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
- NCT06094205Recruiting
BrainGate2 speech study for people with tetraplegia
This study tests a brain-computer interface to help people with severe paralysis communicate by decoding attempted speech or movements. It may offer a new way to interact with devices using only brain signals.
Sacramento, CaliforniaAges 18–80 - NCT06096116RecruitingPhase 3
Trial of a blood product for heparin resistance during heart surgery
This study tests a blood-derived medicine (antithrombin) to help people who do not respond well to the blood thinner heparin during heart surgery. It aims to make surgery safer by improving blood thinning.
Stanford, CaliforniaAges 18–85 - NCT06096805Recruiting
Predicting fetal growth restriction in high-risk pregnancies
This study aims to better predict fetal growth restriction (when a baby doesn't grow as expected) in women who have certain risk factors. By joining, you may help doctors develop better screening tools for future pregnancies.
Shanghai, Shanghai MunicipalityAges 18–45 - NCT06096857RecruitingPhase 2
Cardamom and topical bacteria cream for eczema
This study tests whether taking cardamom by mouth and applying a cream made from a harmless bacteria (Roseomonas) can help treat eczema (atopic dermatitis) in people age 2 and older.
Bethesda, MarylandAges 2–100 - NCT06098833RecruitingPhase 2
Sildenafil for newborn brain injury after birth asphyxia
This trial tests whether a liquid form of the medicine sildenafil can help repair brain damage in newborns who had a difficult birth and were treated with cooling. If your baby meets the cooling criteria and has brain injury on an MRI, this study may offer an additional treatment option.
Montreal, QuebecAges birth–2 days - NCT06099015Recruiting
Cera vascular plug study for blocking blood vessels
This study tests a device called the Cera Vascular Plug System to block blood flow in certain arteries or veins. It may help patients who need this type of treatment for conditions like abnormal blood vessels or bleeding.
Bad HersfeldAges 18–85 - NCT06100614Recruiting
Using a blood test to reduce antibiotics in preterm newborns
This study checks if a blood test (presepsin) can help doctors safely stop antibiotics earlier in preterm babies at moderate risk of infection. The goal is to reduce unnecessary antibiotic use.
Amsterdam, North HollandAges Any age - NCT06105099Recruiting
Speech therapy for children with cleft palate
This study tests a special speech therapy program for children with a cleft palate who have speech errors. The therapy focuses on improving speech sounds and seeing if the improvements last over time.
GhentAges 4–12 - NCT06107400RecruitingEarly Phase 1
RM-004 cell therapy for Hemoglobin H-Constant Spring disease
This early-phase trial tests a new cell therapy called RM-004 for people with Hemoglobin H-Constant Spring disease, a form of alpha thalassemia that requires regular blood transfusions. The goal is to see if the treatment is safe and can reduce or eliminate the need for transfusions.
Nanning, GuangxiAges 12–35 - NCT06111638RecruitingPhase 2/Phase 3
Gene therapy for severe hemophilia A
This trial tests a gene therapy to help your body produce more of the clotting protein (factor VIII) it's missing. It may reduce or stop the need for regular factor infusions.
Beijing, Beijing MunicipalityAges 18 years+ - NCT06112340RecruitingPhase 2/Phase 3
Extension study of linsitinib for thyroid eye disease
This trial tests two doses of an oral medication (linsitinib) for people with active, moderate-to-severe thyroid eye disease who were in a previous study and didn't improve enough or had their bulging come back. It aims to see if continuing treatment can help with eye bulging and other symptoms.
Miami, FloridaAges 18 years+ - NCT06111950Recruiting
Study of RNU4ATAC and RTTN gene conditions
This study looks at rare genetic syndromes caused by changes in the RNU4ATAC or RTTN genes. It aims to understand how these changes affect the body by studying patients, their parents, and fetal tissue.
BordeauxAges Any age - NCT06114329RecruitingPhase 2
Oral AL01211 for untreated men with Fabry disease
This study tests an oral medication called AL01211 for men who have classic Fabry disease but have never been treated before. The goal is to see if it is safe and if it can help reduce the build-up of fatty substances in the body.
Guangzhou, GuangdongAges 18–60 - NCT06115122Recruiting
PEPPI Study: Finding early signs of placental problems in pregnancy
This study looks at blood markers to find women at risk for placental problems during pregnancy. It involves blood tests in early and late pregnancy, and at birth, to see how iron levels and other factors affect the placenta and baby's health.
OuluAges Any age - NCT06116526RecruitingPhase 4
Reducing Dupixent in kids with well-controlled eczema
This study looks at whether children with well-controlled eczema can safely reduce how often they take Dupixent. It aims to find the lowest effective dose to keep eczema under control.
Baltimore, MarylandAges 1–17 - NCT06120270Recruiting
CeraFlex PFO closure study for migraine or stroke
This study tests a device called CeraFlex PFO Occluder to close a small hole in the heart (PFO) that may be causing migraines or strokes. If you have a PFO with related symptoms, the device might help prevent future events.
BremenAges 18–85 - NCT06120582RecruitingPhase 1/Phase 2
Gene therapy safety study for severe hemophilia B
This study tests a new gene therapy called ANB-002 for men with severe hemophilia B (factor IX levels at or below 2%). The goal is to see if it's safe and how well it might help your blood clot normally.
HomyelAges 18 years+ - NCT06121011Recruiting
Pompe disease patient registry study
This is a registry study that collects information over time from people with Pompe disease. It helps doctors and researchers better understand the condition and how it affects daily life, without testing any new treatments.
Little Rock, ArkansasAges Any age - NCT06122805Recruiting
Electric vs. Manual Toothbrush for Kids with Cleft Lip/Palate
This study compares a special dual-light electric toothbrush to a regular manual toothbrush for kids and teens with cleft lip or palate who wear braces. The goal is to see which brush keeps teeth cleaner and gums healthier during orthodontic treatment and after oral surgery.
Los Angeles, CaliforniaAges 6–20 - NCT06124196Recruiting
Wearable device study for Duchenne muscular dystrophy
This study uses a wearable device to track your blood sugar levels and heart rate patterns over time. It aims to understand how these are related to Duchenne muscular dystrophy (DMD) and may help improve care.
Nashville, TennesseeAges 10 years+ - NCT06125847RecruitingEarly Phase 1
Gene therapy for inherited high cholesterol
This trial tests a one-time gene therapy (NGGT006) to treat a severe form of inherited high cholesterol that doesn't respond well to standard medications. It aims to lower cholesterol and reduce the risk of heart disease.
Xi'an, ShaanxiAges 12–55 - NCT06128629RecruitingPhase 3
A study of NTLA-2001 for transthyretin amyloidosis with heart problems
This study tests a new treatment, NTLA-2001, for people with a type of heart disease called transthyretin amyloidosis with cardiomyopathy. It aims to see if the medicine can help improve heart function and quality of life.
Tucson, ArizonaAges 18–90 - NCT06129656Recruiting
Cardiac Amyloidosis Registry at Leipzig Hospital
This study is a registry that collects information from people with cardiac amyloidosis. It helps doctors learn more about the condition and how to better treat it.
Leipzig, SaxonyAges Any age - NCT06136403RecruitingPhase 2
Deucravacitinib for genetic skin disorders
This study tests a medication called deucravacitinib for adults with rare genetic skin conditions that cause blisters or severe scaling. The goal is to see if it can reduce symptoms safely.
Nice, CHU de NICEAges 18–99
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.