Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,858 recruiting studies in this area right now.
By phase
- Not applicable2,772
- Phase 2282
- Phase 1235
- Phase 3215
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 353
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis129
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome54
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease38
- Hemophilia A38
- Cystic Fibrosis (CF)37
- Cardiac Amyloidosis35
- Huntington Disease32
- Lynch Syndrome32
- Heart Failure31
- Retinitis Pigmentosa31
- Bronchopulmonary Dysplasia30
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)27
Leading sponsors
- Assistance Publique - Hôpitaux de Paris62
- Boston Children's Hospital42
- National Institute of Allergy and Infectious Diseases (NIAID)38
- Mayo Clinic37
- Children's Hospital Medical Center, Cincinnati33
- Hospices Civils de Lyon32
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Riphah International University26
- Massachusetts General Hospital23
- National Cancer Institute (NCI)23
Where studies are running
- California340
- Maryland161
- Alabama132
- Massachusetts116
- New York84
- Arizona79
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT07244172Recruiting
Mud walking for kids with flexible flat feet
This study tests whether walking on mud can improve foot posture and balance in children with flexible flat feet. It may help strengthen the feet without surgery or special equipment.
Lahore, Punjab ProvinceAges 6–11 - NCT07244614Recruiting
Study of ustekinumab or apremilast for Behçet's disease symptoms
This study tests if ustekinumab or apremilast can help control Behçet's disease symptoms like mouth and genital ulcers, skin sores, and joint pain. It may be an option if standard treatments haven't worked well.
BordeauxAges 18 years+ - NCT07246265Recruiting
Morphine clearance study for sickle cell crisis in ICU
This study looks at how the body clears morphine in adults with sickle cell disease who are in the intensive care unit (ICU) for a pain crisis or acute chest syndrome and are receiving morphine through a patient-controlled analgesia (PCA) pump. The goal is to understand how kidney function affects morphine breakdown.
Tours, FranceAges 18 years+ - NCT07246356Recruiting
Online group therapy for birth trauma
This trial tests a structured online group therapy for people experiencing trauma symptoms related to childbirth. It aims to see if the therapy is feasible and helpful for those under a specific perinatal mental health service.
WolverhamptonAges 18–65 - NCT07247058RecruitingPhase 4
Testing Isturisa for mild cortisol excess from adrenal tumors
This study tests whether a drug called Isturisa (osilodrostat) can safely lower mild cortisol excess in people with adrenal tumors. It aims to help patients avoid the long-term effects of too much cortisol.
Baltimore, MarylandAges 18 years+ - NCT07247188RecruitingPhase 3
Pneumococcal vaccine study for children with sickle cell disease
This trial tests a single dose of a new pneumococcal vaccine (21-valent) in children and teens with sickle cell disease. It aims to see if the vaccine can help protect against serious infections caused by pneumococcus bacteria, which are especially dangerous for people with SCD.
Atlanta, GeorgiaAges 2–17 - NCT07247266RecruitingPhase 1
Testing JTE-162 for CAPS (FCAS and MWS)
This study tests an experimental drug called JTE-162 for people with certain types of CAPS (cryopyrin-associated periodic syndrome). The goal is to see if it's safe and can help control symptoms like fever, rash, and joint pain.
North York, OntarioAges 18–80 - NCT07248267Recruiting
Vojta therapy for children with Down syndrome
This study is testing a therapy called Vojta therapy to see if it helps children with Down syndrome move better and walk more easily. It compares the therapy to other supportive care.
Lahore, Punjab ProvinceAges 2–10 - NCT07248449Recruiting
Exercise study for children with Down syndrome ages 3-8
This trial tests two types of exercise—Cuevas Medak and rebound exercises—to see which one improves physical fitness in children with Down syndrome. It may help your child get stronger and move better.
Lahore, Punjab ProvinceAges 3–8 - NCT07250854RecruitingEarly Phase 1
Using a dye to help diagnose blocked bile ducts in newborns
This study tests if a special dye (ICG) and a camera can help doctors see blocked bile ducts in newborns with jaundice, so they can diagnose biliary atresia faster and without extra procedures.
Birmingham, AlabamaAges 2 weeks–4 months - NCT07250945Recruiting
Laser treatment for birthmarks that are at least 2.5 cm across
This trial tests a laser treatment for vascular birthmarks (like port-wine stains). It might help if your birthmark is on an area of your body that is not the center of your face and you have light to medium skin.
Szeged, Csongrád-Csanád VármegyeAges 7–65 - NCT07250997Recruiting
PALLAS laser study for skin conditions
This trial tests a new laser (PALLAS) that delivers targeted UVB light to treat skin conditions like psoriasis or eczema. It may help if your doctor thinks your skin problem could improve with UVB therapy.
Szeged, Csongrád-Csanád VármegyeAges 18–65 - NCT05022459Recruiting
Prevent bleeding in kids with Hemophilia A who play sports
This study compares two ways to prevent bleeding in people with Hemophilia A while they play sports. It looks at whether factor VIII or emicizumab prophylaxis helps reduce bleeding during higher-risk sports activities.
Little Rock, ArkansasAges 6–19 - NCT06504160RecruitingPhase 1
Testing a bacteria cream for eczema (TIME-2)
This study tests whether a cream made from a friendly skin bacteria (Staphylococcus hominis) can help treat eczema. Eligible participants will apply the cream to eczema patches on their arms, legs, or trunk for several weeks.
San Diego, CaliforniaAges 6 years+ - NCT07127978Recruiting
Study of Givinostat in people with Duchenne muscular dystrophy
This study is for people with Duchenne muscular dystrophy (DMD) who are taking Givinostat, a daily oral medicine. Researchers will look at real-world data to see how the medicine works in everyday life, including lab results and how it's being used.
Little Rock, ArkansasAges 6 years+ - NCT04819841RecruitingPhase 1/Phase 2
Gene correction using your own stem cells to improve sickle disease
This trial tests a gene-correction treatment that changes sickle hemoglobin (HbS) into normal hemoglobin (HbA) using your own stem cells. It may help reduce severe sickle cell complications for people with severe disease who meet specific safety and medical criteria.
Los Angeles, CaliforniaAges 12–50 - NCT07223255Recruiting
Mediterranean diet study for children with cystic fibrosis
This study tests whether a Mediterranean-style diet can improve gastrointestinal health in children with cystic fibrosis. It requires families to follow the diet for 6 months and attend regular clinic visits.
Lebanon, New HampshireAges 3–18 - NCT06057714Recruiting
MRI study of airway function in cystic fibrosis
This study uses a special MRI technique (19F MRI) to look at how well your lungs are working and where any blockages are. The goal is to understand airway problems in cystic fibrosis better.
Chapel Hill, North CarolinaAges 18–99 - NCT07585175Recruiting
Moving from pediatric to adult heart disease care
This study helps doctors understand how to smoothly transition young people with heart problems they were born with from pediatric (children's) hospitals to adult care. It may help improve care coordination and outcomes as patients grow up.
Ottawa, OntarioAges 18 years+ - NCT07312734Recruiting
Testing new ways to find lung germs in CF
This study tests a new method to detect bacteria in the lungs of people with cystic fibrosis (CF) by analyzing mucus samples. It aims to improve how we diagnose and treat lung infections.
La Jolla, CaliforniaAges 16 years+ - NCT07516288RecruitingPhase 1
Second CAR-DC Cell Infusion for Advanced Heart Failure
This trial tests a second dose of specialized immune cells (called CAR-DC) in patients with severe dilated cardiomyopathy (a weakened heart muscle) who received one dose before but saw their heart function decline back to baseline. The goal is to see if a second dose is safe and can improve heart function when the first treatment's benefits wore off.
Hangzhou, Zhejiang / 浙江Ages 18–75 - NCT07649031Recruiting
MRI Scanning to Monitor Mouth Cancer Risk in Fanconi Anemia
This study uses MRI (a safe imaging scan) to detect and track dangerous changes in the mouth of people with Fanconi Anemia, a rare genetic condition. The goal is to find a better way to catch oral cancer early.
Minneapolis, MinnesotaAges 18 years+ - NCT07558733RecruitingPhase 1/Phase 2
New drug combinations for advanced hormone-receptor positive breast cancer
This trial tests two new drugs (sapanisertib and serabelisib) alone or together to treat advanced breast cancer that is hormone-receptor positive. These drugs target specific pathways that help cancer grow, and the trial will see if they work better than standard treatments.
Los Angeles, CaliforniaAges 18 years+ - NCT07252050RecruitingPhase 1/Phase 2
Ruxolitinib and half-matched transplant for severe sickle cell disease
This trial tests a new approach using the drug ruxolitinib to help a half-matched bone marrow transplant work better for people with severe sickle cell disease. It aims to reduce complications and improve outcomes.
Aurora, ColoradoAges 12–45
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,858 recruiting medical genetics studies from the U.S. registry right now, out of 5,794 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.