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
- NCT06238908RecruitingEarly Phase 1
Gene therapy NGGT003 for severe hemophilia A
This early-phase trial tests a new gene therapy called NGGT003 for men with severe hemophilia A. It aims to see if the treatment is safe and can help reduce bleeding episodes.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT06239116RecruitingPhase 1/Phase 2
RM-718 for obesity from hypothalamus injury or Prader-Willi syndrome
This study tests a new medicine called RM-718 for people with severe obesity linked to a specific brain pathway (MC4R). It may help if your obesity is due to a brain injury or tumor affecting the hypothalamus, or if you have Prader-Willi syndrome.
Birmingham, AlabamaAges 12–65 - NCT06242379RecruitingPhase 1/Phase 2
Testing stem cell particles for retinitis pigmentosa
This trial tests whether tiny particles from stem cells, called extracellular vesicles, can help people with retinitis pigmentosa. It aims to see if these particles are safe and can improve vision.
Bangkok Noi, BangkokAges 18 years+ - NCT06244940Recruiting
Genetic testing study for heart defects in pregnancy
This study tests a new way to find genetic causes of congenital heart disease (a heart problem found before birth) using samples from amniocentesis or chorionic villus sampling. It may help parents get a clearer diagnosis and plan for their baby's care.
San Diego, CaliforniaAges 18 years+ - NCT06245356RecruitingPhase 2
A safety trial of trifluridine/tipiracil for DPD-deficient patients
This trial tests a chemotherapy drug (trifluridine/tipiracil) in people with metastatic colorectal or gastroesophageal cancer who have a DPD deficiency. DPD deficiency means your body may have trouble breaking down certain chemotherapy drugs, which can lead to serious side effects. This study aims to see if this drug is safe and effective for you.
AmiensAges 18 years+ - NCT06247085RecruitingPhase 3
A study testing pegtibatinase for classical homocystinuria
This trial tests an injectable drug called pegtibatinase for people with a rare condition called homocystinuria. It aims to lower high levels of homocysteine in the blood compared to a placebo, while you continue your usual treatments.
Phoenix, ArizonaAges 12–65 - NCT06250257RecruitingPhase 3
Bromocriptine for Heart Weakness in Women 18-50
This trial tests a medicine called bromocriptine to see if it helps women with a weak heart muscle (dilated cardiomyopathy). It is for women between 18 and 50 whose heart weakness was found recently or is caused by blocked arteries, and who are not pregnant or planning pregnancy.
Jimma, OromiyaAges 18–50 - NCT06250595Recruiting
European rare blood disorder registry
This study is building a registry of people with rare blood diseases. By joining, you help doctors learn more about these conditions and improve future care.
Barcelona, CataloniaAges Up to 100 years - NCT06251778Recruiting
National study of ATTR heart amyloid patients on tafamidis
This study follows patients with a specific type of heart amyloidosis (wild-type ATTR) who are taking or could start the drug tafamidis. It aims to learn more about how the condition and treatment affect people over time.
PaviaAges 18–99 - NCT06255886RecruitingPhase 4
Treatment for acid reflux in babies under 1 year
This trial tests a treatment for babies under 1 year old who have frequent and troublesome reflux (GERD). It aims to find a safe and effective way to reduce symptoms like crying, feeding problems, and breathing pauses.
Aabenraa, Region SyddanmarkAges 1 month–1 year - NCT06259292Recruiting
HHT patient registry tracking long-term outcomes
This registry collects health information from people with HHT over time to better understand the disease and improve care. Joining does not involve any experimental treatment, but your data will help researchers learn more about HHT.
Birmingham, AlabamaAges Any age - NCT06259669Recruiting
Studying eczema cream safety during pregnancy
This study compares the health of mothers and their babies in two groups: women with eczema who used ruxolitinib cream during pregnancy and those who did not. It aims to see if the cream is safe for both mother and child.
Morrisville, North CarolinaAges 15–50 - NCT06260059RecruitingPhase 4
Jardiance for adults with congenital heart disease and reduced heart function
This study tests a medicine called Jardiance (an SGLT2 inhibitor) to see if it helps improve heart function in adults with congenital heart disease who have recently experienced a drop in their heart pumping ability. Participants will take the medication alongside standard heart failure treatments.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06260891RecruitingPhase 2
Zinc study for sickle cell disease and bone health
This study tests if taking zinc supplements can improve bone health in people with sickle cell disease. It's for those with certain types of sickle cell disease (SS or S-Beta zero thalassemia) who are not on chronic transfusions or zinc supplements.
Oakland, CaliforniaAges 15–40 - NCT06261346Recruiting
Growth factors in corneal transplant surgery
This study tests whether adding a special growth factor solution to the fluid used during corneal transplant surgery helps the transplant heal better. It is for patients undergoing a specific type of corneal transplant called DSEK or DMEK.
Miami, FloridaAges 18–80 - NCT06261710Recruiting
Ultrasound in labor: fewer internal exams
This study uses ultrasound during labor to see if it can reduce the number of internal exams (like cervical checks) you need. It aims to make labor more comfortable and less invasive for mothers with a head-down baby in spontaneous or induced labor.
BerlinAges 18–60 - NCT06263075Recruiting
Monitoring during craniosynostosis surgery: comparing newer vs. traditional monitors
This study compares two types of monitors that track your baby's heart and blood flow during surgery to fix craniosynostosis. The goal is to see if a newer monitor works as well as the traditional one.
Bologna, BOAges 3 months–8 months - NCT06264531RecruitingPhase 2/Phase 3
Bevacizumab for adults with symptomatic, untreatable brain AVMs
This trial tests a medication called bevacizumab (given as an IV infusion) to see if it can improve symptoms in people with brain arteriovenous malformations (AVMs) that cannot be treated with standard therapies. It is for those whose AVMs cause significant disability.
Paris, Île-de-France RegionAges 18 years+ - NCT06264570Recruiting
Personalized supplements for high homocysteine and cholesterol
This study tests a personalized approach based on your genes to choose the right natural supplements for people with high homocysteine and high LDL cholesterol. It aims to help lower these levels safely.
Novosibirsk, Novosibisk RegionAges 18–80 - NCT06264934Recruiting
Testing a new breast scanner using electric fields
This study is testing a new type of scanner that uses safe electric fields to create images of breast tissue. It may help doctors see breast changes without needing a biopsy or X-ray. By joining, you help researchers understand how well this scan works.
LondonAges 18 years+ - NCT06265363Recruiting
Eye screening study for heavier or older premature babies
This study looks at how often premature babies weighing more than 1500 grams or born at 33 weeks or later get a serious eye condition called ROP (retinopathy of prematurity). It aims to find out what puts these babies at risk and what happens to them, helping doctors improve screening guidelines.
AnkaraAges 4 weeks+ - NCT06267508Recruiting
HIV testing and treatment for newborns
This study looks at ways to test and treat newborns for HIV early to improve their long-term health. It is for mothers with HIV whose babies are less than 3 days old and who are willing to have their babies tested and followed at home.
BeiraAges 18 years+ - NCT06267859RecruitingEarly Phase 1
New treatments for frequently ill children
This study tests new ways to help children who get sick often (frequently ill children) feel better and avoid common illnesses. It focuses on children who do not have severe chronic diseases.
Tashkent, Yunus-obad DistrictAges 3 days–15 years - NCT06270199RecruitingPhase 2
Stem cells for lung disease in preterm infants
This trial tests if stem cells can help premature babies with severe lung disease (bronchopulmonary dysplasia). It is for very small, early-born babies who still need a breathing machine between 5 and 14 days after birth.
Pozuelo de Alarcón, MadridAges 1 month–6 months
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.