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
- NCT07659756Recruiting
Global study on atopic dermatitis (eczema)
This study gathers information from people around the world who have atopic dermatitis (eczema). It aims to better understand the condition and how it affects people's lives.
ZurichAges 14 years+ - NCT07655102Recruiting
Brugada Syndrome and Anesthesia: A Registry Study
This study is collecting information from people with Brugada syndrome who are having anesthesia or sedation for any procedure. The goal is to learn more about how these medications affect people with Brugada syndrome, so doctors can provide safer care in the future.
Barcelona, BarcelonaAges Any age - NCT07661784Recruiting
Saliva tests to track gum disease
This study looks at whether substances in your saliva can show if gum disease is getting worse. It might help find a simple way to monitor gum health without needles or X-rays.
IspartaAges 18–65 - NCT06677866Recruiting
Group therapy for teens with Fragile X or autism
This trial tests if group cognitive behavioral therapy (CBT) can help teens with Fragile X syndrome or autism spectrum disorder improve social and emotional skills. It looks at how teens with these conditions respond to a structured group program.
RomeAges 13–19 - NCT06603831Recruiting
3D digital surgery study for bone and joint procedures
This study tests whether 3D digital planning tools make surgeries for certain bone and joint problems more effective and cost-efficient. If you need a specific type of surgery on your forearm, pelvis, or spine, this trial could help determine if digital surgery improves your recovery.
Sabadell, BarcelonaAges 18–80 - NCT07654114RecruitingPhase 2
Testing CTH120 for men with Fragile X syndrome
This trial tests a new medicine called CTH120 to see if it is safe and helpful for adult men with Fragile X syndrome. You may be able to join if you meet certain health and age requirements.
Barcelona, BarcelonaAges 18–45 - NCT07654855Recruiting
Long-term safety of eplontersen in ATTR patients
This study follows people who already took part in an earlier trial (D8450R00003) to see how safe eplontersen is over the long term. It also includes people who had a liver transplant or severe liver problems.
Los Angeles, CaliforniaAges 18 years+ - NCT07653139Enrolling by invitation
Survey on treatment preferences for hemophilia A in Germany
This study asks adults with hemophilia A in Germany about their treatment preferences. It aims to understand what matters most to patients, which can help improve care.
Mainz, GonsenheimAges 18 years+ - NCT07646600Recruiting
Natural History Study of BAG3 Heart Disease in Chinese Adults
This study follows adults with a specific genetic mutation (BAG3) that causes heart muscle weakness. Researchers will track how the disease develops over time to better understand it and help improve future treatments.
BeijingAges 18 years+ - NCT07646704Recruiting
Study of HeartR PDA Heart Device Safety and Function
This study follows patients who had a small heart device (HeartR PDA Occluder) placed to close an opening in their heart called a PDA. Researchers will check how well the device is working and track the patient's health over time.
Pekanbaru, RiauAges 6 months+ - NCT07654829RecruitingPhase 4
Sebetralstat to prevent swelling before procedures in HAE
This trial tests if sebetralstat can prevent swelling attacks before medical procedures in people with HAE types I or II. You take the medicine short-term before your procedure to see if it helps protect you from an attack.
Colorado Springs, ColoradoAges 12 years+ - NCT07652879RecruitingPhase 2
KCD drug combination for newly diagnosed POEMS syndrome
This trial tests a combination of three drugs (carfilzomib, cyclophosphamide, and dexamethasone) for people newly diagnosed with POEMS syndrome. It aims to see how well this treatment helps control the disease and its symptoms.
ShanghaiAges 18–75 - NCT07651631Recruiting
Brain Bleeding Surgery Registry and Treatment Study
This study tracks patients who have had sudden, severe bleeding in the brain and received minimally invasive surgery (a less-invasive surgical approach) to treat it. Researchers want to understand outcomes and improve treatment for this serious condition.
Atlanta, GeorgiaAges 18 years+ - NCT07649031Recruiting
MRI Scanning to Monitor Mouth Cancer Risk in Fanconi Anemia
This study uses MRI (a safe imaging scan) to detect and track dangerous changes in the mouth of people with Fanconi Anemia, a rare genetic condition. The goal is to find a better way to catch oral cancer early.
Minneapolis, MinnesotaAges 18 years+ - NCT07650799Recruiting
Using artificial intelligence to help diagnose rare diseases
This study is testing whether an artificial intelligence (AI) tool can help doctors diagnose rare diseases that have been hard to identify. If you join, the AI will review your medical information and previous test results to suggest possible diagnoses, which might speed up finding the right answer for you.
CangzhouAges birth+ - NCT07648303Recruiting
Study of blood clots in hearts with amyloidosis
This study looks at how often blood clots form in the hearts of people with cardiac amyloidosis (a disease where abnormal proteins build up in heart tissue). Understanding clot risk may help doctors better protect patients from stroke and other complications.
DijonAges 18 years+ - NCT07654647Recruiting
Custom 3D-printed implants for facial bone repair
This trial tests personalized 3D-printed titanium implants to fix facial bone problems, like from cysts, tumors, or birth defects. It's for people who need a custom-shaped implant to improve their face's look and function.
LatakiaAges 18–60 - NCT07652684RecruitingPhase 4
Fat supplement to help premature babies' lungs
This study tests whether adding a special fat supplement to the feeding of very premature babies can help prevent or reduce lung damage, which is common in babies born early. The supplement is given through a feeding tube.
ThessalonikiAges 6 months–7 months - NCT07640893RecruitingPhase 2
Testing a new injection for von Willebrand disease
This study tests whether SR604, a new injection given under the skin, can reduce bleeding episodes in people with von Willebrand disease (a condition where blood doesn't clot properly). The trial will measure how well it works, how safe it is, and how your body processes it.
ChangshaAges 18–65 - NCT07645820Recruiting
Testing a device to detect eczema flares early
This study tests a non-invasive home device called Nevisense Go that may help detect atopic dermatitis (eczema) flares before they become severe. You'll use the device at home, track your symptoms, and visit the clinic a few times to see if it helps predict when flares are coming.
Phoenix, ArizonaAges 12–89 - NCT07644832RecruitingPhase 1/Phase 2
New Blood-Clotting Injection for Hemophilia and Factor VII Deficiency
This early-stage trial tests a new injectable medicine called SR604 designed to help people with hemophilia A, hemophilia B, or congenital Factor VII deficiency—conditions where the blood doesn't clot properly. The study will look at how safe the medicine is and whether it reduces bleeding episodes.
ChangshaAges 18–65 - NCT07640984RecruitingPhase 1/Phase 2
Gene therapy for rare childhood brain disorder MPS IIIB
This early-stage trial tests JR-446, a new treatment for MPS IIIB, a rare genetic disorder that affects the brain and body. The treatment is delivered directly into the fluid around the spinal cord and aims to slow or improve symptoms in young children.
Oakland, CaliforniaAges Up to 5 years - NCT07643766RecruitingPhase 2
Testing ENV-294 for moderate-to-severe atopic dermatitis
This study tests whether a new medication called ENV-294 can help adults with moderate-to-severe atopic dermatitis (eczema) that hasn't responded well to other treatments. The trial will check if the drug reduces itching and skin inflammation safely.
Encinitas, CaliforniaAges 18 years+ - NCT06128226Recruiting
Screening study for CLN2 disease in 6-year-olds
This study is screening children for a rare genetic condition called CLN2 disease. It looks for signs like seizures, speech loss, and movement problems to find out how common this condition is among 6-year-olds.
AdanaAges 2–6
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.