Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,859 recruiting studies in this area right now.
By phase
- Not applicable2,773
- Phase 2283
- Phase 1236
- Phase 3215
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis128
- Congenital Heart Disease85
- 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
- Pennsylvania66
- Ohio57
Recruiting studies3,859 total
- NCT04645160RecruitingPhase 1/Phase 2
Tivozanib for bile duct and gallbladder cancers after chemotherapy
This Phase 1/2 trial tests tivozanib (a targeted anti-cancer drug) in people with bile duct or gallbladder cancer that cannot be removed by surgery and has already been treated with first-line chemotherapy. The study looks at how well the drug works and how safe it is in this setting.
Bethesda, MarylandAges 18 years+ - NCT04645199Recruiting
Long-term follow-up study for blood and bone marrow diseases
This study is collecting long-term follow-up information from people diagnosed with certain blood and bone marrow conditions. It may help doctors understand what happens over time and improve future care, but you’re mainly contributing data rather than receiving a new treatment.
Tianjin, Tianjin MunicipalityAges Any age - NCT04645862Recruiting
4D heart scan for people with a bicuspid aortic valve
This study uses a special 4D heart scan to learn how well your bicuspid aortic valve works. It may help doctors better understand valve function and plan care, but the scan involves imaging contrast in some cases.
Philadelphia, PennsylvaniaAges 18 years+ - NCT04647227RecruitingPhase 4
SEVENFACT® to reduce bleeding in hemophilia with inhibitors
This trial studies whether SEVENFACT® can reduce bleeding episodes in people with hemophilia A or B who have inhibitors (proteins that make standard treatments less effective). It also tests how well people can track bleeding and medicine use during the study.
Phoenix, ArizonaAges 12–100 - NCT04652908RecruitingPhase 1/Phase 2
Cell therapy given during pregnancy to repair spina bifida
This trial studies a cell-based treatment given during fetal surgery to help repair myelomeningocele (spina bifida) before birth. It aims to improve outcomes for the baby by supporting “in utero” repair, but you must meet strict pregnancy and health criteria to be considered.
Sacramento, CaliforniaAges 4 months–6 months - NCT04657822RecruitingPhase 4
Crizanlizumab rollover study for people with sickle cell disease
This study lets people with sickle cell disease who already finished a Novartis crizanlizumab trial continue treatment under a rollover plan. It’s designed for participants who benefited from crizanlizumab and completed the earlier study schedule without major ongoing side effects.
Birmingham, AlabamaAges 6 months–100 years - NCT04661059Recruiting
Study of how a fetus develops late in pregnancy
This study looks at how a baby’s organs develop in late pregnancy, using situations where someone is already being evaluated for a possible fetal abnormality and chooses to end the pregnancy. The goal is to better understand late-stage fetal development and related causes of abnormalities.
LondonAges 18 years+ - NCT04665726Recruiting
Natural history study of Usher syndrome hearing and vision changes
This study follows people with Usher syndrome over time to better understand how hearing and vision symptoms change. It may help researchers measure disease progression and support future treatments.
ParisAges Any age - NCT04671212Recruiting
Using discarded bone marrow from certain orthopedic surgeries
This study looks at bone marrow cells that would otherwise be discarded during some orthopedic surgeries. It may help researchers learn more about specific blood disorders (certain types of thalassemia and sickle cell–related conditions).
Memphis, TennesseeAges Any age - NCT04676048RecruitingPhase 1/Phase 2
Gene therapy study for adults with hemophilia A
This Phase 1/2 study tests a one-time gene therapy (ASC618) to help the body make clotting factor for people with hemophilia A. It may reduce bleeding and the need for factor infusions, but you must meet several safety and eligibility checks.
Little Rock, ArkansasAges 18 years+ - NCT04676828Recruiting
SPECT-guided radiation to avoid healthy lung in lung cancer
This study tests a special imaging-guided radiation plan that aims to treat lung cancer while reducing radiation to healthy lung tissue. It may help lower lung side effects while keeping the cancer treatment focused on tumor areas.
Sydney, New South WalesAges 18 years+ - NCT04677998Recruiting
Personalized monitoring and treatment plan for stomach polyps
This trial tests a personalized plan for regularly checking and treating stomach and first-part of intestine polyps in people with familial adenomatous polyposis (FAP). It may help by catching problems early and choosing the right intervention at the right time.
Houston, TexasAges 18 years+ - NCT04678011Recruiting
Personalized follow-up to prevent cancer after FAP surgery
This trial studies a tailored plan for surveillance (check-ups) and interventions to find and remove new growths early in people with familial adenomatous polyposis (FAP) after bowel surgery. It may help lower the chance of cancer by closely monitoring and treating polyps when they appear.
Houston, TexasAges 18 years+ - NCT04682470Recruiting
Research study following young adults with cancer
This is an observational study that follows young adults with a confirmed cancer diagnosis to learn more about their experience and care. It does not test a new drug, but your information may help improve future cancer treatment and support.
Amsterdam, North HollandAges 18–39 - NCT04686253Recruiting
Study how a heart opening and its flow link to stroke
This study looks at the detailed flow pattern through a patent foramen ovale (PFO)—a small opening in the heart—and how it may relate to stroke, TIA, or migraine. Doctors use special ultrasound testing with contrast to better understand who is most affected.
Beijing, Beijing MunicipalityAges 16–65 - NCT04691622RecruitingPhase 1
Treat chronic norovirus using donor immune cells
This Phase 1 study tests whether giving immune cells made from a donor can help children and adults with long-lasting (chronic) norovirus infection. It’s aimed at people whose immune system is weakened after a stem cell transplant, solid organ transplant, or due to primary immune problems.
Washington D.C., District of ColumbiaAges 3 months–80 years - NCT04694248Recruiting
Blood thinner plus antiplatelet after iliac vein stent
This trial looks at whether taking a blood thinner and an additional clot-prevention medicine (antiplatelet) after iliac vein stenting can improve treatment of certain deep vein clots. It may help prevent the clot from coming back or spreading, but it also increases bleeding risk, so careful eligibility screening is important.
Hangzhou, ZhejiangAges 18 years+ - NCT04695028Recruiting
Study to better understand crystal-related joint pain
This study looks at people with certain types of crystal deposits in joints (like gout, CPPD, or hydroxyapatite crystal disease). It uses scans such as ultrasound and a special CT-type scan called DECT to confirm and better understand what’s causing your symptoms.
LommeAges 18 years+ - NCT04700358Recruiting
Study of antibacterial antibodies in adults with cystic fibrosis
This study looks at how your body’s antibacterial antibodies work in people with cystic fibrosis. It may help researchers understand immune responses in cystic fibrosis and guide better future treatments.
CologneAges 18 years+ - NCT04704089Recruiting
Dental enamel defect study using tooth photos and measurements
This study compares different types of enamel problems by taking detailed pictures of your front teeth. It may help researchers better tell these enamel conditions apart and improve future diagnosis and care.
ParisAges 8–55 - NCT04706910RecruitingPhase 3
PET scan study to optimize imaging in several diseases
This trial improves a special PET scan (a type of brain/body imaging) using 18F-DOPA, to better show how certain conditions behave in the body. If you or your child may have one of the listed conditions, the study may help your doctors get clearer scan results.
Edmonton, AlbertaAges Any age - NCT04708431Recruiting
Study of health and wellbeing in people with androgen insensitivity
This study looks at the long-term health and wellbeing of people with androgen insensitivity, a genetic condition that affects how the body responds to androgens (male-type hormones). It may help your care team understand what to watch for over time and improve future guidance for patients and families.
Bethesda, MarylandAges 8–99 - NCT04713657RecruitingPhase 1
Beta-blocker study for heart muscle function in young babies
This trial tests whether a beta-blocker called propranolol can improve how well heart muscle cells work in very young babies with certain congenital heart conditions. It mainly looks at safety and how the medicine affects the heart, which could help guide future treatments.
New York, New YorkAges 4 weeks–2 months - NCT04712812Recruiting
Study of childhood-onset hereditary spastic paraplegia
This study follows people with hereditary spastic paraplegia that started in childhood to better understand how the condition progresses over time. It may help doctors learn what to expect and how to plan care, using genetic information and family history.
Boston, MassachusettsAges Up to 30 years
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,859 recruiting medical genetics studies from the U.S. registry right now, out of 5,745 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.