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 applicable2,771
- Phase 2284
- Phase 1235
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome55
- 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
- California341
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona78
- Texas72
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- 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 - NCT06174220RecruitingPhase 2
Tideglusib for genetic arrhythmogenic cardiomyopathy
This trial tests a medication called tideglusib to see if it can reduce extra heartbeats in people with a genetic form of arrhythmogenic cardiomyopathy. It may help stabilize the heart's rhythm and slow disease progression.
Calgary, AlbertaAges 18 years+ - NCT06175104Recruiting
HeartGPS: Support for parents after a prenatal heart diagnosis
This study tests a counseling program for parents whose unborn baby has a serious heart condition called single ventricle. The program aims to help parents manage stress and support their baby's development before and after birth.
Los Angeles, CaliforniaAges 18 years+ - NCT06177353Recruiting
Immune study in recessive dystrophic epidermolysis bullosa and stem cells
This study looks at the immune system in people with recessive dystrophic epidermolysis bullosa (RDEB) and tests how placental stem cells affect immune cells in the lab. It aims to understand the disease better and explore potential treatments.
ParisAges 18–80 - NCT06177938Recruiting
Using breath test to diagnose lactose intolerance
This study tests a new way to diagnose lactose intolerance by measuring certain markers in your breath. It may help people who have trouble digesting dairy foods get a faster, easier diagnosis.
Zurich, Canton of ZurichAges 18–65 - NCT06178120Recruiting
Study of disease progression in women with X-linked adrenoleukodystrophy
This study follows women with a genetic condition called X-linked adrenoleukodystrophy (ALD) over time to see how the disease progresses. It includes annual check-ups and MRI scans of the brain and spinal cord to monitor changes.
ParisAges 18 years+ - NCT06178887Recruiting
Cardiac MRI for heart and blood vessel issues in Turner syndrome
This study uses cardiac MRI to look for heart and blood vessel differences in adults with Turner syndrome. It may help find problems early so you can get the right care.
ModenaAges 18 years+ - NCT06182436Recruiting
Virtual reality for pain during eczema injection
This trial tests whether playing a virtual reality game during a dupilumab injection (for eczema) can help reduce pain and anxiety. It's for kids and teens aged 6 to 17 who are already taking or about to start this medicine.
Montreal, QuebecAges 6–17 - NCT06184126Recruiting
Virtual reality for sickle cell pain in the emergency room
This trial tests whether using a virtual reality (VR) headset, along with your usual pain treatment, can help reduce pain during a sickle cell crisis. It is for adults with sickle cell disease who are in the emergency department and receiving IV opioids for a vaso-occlusive crisis.
Baltimore, MarylandAges 18 years+ - NCT06184503Recruiting
How velmanase alfa works in children under 3 with alpha-mannosidosis
This study looks at how the drug velmanase alfa (Lamzede) works in the body of young children with alpha-mannosidosis. It's for children who started the drug before age 3.
Miami, FloridaAges Up to 3 years - NCT06185673RecruitingPhase 1/Phase 2
Gene therapy trial for OPMD with swallowing problems
This trial tests an experimental gene therapy (BB-301) for people with OPMD who have trouble swallowing. It involves a one-time injection into the throat muscles during a short surgery, and researchers will check if it is safe and improves swallowing.
New York, New YorkAges Up to 65 years - NCT06186973Recruiting
Fetal and newborn heart assessment during labor
This study checks how well a baby's heart works during labor and right after birth. It uses a special monitor (CTG with STAN) to see if the baby's heart responds normally to the stress of labor.
OsloAges birth–1 hour - NCT06188741RecruitingPhase 2
Selumetinib to prevent tumor growth in NF1 children
This study tests whether the drug selumetinib can prevent the growth of plexiform neurofibromas (nerve tumors) in young children with NF1. Children are first monitored with MRI scans, and if a tumor appears or grows, they may receive treatment.
Birmingham, AlabamaAges 1–8 - NCT06189092Recruiting
Electrosclerotherapy for low-flow venous malformations
This study is testing a treatment called electrosclerotherapy for low-flow venous malformations. It uses a medicine (bleomycin) injected into the abnormal blood vessels to shrink them. The goal is to see how well it works and how safe it is.
Bologna, Emilia-RomagnaAges 16 years+ - NCT06190210Recruiting
Brain blood flow study in infants with heart defects
This study measures how well blood flows to the brain in infants with serious heart defects. The results may help doctors understand and protect brain development before and after heart surgery.
Leuven, Vlaams-BrabantAges Up to 6 months - NCT06190626Recruiting
A study of eye disease in Zellweger spectrum disorder over time
This study follows people with Zellweger spectrum disorder (ZSD) to understand how their eye condition develops over time. It aims to help doctors treat the disease better in the future.
Rochester, New YorkAges Any age - NCT06191640Recruiting
Study of sinus disease in young children with cystic fibrosis
This study looks at sinus problems in young children with cystic fibrosis. One group of children starts a new medicine called a highly effective modulator (like Trikafta), and the other group does not take this medicine. The goal is to see how the medicine affects sinus health over time.
Aurora, ColoradoAges 2–8 - NCT06192576Recruiting
Long-term safety of olipudase alfa in young children with ASMD
This study looks at the long-term safety and how well the body accepts olipudase alfa therapy in children under 2 years old who have a certain type of ASMD. It helps understand if the treatment works safely over time.
Chicago, IllinoisAges Up to 2 years - NCT06193928Recruiting
Long-term safety study of Livmarli in ALGS or PFIC
This study is following patients who are already taking Livmarli (maralixibat) for Alagille syndrome or PFIC to see how safe and effective it is over the long term.
Los Angeles, CaliforniaAges Any age - NCT06194539Recruiting
Fertility in Men with Spinal Muscular Atrophy
This study looks at fertility (the ability to have children) in men with spinal muscular atrophy (SMA). It will help doctors understand how SMA affects male fertility by tracking sperm health and related factors.
Cresskill, New JerseyAges 18–50 - NCT06194669Recruiting
VHL disease and kidney tumor risk study
This study looks at how early changes in kidney cells can lead to tumors in people with VHL disease. It may help understand why some people develop kidney cancer and how to prevent it.
MilanAges 18 years+ - NCT06197165Recruiting
Shiatsu for amyloidosis symptoms and quality of life
This study tests whether shiatsu (a gentle Japanese touch therapy) can improve symptoms and quality of life for people with amyloidosis. If you have a bothersome symptom like pain, shortness of breath, or anxiety, this trial may help you feel better.
CréteilAges 18 years+ - NCT06198296RecruitingPhase 1
Armored CAR T cells for GPC3-positive solid tumors
This trial tests a new type of immunotherapy called CAR T cells that are trained to find and attack cancer cells with a specific marker (GPC3). The CAR T cells are also 'armored' with extra proteins (IL-15 and IL-21) to help them work better. It might be an option for adults with advanced solid tumors, especially liver cancer, that have not responded to standard treatments.
Houston, TexasAges Up to 21 years - NCT06198712RecruitingPhase 2
Testing etavopivat in children with sickle cell disease
This trial tests a new drug, etavopivat, to see how it works in children with sickle cell disease. It may help reduce painful crises and other complications.
Toronto, OntarioAges 6 months–18 years
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 5,764 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.