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 Disease84
- 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
- California325
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York85
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT07484607Recruiting
Study on infections and treatments in cystic fibrosis exacerbations
This study looks at how lung infections and inflammation affect people with cystic fibrosis during severe flare-ups. It aims to understand which antibiotics work best and why, so your care team can choose the right treatment for you.
CardiffAges 16 years+ - NCT07483424Enrolling by invitation
Growth restriction in full-term babies: body composition study
This study looks at how body composition differs in full-term babies who had intrauterine growth restriction. It may help doctors better understand and care for these infants.
LisbonAges 9 months–9 months - NCT07483255Recruiting
Fetal embolization trial for vein of Galen malformation
This trial tests a fetal procedure to treat a rare brain blood vessel condition called vein of Galen malformation. The goal is to improve the baby's outcome by closing off abnormal connections inside the brain before birth.
Boston, MassachusettsAges 18 years+ - NCT07484945Recruiting
Multiomics study in adults with phenylketonuria
This study looks at adults with phenylketonuria (PKU) using advanced testing (called multiomics) to understand how the condition affects the body. If you took part in a previous PKU study (ECOPHEN) and are willing to give a saliva sample for DNA analysis, this trial may be for you.
AngersAges 18 years+ - NCT07485543RecruitingEarly Phase 1
Inhaled genetic therapy for cystic fibrosis patients
This trial tests an inhaled medicine that delivers a corrective genetic message (mRNA) to lung cells in people with cystic fibrosis. It aims to see if the treatment is safe and can improve lung function over time.
Beijing, Beijing MunicipalityAges 12 years+ - NCT06775561Recruiting
Study of hidden DNA causes for rare neuromuscular and epilepsy diseases
This study is for people with a neuromuscular disease or epilepsy whose genetic testing hasn't found a clear answer. It uses advanced techniques to look at parts of the DNA that don't code for proteins, to find hidden causes and help guide future treatments.
BolognaAges Any age - NCT06775639Recruiting
Can we skip a heart artery test before surgery?
This study looks at whether people with adult congenital heart disease can skip a pre-surgery artery test (coronary angiography) if they already had a CT scan. If you qualify, you might help doctors find safer, simpler ways to prepare for heart surgery.
BolognaAges 18 years+ - NCT06776380Recruiting
Puberty development in people with RASopathies
This study looks at how puberty develops in people with RASopathies. It aims to understand the process better by studying those who have already completed puberty.
Bologna, BolognaAges 8–35 - NCT06776133Recruiting
Predicting heart pressure from vein size in children
This study looks at a possible link between the size of a large vein (the inferior vena cava) and the pressure in the right side of the heart in children. If the link is strong, doctors might one day use vein size to estimate heart pressure without more invasive tests.
Bologna, BolognaAges Up to 18 years - NCT06777394Recruiting
High intensity interval training for Down syndrome
This study tests whether high intensity interval training (short bursts of intense exercise) can help children with Down syndrome. Doctors want to see if it improves strength and overall health.
Lahore, Punjab ProvinceAges 3–14 - NCT01756222Recruiting
Bicuspid aortic valve registry for adults
This study is a registry that collects health information from adults with bicuspid aortic valve disease. It may help doctors better understand outcomes and guide future care for patients and families affected by this condition.
Ann Arbor, MichiganAges 18 years+ - NCT05373498Recruiting
Registry for children’s heart emergencies after resuscitation
This trial is a registry that collects information about children (under 18) who have congenital heart problems and need resuscitation with chest compressions. It helps doctors understand these emergencies and may improve future care for similar patients.
Leipzig, SaxonyAges Up to 18 years - NCT07474935Recruiting
Real-world stroke treatment device registry
This trial collects information about the use of a specific stroke treatment device in real-world medical practice. It helps researchers see how well this device works for a wide range of patients, not just those in strict study conditions.
BrestAges Any age - NCT07476183RecruitingPhase 1
Testing APR-2020 for RPS19-deficient Diamond-Blackfan Anemia
This trial is testing a new treatment called APR-2020 for children and young adults with a specific type of Diamond-Blackfan Anemia (caused by a change in the RPS19 gene). The goal is to see if this treatment is safe, tolerable, and can help reduce the need for blood transfusions.
Stanford, CaliforniaAges 2–25 - NCT07477691RecruitingPhase 4
Immune therapy during Palynziq treatment for PKU
This study tests whether combining a drug called methotrexate with Palynziq (a treatment for PKU) can help control immune responses and improve treatment outcomes. It may be an option for adults with PKU who have never used Palynziq or who have not reached target blood levels after at least 24 weeks.
Los Angeles, CaliforniaAges 18–65 - NCT07473843RecruitingPhase 3
Study of zodasiran for teens with homozygous familial hypercholesterolemia
This study tests an experimental medicine called zodasiran in teenagers who have a rare inherited condition that causes very high cholesterol. The goal is to see if it can lower cholesterol levels and reduce the risk of heart problems.
St Louis, MissouriAges 12–17 - NCT06776471Recruiting
Sealant study for children with weak enamel
This study compares two types of dental sealants applied to the first permanent molars of children aged 6-12 who have a condition called molar incisor hypomineralization (MIH), which means their back teeth have soft or chalky spots. The goal is to see which sealant works better at protecting these teeth from cavities.
AnkaraAges 6–12 - NCT06781437Recruiting
Lymphoma medical record review study
This study looks back at the medical records of people with lymphoma to learn more about the disease. It does not involve any new treatments or tests—just reviewing what happened in the past.
Bologna, BolognaAges 18 years+ - NCT06780514Recruiting
Exercise testing for girls with Turner syndrome
This study will have girls with Turner syndrome do a cardiopulmonary exercise test (a special bike or treadmill test) to check how their heart and lungs work during exercise. It may help doctors better understand fitness and health in Turner syndrome.
GhentAges 8–18 - NCT07478224Recruiting
Blood flow restriction training for osteogenesis imperfecta type I
This study tests whether a special type of strength training, called blood flow restriction training, can improve muscle, bone, and quality of life in adults with osteogenesis imperfecta type I.
GhentAges 18–65 - NCT07477873Enrolling by invitation
Emotions and jaw surgery recovery study
This study looks at how patients feel emotionally and physically before and after jaw surgery. It uses special software and questionnaires to track emotions, and checks the jaw joint and muscles after surgery. The goal is to see how these relate to recovery in patients having planned jaw surgery.
Kaunas, KaunoAges 18 years+ - NCT07477197Recruiting
Heart Institute Biobank for Adult Congenital Heart Disease
This study collects blood and tissue samples and health information from adults with or at risk for congenital heart disease or related heart conditions. It aims to help researchers better understand these conditions and improve future treatments.
Cincinnati, OhioAges 16 years+ - NCT07473739Recruiting
AI ultrasound for predicting pregnancy outcomes in early FGR
This study uses artificial intelligence (AI) to analyze ultrasound images and predict pregnancy outcomes for babies with early-onset fetal growth restriction (FGR) caused by placenta problems. If you are carrying a single baby with this condition and your doctor is continuing to monitor the pregnancy without immediate delivery, this trial may apply to you.
ShanghaiAges 20–43 - NCT07479147Recruiting
Stroke prevention in elderly by PFO closure vs blood thinners
This trial compares two treatments for preventing another stroke in older adults who have a PFO (a small hole in the heart). It tests whether closing the hole with a device works better than using blood-thinning medication.
TaichungAges 18–90
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 5,456 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.