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
- NCT07366086RecruitingPhase 3
Long-term safety of romosozumab for osteogenesis imperfecta in children
This study follows children who previously received romosozumab in an earlier trial to check their long-term safety. It helps understand if the treatment's benefits last safely over time.
Indianapolis, IndianaAges 5–19 - NCT07369505RecruitingPhase 1
A study of Sapu003 for advanced tumors sensitive to mTOR
This trial tests a new drug, Sapu003, in people with certain advanced cancers that may respond to mTOR-targeted therapy. It is looking at two groups: postmenopausal women with a type of breast cancer (HR+/HER2-), and adults with kidney, liver, neuroendocrine, or TSC-related tumors. The goal is to see if Sapu003 is safe and effective.
AdelaideAges 18 years+ - NCT07367971RecruitingPhase 1
ABP-671 interaction study for gout patients
This study tests how a new drug, ABP-671, interacts with allopurinol (a common gout medicine) in people with gout. It is looking for participants with normal or moderately reduced kidney function.
Cary, North CarolinaAges 18–75 - NCT03900780Recruiting
Genetic testing of embryos for repeat IVF failure
This study tests embryos before pregnancy using genetic screening to look for extra or missing chromosomes, in people who have had repeated IVF embryo transfers without success. It may help reduce the chance of transferring embryos that are unlikely to result in a healthy pregnancy.
LeuvenAges 18–40 - NCT06844734Recruiting
Study of ITB treatment for hereditary spastic paraplegia
This trial tests if a treatment called ITB (intrathecal baclofen) can help reduce muscle stiffness in people with hereditary spastic paraplegia (HSP). Participants will receive the treatment and be followed over time to see how well it works.
Shanghai, Shanghai MunicipalityAges 14–70 - NCT06224660RecruitingPhase 1
Gene therapy for heart problems in Duchenne muscular dystrophy
This trial tests an experimental therapy for heart weakness caused by Duchenne muscular dystrophy. It aims to improve how heart cells handle calcium to protect heart function.
Kansas City, KansasAges 18 years+ - NCT07354724RecruitingPhase 1
Study of DNL952 for adults with late-onset Pompe disease
This trial tests a new drug, DNL952, in adults with late-onset Pompe disease. It aims to see if the drug is safe and how it works in the body, with different groups for people currently on enzyme replacement therapy or those who haven't had it in the past year.
Irvine, CaliforniaAges 18–75 - NCT07358156RecruitingPhase 1
Comparing a new dupilumab medicine to Dupixent in healthy adults
This trial tests a new version of Dupixent® (called CKD-706) to see how it compares to the approved medicine in healthy adults. It's a first step to make sure the new medicine works similarly and is safe.
Berlin, State of BerlinAges 18–55 - NCT07355075RecruitingPhase 2/Phase 3
Study of a new cream for moderate to severe eczema
This study tests an investigational cream for moderate to severe eczema. It helps see if the cream reduces symptoms like itching and skin inflammation.
Irving, TexasAges 18–80 - NCT07352813Recruiting
Comparing numbing methods for kids with MIH teeth
This study tests two ways to numb teeth—in the bone or in the gum—to see which works better for children with weak enamel on their back teeth and front teeth. It may help find a pain-free way to treat these teeth.
AnkaraAges 6–12 - NCT07359937Enrolling by invitation
Reducing fluoride risk in school children
This trial tests a program to lower children's exposure to fluoride in drinking water. It includes education and urine tests to measure fluoride levels. Families with kids in kindergarten who get meals at school may benefit.
PalermoAges 2–5 - NCT07361536Recruiting
Heart health in MPS
This study looks at heart structure and function in people with mucopolysaccharidosis (MPS), a rare genetic disorder. It aims to understand how MPS affects the heart over time.
Orange, CaliforniaAges birth–99 years - NCT07356050Recruiting
Measles vaccine study for children with sickle cell disease
This study checks if the measles vaccine works well in children with sickle cell disease. It aims to help protect children with this condition from measles.
Cincinnati, OhioAges 6 months–6 years - NCT07354646Recruiting
Understanding heart disease with T1 mapping MRI
This study uses a special type of MRI (T1 mapping) to better understand different heart conditions. It may help doctors improve diagnosis and treatment by looking at detailed images of your heart muscle.
Beijing, Beijing MunicipalityAges Any age - NCT07359690Recruiting
Study of Heart Muscle Biopsies
This study collects samples of heart muscle tissue (biopsy) to better understand heart conditions. It is for people who already need this biopsy for their healthcare.
Essen, North Rhine-WestphaliaAges 18 years+ - NCT07356778RecruitingPhase 4
Study of sotatercept for Eisenmenger syndrome patients
This trial tests a new drug called sotatercept for people with Eisenmenger syndrome or a related heart defect that hasn't been repaired. It may help if current treatments aren't working well enough.
Fukuoka, Not Required For This CountryAges 18 years+ - NCT07357558Recruiting
Reproductive health experiences in adults with PCD
This study is a conversation-based research project to understand how reproductive issues affect adults with Primary Ciliary Dyskinesia (PCD), a rare lung condition. By sharing your experiences, you can help doctors and researchers better support patients like you.
SouthamptonAges 18 years+ - NCT07358000Recruiting
Comparing two moisturizers for mild to moderate eczema
This trial tests if two different moisturizing balms work equally well for treating mild to moderate eczema (atopic dermatitis) in children and adults. It may help find a more affordable or better-tolerated option.
Caen, NormandyAges Any age - NCT07358013Recruiting
Endothelial cells in von Willebrand disease and related conditions
This study looks at special cells from blood vessels (endothelial colony-forming cells) in people with von Willebrand disease (a bleeding disorder) or a related acquired condition. The goal is to better understand these conditions using blood donations.
MilanAges 16 years+ - NCT06850311RecruitingPhase 2/Phase 3
Herbal ointment for mild to severe eczema
This trial tests whether a special herbal ointment can help treat atopic dermatitis (eczema). It is for adults with mild, moderate, or severe eczema covering less than 10% of their body surface. The ointment is applied directly to the skin, and participants will also use a gentle, fragrance-free moisturizer.
New Taipei CityAges 18–65 - NCT00005095Recruiting
Ovarian cancer risk study: specimens and health data
This study collects tissue samples and medical information from people at higher risk for ovarian cancer or undergoing gynecologic surgery. Researchers use this data to develop better ways to detect and prevent ovarian cancer early.
Chicago, IllinoisAges 18–80 - NCT05509153RecruitingPhase 2
N-acetylcysteine trial for people at risk of Huntington’s disease
This Phase 2 study tests whether NAC (a supplement-like medicine) can delay symptoms in people who carry the Huntington’s disease gene expansion but do not yet have clear movement symptoms. You may be asked to take NAC and attend regular visits, scans, and blood tests to see if it changes how likely you are to develop Huntington’s disease within the next 10 years.
Westmead, New South WalesAges 18 years+ - NCT06850194Recruiting
Comparing two jaw surgeries for severe underbite
This trial compares two surgical techniques to fix a severe underbite caused by a small lower jaw. One method uses a special cut in the jawbone and a piece of hip bone to lengthen it, while the other uses a different jawbone cut to move the jaw forward. It will help find which approach works better for people with this condition.
Beni-seuf, EgyptAges 18 years+ - NCT06851338Recruiting
Inspire therapy study for teens with Down syndrome and sleep apnea
This study tests a device called Inspire that stimulates the airway during sleep to treat obstructive sleep apnea in teenagers with Down syndrome who cannot use a CPAP machine or had a tonsillectomy that did not work well.
Phoenix, ArizonaAges 13–18
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.