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
- NCT06281093Recruiting
Radio waves and preemies' brain development
This trial checks if radiofrequency fields (like from phones or WiFi) affect the nervous system of preterm babies born between 26 and 34 weeks of pregnancy. It helps us understand if we need to protect these babies from such fields.
Amiens, PicardieAges 1 day–1 month - NCT04567511RecruitingPhase 4
Hemlibra for mild hemophilia A patients
This trial studies Hemlibra (emicizumab) in people with mild congenital hemophilia A to see how well it works and what safety issues may occur. It may help reduce bleeding in patients who meet the study’s specific health and safety requirements.
Indianapolis, IndianaAges 5–45 - NCT05285839RecruitingPhase 4
Dupixent plus UVB light for moderate to severe eczema
This study tests whether taking Dupixent and receiving narrowband UVB light can improve moderate to severe atopic dermatitis (eczema). It may help people whose symptoms are significant despite usual care.
East Windsor, New JerseyAges 18 years+ - NCT05038462Recruiting
Fetal care study for babies with slow growth
This study looks at therapies meant to support brain development in babies whose growth is slower than expected before birth. It enrolls pregnancies in a specific time window and uses ultrasound/Doppler testing to make sure the baby is not likely needing delivery immediately.
BarcelonaAges 18 years+ - NCT03154697Recruiting
Study for parents of children with Smith-Magenis syndrome and sleep issues
This study builds a database about people with Smith-Magenis syndrome and their sleep problems. It wants information from the child’s parent or legal guardian to better understand sleep disturbances.
Washington D.C., District of ColumbiaAges 3–65 - NCT06314529Recruiting
Long-term follow-up of BRL-101 for thalassemia
This study follows people who have already received BRL-101 treatment for transfusion-dependent beta-thalassemia to check their health over time. It helps doctors understand how well the treatment works in the long run.
Nanning, GuangxiAges 5–35 - NCT05395195RecruitingPhase 3
Erythropoietin for newborn brain injury in low-resource settings
This Phase 3 trial studies whether erythropoietin (a medicine related to red-blood-cell growth) can improve outcomes for newborns with moderate to severe brain injury after birth. The study focuses on babies born in low- and middle-income countries and aims to reduce harm from this early brain condition.
DhakaAges 1 hour–6 hours - NCT05799118Recruiting
Genetic modifiers study for inherited blood disorders
This study looks at how your genes can affect inherited blood conditions like sickle cell disease and thalassemia. It may help researchers understand why symptoms differ from person to person, which could guide better future treatments.
Boston, MassachusettsAges 2 years+ - NCT06325618Recruiting
Studying lymphedema and blood vessels in Turner syndrome
This study looks at swelling (lymphedema), inflammation, and blood vessels in women with Turner syndrome. It uses MRI scans to understand these issues better.
Aarhus NAges 18–100 - NCT05946278Recruiting
Study genes that may affect bone health after major spine surgery
This study looks at genetic (inherited) factors that may influence bone healing in people undergoing major spine procedures. It may help researchers understand why some people heal differently and could guide better care in the future.
Aurora, ColoradoAges 18–85 - NCT05761314Recruiting
Study treatments for cancers in people with RASopathy
This study looks at people who have a RASopathy—a genetic condition—and also have solid tumors (tumor growths not in blood). To join, your RASopathy must be confirmed by genetic testing, not only by symptoms.
RomaAges Any age - NCT03564561Recruiting
Pompe disease study of walking and symptoms over time
This study follows people with Pompe disease to better understand how the disease progresses and how walking function changes. You may help researchers learn what symptoms and test results matter most, without necessarily receiving a new treatment.
Garches, Hauts-de-SeineAges 18–80 - NCT06353620Recruiting
Brain connections study for certain epilepsies and genetic syndromes
This study uses MRI and a special EEG to understand brain connections in people with specific types of epilepsy or genetic conditions like Angelman syndrome and Dup15q. It may help find better ways to treat these conditions.
Conegliano, TrevisoAges 6–75 - NCT06018792Recruiting
New blood test for faster sepsis diagnosis in children
This trial tests a new, faster way to find bacteria in the blood of children with suspected sepsis. It uses a molecular test that may give results sooner than standard cultures, helping doctors treat infections more quickly.
AmsterdamAges birth–18 years - NCT06356727Recruiting
Blood vessel health test for heart recovery
This study checks if tiny blood vessel problems in the heart affect how well the heart muscle recovers after being weak. It uses special imaging during a routine heart catheterization to look at these small vessels.
FerraraAges 18–85 - NCT06359795Recruiting
PET Scan for Thyroid Eye Disease Activity
This study uses a special PET/CT scan (with a tracer called 18F-AlF-FAPI) to see how active your thyroid eye disease is. It may help doctors better understand and manage the condition.
Beijing, Beijing MunicipalityAges 18–80 - NCT05034172Recruiting
Study of markers in inherited movement disorders
This study looks for biological “markers” (measurable signs in the body) in people with inherited movement disorders, and in some related family members or healthy volunteers. It may help researchers better understand these conditions, especially for future diagnosis and care.
ParisAges 7 years+ - NCT05718440Recruiting
Checking risk factors for urinary and related issues
This study looks at why some adults with spinal dysraphism develop problems with bladder, bowel, and/or sexual function. It gathers information during a one-day visit to help doctors better understand risk factors.
ParisAges 18 years+ - NCT05678543Recruiting
Diabetes family registry for mothers, partners, and children
This study builds a registry to learn about diabetes in families over time. It includes mothers with diabetes, their partners, and their children, to help researchers understand how diabetes affects families.
AalborgAges 18 years+ - NCT06360913Recruiting
Blood and urine test for rare metabolic diseases
This study uses a simple blood spot and urine test to look for chemical signs of rare metabolic diseases. It aims to improve early detection and understanding of these conditions across all ages, including healthy individuals.
BrusselsAges 1 day–99 years - NCT05952583Recruiting
Study of growth-restricted twin babies in shared placenta
This study looks at babies in monochorionic (shared-placenta) twin pregnancies where one or both babies are growing too slowly. It aims to better understand this condition and follow families over time to improve care, with check-ins through age 2 after birth.
Boston, MassachusettsAges 18–50 - NCT06169046RecruitingPhase 2
Clenbuterol for spinal and bulbar muscular atrophy
This study tests if clenbuterol (a medication that may help strengthen muscles) can improve symptoms in people with spinal and bulbar muscular atrophy (SBMA, also called Kennedy's disease). It compares clenbuterol to a placebo (a dummy pill) over several months.
Padova, PDAges 18–75 - NCT06352216Recruiting
Joint inflammation in hemophilia A patients
This study looks at how many adults with moderate or severe hemophilia A have inflammation in their joints (synovitis). It aims to better understand joint health in people with hemophilia A, including those with inhibitors or joint replacements.
Bonn, North Rhine-WestphaliaAges 18 years+ - NCT05288894Recruiting
Italian registry for people with repaired Tetralogy of Fallot
This trial is a registry that collects information about people who have had Tetralogy of Fallot (TOF) repaired. It may help doctors better understand long-term outcomes and improve future care.
Ancona, The MarchesAges 10 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.