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
- NCT07716488Recruiting
Screening for high cholesterol in children with chronic conditions
This study screens children and teens with certain chronic conditions (like heart defects or inflammatory bowel disease) for early signs of high cholesterol or other lipid problems. The goal is to find and manage these risks sooner.
JenaAges 6–18 - NCT07720570Enrolling by invitation
Bleomycin electrosclerotherapy for slow-flow vascular malformations
This trial tests a treatment called bleomycin electrosclerotherapy for people with slow-flow vascular malformations (abnormal blood vessels that do not flow quickly). The goal is to see if this treatment works well and is safe for these conditions.
Copenhagen, Capital Region of DenmarkAges 2 years+ - NCT07718997RecruitingPhase 1
Social skills training for people with sex chromosome differences
This trial tests a social management training program for adults with sex chromosome aneuploidies (SCA). The goal is to see if it can help improve social skills and daily life. It is a small feasibility study to prepare for larger future trials.
OsloAges 16–65 - NCT07716397Recruiting
MRI study for thyroid eye disease diagnosis and care
This study uses MRI scans of the eye area to better understand and classify thyroid eye disease (TED). It aims to help doctors predict outcomes and personalize treatment. You may be eligible if you have stable TED or are a healthy volunteer.
Shanghai, Huangpu DistrictAges 18–75 - NCT07715617Recruiting
Creating a prediction score for lung transplant or death in Alpha-1 Antitrypsin Deficiency
This study is creating a simple scoring system to predict who with Alpha-1 Antitrypsin Deficiency-related emphysema might need a lung transplant or die from the disease. Your medical records from 2010–2025 will help build this tool, with no extra tests or visits required.
BronAges 18 years+ - NCT06641011Recruiting
Sealing molars with enamel spots: rinsing vs blotting
This study tests whether rinsing off or blotting dry a special silver fluoride liquid before placing a tooth sealant helps the sealant last longer on molars affected by a common condition called molar-incisor hypomineralization. The goal is to find the best way to protect these teeth from cavities.
AnkaraAges 6–14 - NCT06362629Recruiting
Smartphone App for Managing Eczema
This trial tests a smartphone app designed to help people manage their atopic dermatitis (eczema). If you or your child has eczema and can use a smartphone, this app may help track symptoms and provide advice.
Chengdu, SichuanAges 1–75 - NCT06557551Recruiting
Surfactant for preterm babies born before 28 weeks
This trial tests giving surfactant through a thin tube into the windpipe right after birth for babies born before 28 weeks. It aims to help their lungs work better and reduce breathing problems.
Dublin, None SelectedAges Up to 30 minutes - NCT06631534RecruitingPhase 4
Testing dexmedetomidine for kids having heart defect closure
This trial tests adding a sedative medicine called dexmedetomidine to standard anesthesia for children aged 3 to 12 who are having a procedure to close a hole in their heart (atrial septal defect). The goal is to see if it helps them recover more smoothly.
Alexandria, Alexandria GovernorateAges 3–12 - NCT07710885RecruitingPhase 3
Futibatinib for advanced bile duct cancer
This trial tests a new targeted therapy, futibatinib, for people with advanced or unresectable bile duct cancer who have not had prior treatment for advanced disease. It aims to see if this drug can help shrink or control the cancer.
BrisbaneAges 18 years+ - NCT07710196RecruitingPhase 3
NPI-001 for preserving vision in usher syndrome
This study tests a 24-month treatment with NPI-001, an oral medication, to see if it can slow the loss of light-sensing cells in the retina for people with retinitis pigmentosa tied to Usher syndrome. It aims to preserve remaining vision and delay further damage.
Dallas, TexasAges 18 years+ - NCT07713758RecruitingPhase 1
How the body handles repinatrabit with liver or kidney issues
This trial checks how repinatrabit is processed in people with moderate liver problems or severe kidney problems compared to healthy adults. It helps doctors understand if the dose should be adjusted for these conditions.
Miami, FloridaAges 18–65 - NCT07709702Recruiting
Enhanced recovery after surgery for children with bile duct issues
This trial tests an enhanced recovery program (ERAS) to help children heal faster after surgery for two bile duct conditions: choledochal cyst and biliary atresia. The program includes steps to improve recovery, like better pain control and early eating.
Lahore, Punjab ProvinceAges 1 month–14 years - NCT07714161Enrolling by invitation
Mayo Clinic study for rare and undiagnosed conditions
This study brings together experts to try to find answers for people with rare or undiagnosed conditions. If standard genetic tests haven't given you a diagnosis, this research may help uncover new clues.
Rochester, MinnesotaAges Any age - NCT07708714RecruitingPhase 1/Phase 2
Hydroxyurea study for teens with sickle cell anemia
This study tests how well a higher dose of hydroxyurea works for teenagers with sickle cell anemia living in areas where malaria is common. It looks at safety and how the treatment affects their daily lives.
KampalaAges 11–18 - NCT07711730Recruiting
Telehealth support for kids with limb-girdle muscular dystrophy
This study tests an online program to help children and teens with limb-girdle muscular dystrophy improve their thinking, social skills, and emotional well-being. You join from home using a computer with a camera and internet.
Bilbao, BiscayAges 7–17 - NCT07713433Recruiting
Antibiotics versus none for pediatric colorectal surgery
This trial tests whether giving oral antibiotics before surgery is better than giving no antibiotics for children who are having their colostomy closed or a pull-through procedure. It aims to reduce infections and improve recovery in children with conditions like anorectal malformation or Hirschsprung's disease.
Lahore, Punjab ProvinceAges 1–15 - NCT06302985Recruiting
Inhaled dye for lung malformations in children
This trial tests a new way to use a dye called ICG (indocyanine green) that is inhaled like a mist. It may help doctors see and treat congenital lung malformations in children more clearly.
Wuhan, HubeiAges 3 months–14 years - NCT06632834RecruitingPhase 2
Targeted treatment study for dilated cardiomyopathy
This study tests whether a personalized treatment approach can improve heart function in people with dilated cardiomyopathy (a condition where the heart becomes enlarged and weak). It focuses on patients who still have low heart function despite standard medications.
Taipei County, Zhongzheng DistrictAges birth–99 years - NCT07704099RecruitingPhase 2
Study of KER-065 for Duchenne Muscular Dystrophy
This study tests a new medication called KER-065 to see if it helps people with Duchenne Muscular Dystrophy (DMD). It may help boys and men who are still walking, as well as those who use a wheelchair, as long as they are on a stable corticosteroid treatment.
Auckland, North IslandAges 9 years+ - NCT07698951Recruiting
Testing a new device to close PFO after stroke
This trial tests a new device to close a PFO (a small hole in the heart) in people who have had a stroke caused by a blood clot passing through that hole. The goal is to see if the device can prevent another stroke.
BelgradeAges 18–65 - NCT07700966Recruiting
Long-term safety registry for EB treatment
This study follows patients who have already received a gene therapy treatment called prademagene zamikeracel for a severe skin condition called recessive dystrophic epidermolysis bullosa (RDEB). The goal is to track long-term safety and health outcomes after treatment.
Palo Alto, CaliforniaAges Any age - NCT07702123Recruiting
Physical activity study for women with Behcet's and metabolic syndrome
This study tests if a physical activity program helps women who have both Behcet's disease and metabolic syndrome. It aims to improve their health and well-being.
Giza, DokkiAges 40–50 - NCT07702448Recruiting
Jaw surgery and its effect on mood and looks
This study looks at how jaw surgery affects your mood and how you feel about your appearance. You will answer questions one week before surgery and again 6 months after, and you need to use social media as part of the research.
Kirikkale, KırıkkaleAges 18–40
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.