Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,762 recruiting studies in this area right now.
By phase
- Not applicable2,682
- Phase 2280
- Phase 1239
- Phase 3209
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease133
- Atopic Dermatitis124
- Congenital Heart Disease84
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy36
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)29
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
- Children's Hospital of Philadelphia23
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
Where studies are running
- California325
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York85
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT04001842Recruiting
Tested new jaw bone repair to support future dental work
This trial is studying a method to rebuild part of the lower jaw using a blood-supply bone replacement, so you can later get dental rehabilitation. It’s for adults who need a sizable, tumor-free jaw bone reconstruction and who can safely undergo surgery.
AlexandriaAges 18–65 - NCT03900286Recruiting
Testing a low-calorie diet for partial fat loss diabetes
This trial studies whether a low-calorie (low energy) diet can improve diabetes control in adults with familial partial lipodystrophy. It likely helps by changing how your body handles blood sugar and fat use, but you must meet specific safety and eligibility checks.
CambridgeAges 18–99 - NCT05231876Recruiting
French Wilson disease registry
This study keeps a registry (a confidential list) of people with Wilson disease. It helps researchers learn more about the disease and improve future care, without requiring specific treatment changes.
Paris, Île-de-France RegionAges birth–99 years - NCT05500989Recruiting
Compare early pregnancy blood-flow markers in preeclampsia
This study looks at blood-flow and artery health signs during pregnancy to better understand why preeclampsia happens. It compares women with preeclampsia to women with an uncomplicated pregnancy, and may help improve early detection and care.
MaastrichtAges 18 years+ - NCT06718842Recruiting
Walking program for children with PKU and fatty liver
This study tests whether a walking program can help children with PKU who also have fatty liver and obesity. It aims to see if regular walking improves liver health and overall fitness.
Dokki, Giza GovernorateAges 9–12 - NCT06712095Recruiting
Video Capsule for Lynch Syndrome Screening
This study tests whether a video capsule (a pill-sized camera you swallow) can help find polyps in people with Lynch syndrome, without needing a colonoscopy. It may be an easier way to screen for colon cancer.
London, (Select)Ages 18 years+ - NCT04469517Recruiting
Study of low-oxygen factors in hereditary bleeding disorder
This study looks at how “low-oxygen” signals may affect people with hereditary hemorrhagic telangiectasia (HHT), a genetic bleeding disorder. It may help researchers understand what drives symptoms and could guide future treatments.
Essen, North Rhine-WestphaliaAges 18 years+ - NCT07591298RecruitingPhase 2
SYH2070 injection for homozygous familial high cholesterol
This study tests a new injectable medication (SYH2070) designed to lower cholesterol in people with homozygous familial hypercholesterolemia—a genetic condition that causes extremely high cholesterol levels despite treatment. The goal is to see if this drug safely reduces cholesterol better than current options.
Changsha, HunanAges 18 years+ - NCT07594236RecruitingPhase 1
Experimental Cell Therapy for Retinal Degeneration
This early-stage study tests a new cell-based treatment (C.001) for inherited retinal diseases that affect the back of the eye. The treatment may help slow vision loss or improve sight in people with geographic atrophy or Stargardt disease.
Beverly Hills, CaliforniaAges 18 years+ - NCT07587242RecruitingPhase 3
Testing a New Therapy for DMD with Exon 44 Mutations
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
Westmead, New South WalesAges 7–16 - NCT07591584Recruiting
Digestive Enzyme Blend for Bloating and Gas Relief
This study tests whether a daily digestive enzyme supplement designed to break down certain hard-to-digest foods can reduce bloating and belly pain. You'll use a smartphone app to track your symptoms over several weeks to see if the enzyme helps.
Los Angeles, CaliforniaAges 18 years+ - NCT07592585RecruitingPhase 3
Testing VC005 cream for mild-to-moderate eczema in teens and adults
This study tests a new medication called VC005 to treat atopic dermatitis (eczema)—a skin condition causing itching, redness, and inflammation. Researchers want to see if VC005 can safely reduce symptoms in people aged 12 and older with mild-to-moderate cases.
Nanjing, JiangsuAges 12 years+ - NCT07587021RecruitingPhase 2
YOLT-203 treatment study for primary hyperoxaluria type 1
This study tests a new drug called YOLT-203 for people with primary hyperoxaluria type 1 (PH1), a rare genetic condition where the body produces too much oxalate, which can damage the kidneys. The trial aims to see if this drug can help reduce oxalate levels and prevent kidney damage.
Birmingham, AlabamaAges 6 years+ - NCT06032273Recruiting
Lung Transplant Study for CF Patients and Their Caregivers
This study looks at a website-based program to support people with cystic fibrosis (CF) and their caregivers, helping them prepare for a possible lung transplant. It aims to ease stress, provide information, and improve the transplant journey.
Seattle, WashingtonAges 18 years+ - NCT06067958RecruitingPhase 3
Nose spray medicine for pain during eye exams in preemies
This study tests a nose spray medicine (dexmedetomidine) to help reduce pain and discomfort in premature babies who need eye exams for retinopathy of prematurity (a condition that can affect vision). The goal is to see if the spray works better than the usual care for calming babies during the exam.
Be’er Ya‘aqov, CenterAges 4 weeks+ - NCT06023472Recruiting
Comparing sperm sorting methods for better genetic testing in IVF
This study compares two ways of preparing sperm for IVF (a microfluidic chip versus a centrifuge method). It aims to see which method leads to more genetically normal embryos (euploid) when doing PGT (pre-implantation genetic testing).
Hong Kong, Hong KongAges Up to 43 years - NCT06082713Recruiting
Extracellular Vesicles Study for Huntington's Disease
This study is collecting tiny particles called extracellular vesicles from blood and spinal fluid to learn more about Huntington's disease. It aims to find better ways to measure the disease in people who have HD and in those who do not.
Orlando, FloridaAges 18–75 - NCT07587684RecruitingPhase 1
Testing IN026 for Hard-to-Treat Gout
This study tests a new drug called IN026 for people with refractory gout—gout that doesn't respond well to current treatments and causes severe symptoms. The goal is to see if IN026 can lower uric acid levels and reduce gout attacks.
WuhanAges 18–75 - NCT07590882Enrolling by invitation
Sports Recovery After Hip Realignment Surgery
This study follows patients who had hip socket realignment surgery (PAO) to understand how well they return to sports and physical activities. Researchers will review medical records from the past 7+ years to learn what helps patients get back to the sports they love.
BolognaAges 12–40 - NCT06719830Recruiting
Finding the exact spot of a heart extra wire
This study looks at a new way to figure out exactly where a tiny extra electrical wire in your heart (called a septal accessory pathway) is located, to help guide the procedure that fixes it. It might help make the treatment more precise and safe.
Asyut, Asyut GovernorateAges Any age - NCT06720571Recruiting
Nerve stimulation for emotion and thinking in Prader-Willi syndrome
This trial tests whether a gentle, non-invasive nerve stimulation (like a small device on the neck) can help people with Prader-Willi syndrome regulate emotions and improve planning and focus. It may be a new option if you have trouble with emotional control or daily organization.
BordeauxAges 18 years+ - NCT02505464Recruiting
Pregnancies with baby birth differences
This study looks at pregnancies that are considered high-risk because the baby is affected by a difference found during pregnancy or at birth. It may help researchers understand and improve care for families dealing with these fetal (baby) anomalies.
Memphis, TennesseeAges 1 minute–50 years - NCT07585097Recruiting
Long-term Safety Study of Odevixibat for Alagille Syndrome
This study follows patients with Alagille syndrome who are taking odevixibat to see how safe the medicine is over time. Alagille syndrome is a rare liver disease that affects bile flow, and odevixibat is a treatment that may help reduce itching and improve liver function.
BronAges 6 months+ - NCT07580365Recruiting
Virtual Reality Training for Kids With Muscle Diseases
This study tests whether a virtual reality-based exercise program can help children and teens with diagnosed muscle diseases improve their strength and function. Participants use a special exercise bike while wearing a VR headset to make training more engaging.
Bosisio Parini, LCAges 6–18
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,762 recruiting medical genetics studies from the U.S. registry right now, out of 5,456 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.