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
- NCT07414446Recruiting
Detecting lung blood flow issues with a new ultrasound device
This trial uses a non-invasive ultrasound device to detect abnormal blood flow connections in the lungs, which are common in people with hereditary hemorrhagic telangiectasia (HHT) or a type of stroke called cryptogenic stroke. The goal is to see if the device can find these issues safely and easily without needing an invasive procedure.
Nieuwegein, UtrechtAges 18 years+ - NCT07414511Recruiting
Hemophilia A research study for pregnant families
This study follows pregnant people who carry a severe hemophilia A gene and their babies after birth. It looks at how hemophilia A is passed down and how it affects families, with the goal of improving understanding and care.
Seattle, WashingtonAges Any age - NCT07411716Recruiting
Children's liver disease registry in Canada
This study creates a registry to track children with specific genetic liver conditions that cause cholestasis (slow bile flow). Being in the registry helps researchers better understand these diseases and may help future patients.
Calgary, AlbertaAges Up to 18 years - NCT07414251Recruiting
Preparing children for heart procedures
This study looks at how children prepare for heart surgery or a heart catheterization. It will ask families to fill out questionnaires to help improve preoperative care.
Berlin, State of BerlinAges Up to 18 years - NCT07412821Enrolling by invitationPhase 1
Study of ASA-001 for ADSS1 deficiency muscle disease
This trial tests a drug called ASA-001 (adenylosuccinic acid) for people with a rare genetic muscle disease caused by ADSS1 deficiency. The goal is to see if it is safe and helps muscle function.
Los Angeles, CaliforniaAges 18 years+ - NCT07414069Recruiting
Laser therapy for carpal tunnel syndrome
This trial tests high-intensity laser therapy as a treatment for mild to moderate carpal tunnel syndrome. It aims to see if the laser can improve symptoms and hand function without surgery or injections.
Ankara, Ankara, ÇankayaAges 18–65 - NCT06821776Recruiting
Comparing feeding methods for infants with severe lung disease
This study compares two ways of feeding infants with severe bronchopulmonary dysplasia (a chronic lung condition in premature babies) to see which helps with feeding problems such as reflux or aspiration. Infants will try both methods at different times to find out which works best.
Memphis, TennesseeAges 1 month–1 year - NCT06823219Recruiting
Long-term endurance training for sickle cell disease
This study tests whether a long-term endurance exercise program can improve physical fitness, reduce complications, and enhance quality of life for people with sickle cell disease. It is designed for patients whose condition is stable but who have had at least one pain crisis in the last three years.
CréteilAges 15 years+ - NCT05099315Recruiting
Gut microbiome study in adults with severe eczema
This trial studies the gut microbiome in people with moderate to severe atopic dermatitis (eczema) who are starting or already receiving systemic (whole-body) treatment. It may help researchers understand what changes in gut bacteria are linked to eczema and to treatment response.
Berlin, State of BerlinAges 18 years+ - NCT05525039Recruiting
Vibration plus supplement trial for weak muscles and nerves
This trial studies whether a vibration treatment combined with an HMB supplement can improve weak muscle quality and reduce early nerve-to-muscle connection problems. It is mainly for older adults who already have signs of low muscle strength or function.
Hong KongAges 60 years+ - NCT06082050RecruitingEarly Phase 1
YOLT-201 for transthyretin heart disease
This trial tests a new intravenous medicine called YOLT-201 for people with a heart condition caused by buildup of a protein called transthyretin (ATTR-CM). The goal is to see if it can slow or improve the disease.
Hangzhou, ZhejiangAges 18–80 - NCT06313827Recruiting
Online program to prevent flare-ups in cystic fibrosis
This trial tests an online program designed to help people with cystic fibrosis avoid exacerbations (sudden worsening of symptoms). You participate from home using a device with internet, camera, and microphone.
ValenciaAges 6 years+ - NCT06345976Recruiting
How albinism affects daily vision and activities
This study is for adults with any type of albinism. It looks at how albinism affects your everyday vision and activities through a simple questionnaire.
ParisAges 18 years+ - NCT06770010Recruiting
Digital brain training for kids with sickle cell disease
This trial is testing a digital program to help improve working memory (the ability to hold and use information in your mind) for children with sickle cell disease. If you join, you'll use a computer-based training program at home and researchers will see if it works and is easy to use.
Washington D.C., District of ColumbiaAges 7–16 - NCT07404111RecruitingPhase 1/Phase 2
Study of VV-14305 for thyroid eye disease
This study tests an investigational medication for thyroid eye disease, which causes bulging eyes and discomfort. The treatment is given as an injection near the eye, and the study will last about 12 months to see how safe and effective it is.
Livonia, MichiganAges 18–80 - NCT07399028Recruiting
Skin changes from biostimulator treatment in women
This study looks at how a biostimulator (a type of injectable treatment that helps your skin produce more collagen) changes your skin. Researchers will use special cameras and scans to see these changes, which may help reduce wrinkles on your cheeks.
New York, New YorkAges 45–65 - NCT07399080Enrolling by invitation
Study of a drug-coated balloon for leg artery disease
This trial is testing a special balloon coated with a drug (paclitaxel) to treat narrowed or blocked arteries in the leg. It aims to see how well this balloon works to keep the artery open.
SeoulAges 19–85 - NCT07403266RecruitingPhase 3
Full-spectrum cannabis oil for seizures in tuberous sclerosis
This trial tests a full-spectrum cannabis extract to see if it reduces seizures in people with tuberous sclerosis complex (TSC) whose seizures have not been controlled by other treatments. If it works, it could offer a new option for managing hard-to-treat epilepsy.
MadridAges 2–65 - NCT07401823Enrolling by invitationPhase 2
Long-term follow-up study for sickle cell disease treatment
This study is a follow-up for people with sickle cell disease who already saw a benefit in fetal hemoglobin from the earlier Pioneer study. It tests the long-term safety and effects of the study drug pociredir (also called FTX-6058) while allowing most standard sickle cell treatments except hydroxyurea.
Little Rock, ArkansasAges 18 years+ - NCT07399067RecruitingPhase 2
Testing IBI3002 for moderate to severe eczema
This study tests a new medication called IBI3002 for people with moderate to severe eczema (atopic dermatitis) that hasn't been well controlled by standard creams. The goal is to see if IBI3002 can reduce skin inflammation and itching.
Beijing, Beijing MunicipalityAges 18–75 - NCT07404644Recruiting
Study of vonicog alfa in children with von Willebrand disease
This study looks at how well vonicog alfa (a lab-made version of a missing blood protein) works to control bleeding in children under 18 with von Willebrand disease. It follows children who are already prescribed this treatment in Japan to see how safe and effective it is in real life.
Tokyo, TokyoAges Up to 17 years - NCT07400757Recruiting
Statin and vitamin D for active thyroid eye disease
This trial tests whether a statin (a cholesterol-lowering medicine) combined with vitamin D can improve active thyroid eye disease. It may help if you have both eye inflammation and high cholesterol.
Taipei, TaiwanAges 20 years+ - NCT06162338Recruiting
Study of LY-M001 for Gaucher Disease Type I
This study tests an injection called LY-M001 for adults with Gaucher disease type I. The goal is to see if it can safely help replace the missing enzyme and improve symptoms.
Hanzhou, ZhejiangAges 18–60 - NCT07400198Recruiting
Walking and Bone Health Study for SMA
This study looks at walking and bone health in people with spinal muscular atrophy (SMA). It helps researchers understand how SMA affects your gait (the way you walk) and your bones, which could lead to better treatments.
New York, New YorkAges 8–50
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.