get safer, better support services when substance use occurred during pregnancy. It may help connect you (or your child) to the right community resources and reduce risk of ongoing alcohol or drug exposure.
Oklahoma City, OklahomaAges 18 years+ - NCT06284577Recruiting
Quality of life and gut health in children with cystic fibrosis
This study looks at how a drug called ETI (a combination of three medicines) affects the quality of life and gut health of children with cystic fibrosis. If you or your child has the right type of CF and is between 2 and 18 years old, this trial may help us understand how treatment improves daily life and digestion.
OsloAges 2–18 - NCT07837336Recruiting
Nutrition assessment for children with protein metabolism disorders
This study looks at the nutrition, body composition, and diet of Filipino children with protein metabolism disorders. It aims to understand their current health to improve future care and treatment.
Batangas, BatangasAges birth–18 years - NCT07836452RecruitingPhase 1
Study of a new drug for healthy people and those with Stargardt disease
This study tests a new drug called CITY-RBP4 in healthy volunteers and in people with Stargardt disease (STGD1), a genetic condition that causes vision loss. The goal is to see if the drug is safe and how it affects the body, which may help guide future treatments for Stargardt disease.
Melbourne, VictoriaAges 12–45 - NCT05773729Recruiting
Gene-modified own stem cell treatment for transfusion-dependent thalassemia
This trial tests a one-time treatment that uses your own (autologous) stem cells that have been changed by gene methods, then returned to you to help manage transfusion-dependent beta-thalassemia. It aims to check safety and whether it can reduce the need for frequent blood transfusions or improve blood health.
ShanghaiAges 3–18 - NCT05776173Recruiting
Gene-modified stem cell treatment for transfusion-dependent thalassemia
This trial tests whether giving you your own (autologous) gene-modified blood stem cells can improve transfusion-dependent beta-thalassemia and its safety. It may help people who do not have a fully matched donor and need an alternative approach to care.
ShanghaiAges 6–35 - NCT07835607Recruiting
Comparing pet scans and mri for thyroid eye disease
This study compares two PET imaging methods and an MRI to see which better shows inflammation in thyroid eye disease. Participation may help improve detection and monitoring of this condition.
Chongqing, Chongqing MunicipalityAges 18–75 - NCT07834320Recruiting
Long-term study of Pompe disease for monitoring
This study follows people with Pompe disease over time, even if they are already receiving treatment. It aims to learn more about the disease and how patients are doing over the long run.
Taipei, TaiwanAges Any age - NCT07834840Enrolling by invitation
Diet education for gout patients on urate-lowering therapy
This trial tests whether extra diet advice helps people with gout who are already taking urate-lowering medicine. It might help them manage their condition better.
Suwon, Gyeonggi-doAges 19 years+ - NCT07841067Recruiting
3D-printed vs. milled plates for jaw surgery
This study compares two types of titanium plates (3D-printed vs. traditionally milled) used to hold the upper jaw in place after corrective jaw surgery. It aims to see which type is more accurate and reduces surgery time, which could mean a faster recovery for you.
Cairo, New Cairo, Fifth SettlementAges 18 years+ - NCT06464757Recruiting
Laryngomalacia and breathing in children: before and after treatment study
This trial is for children with laryngomalacia (a floppy voice box) who have noisy breathing and trouble breathing. It will check how they are doing before and after treatment, and follow up for one year to see how their quality of life improves.
OsloAges 1 week–12 months - NCT06466291Recruiting
Spanish Wilson Disease Registry
This study is a registry that collects information about people with Wilson disease. By joining, you can help researchers learn more about the condition and improve care for others.
BarcelonaAges 18 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.