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
- NCT07690527Enrolling by invitationPhase 1
Long-term follow-up for RB001 gene therapy in children with Phelan-McDermid syndrome
This study follows children who completed the RB001 gene therapy trial for Phelan-McDermid syndrome (caused by changes in the SHANK3 gene) to monitor their health and progress over a longer period of time.
Beijing, ChinaAges Any age - NCT07692256RecruitingPhase 2
Empagliflozin for heart failure in children
This trial tests if empagliflozin, a diabetes medicine that also helps the heart, can improve symptoms in children with heart failure. It may help kids with severe symptoms or high levels of a heart stress marker called BNP.
Cairo, Cairo GovernorateAges 1 month–18 years - NCT07697222Recruiting
Pain block comparison for pectus excavatum repair
This trial compares two types of nerve blocks to control pain after a minimally invasive surgery to fix a sunken chest. It aims to see which block works better for recovery.
Istanbul, IstanbulAges 15–25 - NCT05030337Recruiting
Closed-loop oxygen control for very preterm babies on ventilators
This trial tests a computer-guided (“closed-loop”) oxygen control system for very preterm babies who need breathing support. It aims to keep oxygen levels safer and more consistent than standard care.
LondonAges Any age - NCT06655662RecruitingPhase 1
Gene therapy for severe beta-thalassemia
This trial tests a one-time gene therapy using your own blood stem cells to treat severe beta-thalassemia. It may help you stop needing regular blood transfusions.
GuangxiAges 6–35 - NCT07686341Recruiting
Precision oxygen monitor study for infants
This trial tests a new type of oxygen monitor (pulse oximeter) in babies under 1 year old. It helps doctors better track oxygen levels in critically ill infants to improve care.
Memphis, TennesseeAges birth–1 year - NCT07681089RecruitingPhase 2
Foscenvivint for HIV/HCV cirrhosis with hemophilia
This trial tests a drug called foscenvivint for people with hemophilia and liver cirrhosis caused by both HIV and hepatitis C (already treated). The study aims to see if it can help improve liver health.
Sapporo, HokkaidoAges 18–79 - NCT07688538Recruiting
Mapping vision system development in embryos
This trial studies embryos to understand how the visual system develops normally and abnormally. It compares tissues from embryos with developmental problems to healthy ones, aiming to create a detailed map of early vision system growth.
Guangzhou, GuangdongAges Any age - NCT07682883Enrolling by invitation
Mouth screening to prevent oral cancer
This study checks how well routine mouth exams can find early signs of oral cancer. It is for adults who do not already have a condition called OPMD (a mouth problem that can lead to cancer) and are not being treated for it.
Warsaw, Masovian VoivodeshipAges 18 years+ - NCT07686653Recruiting
Genome sequencing for unusual psychiatric conditions
This study uses trio genome sequencing (comparing DNA from you and both of your parents) to find genetic causes for psychiatric disorders that start early, are hard to treat, or have an unusual pattern. It may help if you or your child have a mental health condition without intellectual disability or birth defects.
DijonAges 3–50 - NCT07682519Recruiting
Home vs. in-person rehab for hemophilia pain
This study compares a rehabilitation program you can do at home using video calls (telerehabilitation) with in-person rehabilitation sessions. The goal is to see if the home-based option works as well as office visits for reducing chronic joint pain in adults with hemophilia.
Santiago, Santiago MetropolitanAges 18–60 - NCT07685210RecruitingPhase 1
GenSci144 tablet safety study for healthy volunteers
This trial tests the safety of a new tablet called GenSci144 in healthy adults. It helps doctors see if the drug is safe and how the body handles it.
Jinan, ShandongAges 18–45 - NCT05927519Recruiting
Compare a laryngoscope tool in obese vs nonobese men
This study compares how well a specific breathing tube placement tool (Airtraq) works in men who are obese versus men who are not, during planned surgeries. It may help improve the safety and ease of putting in a breathing tube.
IamitAges 18–80 - NCT04561557RecruitingEarly Phase 1
Cell therapy for relapsing nervous system autoimmune diseases
This early-phase trial tests CT103A “CAR-T” cell therapy to treat certain relapsing or treatment-resistant autoimmune diseases that affect the nervous system. It aims to see whether the cells are safe and may help reduce disease activity when standard treatments haven’t controlled symptoms.
Wuhan, HubeiAges 18–75 - NCT07673809RecruitingPhase 1/Phase 2
Gene therapy trial for Duchenne muscular dystrophy in young boys
This trial tests a gene therapy called GNR-097 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help improve muscle function, and the study will check how safe and effective it is.
MinskAges 4–9 - NCT07672756RecruitingPhase 1
Testing PL54 injection for adults with PKU
This study tests a new injection called PL54 to see if it is safe and tolerable for adults with phenylketonuria (PKU), a condition where the body cannot break down an amino acid called phenylalanine. It is designed for people with high blood phenylalanine levels and may offer a new treatment option.
Hefei, AnhuiAges 18–55 - NCT07672210Recruiting
Pregnancy-related aortic dissection in China
This study is looking at cases of aortic dissection that happen during pregnancy or shortly after childbirth in China. It aims to understand how these events occur and how they can best be managed.
Shanghai, Shanghai MunicipalityAges 16–55 - NCT07674290RecruitingPhase 4
Study of MC4R Agonist for Bardet-Biedl Syndrome and Genetic Obesity
This trial tests a drug called setmelanotide for people with Bardet-Biedl Syndrome (a rare genetic condition) or other severe genetic obesity. It looks at how well the drug works in real-world settings, not just in a lab.
EssenAges Any age - NCT07675798Recruiting
Caudal block for reducing fistula after hypospadias repair
This study tests whether a numbing injection into the lower back (caudal block) reduces the risk of a leak or fistula after hypospadias repair surgery in young boys. It may help your child have fewer complications and a smoother recovery.
Diyarbakır, Eyalet/YerleşkeAges 6 months–5 years - NCT07673302Recruiting
Study of drugs to boost fetal hemoglobin in beta thalassemia
This trial tests if certain drugs can safely increase fetal hemoglobin (a type of hemoglobin that helps carry oxygen) in people with beta thalassemia major. It may offer a new way to manage the condition and reduce the need for transfusions.
Rawalpindi, Punjab ProvinceAges Any age - NCT07672275Recruiting
How blood transfusions affect stress markers in newborns
This study looks at how blood transfusions change stress-related substances in the blood of newborns. It might help doctors understand how transfusions affect babies in the NICU.
Graz, StyriaAges 5 minutes–5 months - NCT07674758Recruiting
Understanding heart risk in Duchenne and Becker muscular dystrophy
This study looks at heart health in people with Duchenne or Becker muscular dystrophy to find better ways to predict and track heart problems. The goal is to improve future treatment trials.
Sacramento, CaliforniaAges Any age - NCT05602376Recruiting
HIV messages to help men get tested and stay in care
This study tests whether special “U=U” style messages can improve how men move from HIV testing to starting treatment and continuing care. You may be asked to receive guidance and information meant to help you stay engaged in HIV treatment.
East London, Eastern CapeAges 15 years+ - NCT06311708Recruiting
PKP2-related heart condition natural history study
This study looks at how plakophilin 2 (PKP2)-related arrhythmogenic right ventricular cardiomyopathy (ARVC) progresses over time. You'll give blood samples to check for antibodies to a virus used in gene therapy, and help doctors understand the disease better.
San Francisco, CaliforniaAges 14–65
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.