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 Disease83
- 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
- California324
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York84
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT07380594Recruiting
Study of psychiatric symptoms in White-Sutton syndrome
This study looks at mental health symptoms in people with White-Sutton syndrome, a rare genetic condition. By answering questions, you can help doctors better understand what challenges people face and how to support them.
DijonAges 6 years+ - NCT07381985Enrolling by invitation
Managing hereditary cancer risk in rural areas
This study tests a way to help people with inherited cancer gene mutations manage their health, especially if they live in rural areas. You join if you found out about the mutation more than a year ago.
Burlington, VermontAges 18 years+ - NCT07385443Recruiting
Spanish registry for myotonic dystrophy type 1
This registry collects information from people with myotonic dystrophy type 1 (DM1) to better understand the condition and improve care. Anyone with a confirmed genetic diagnosis can join.
Multiple Locations, AndalusiaAges Any age - NCT07381413Recruiting
Eye tracking study for thyroid eye disease
This study uses eye tracking to compare how people with thyroid eye disease move their eyes and pay attention to visual cues. It may help researchers understand eye movement problems better.
ShanghaiAges 18–70 - NCT06821386Recruiting
Genetic testing for seriously ill infants in intensive care
This study offers genetic testing for infants under 18 months who are in intensive care with certain health problems that may be genetic. The goal is to find a genetic cause quickly, which could guide care and treatment.
TaipeiAges Up to 1.5 years - NCT05454774RecruitingEarly Phase 1
Gene therapy for adults with severe hemophilia A
This early-phase study tests an FVIII gene therapy to help your body make more of the missing clotting factor in hemophilia A. It’s aimed at adults with severe low FVIII levels who have already used FVIII replacement products for a long time and have not developed an immune blocker (an inhibitor).
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT05709288RecruitingPhase 1
Gene therapy for teens with hemophilia B
This Phase 1 study tests a one-time gene therapy treatment (BBM-H901) for boys ages 12 to 18 with hemophilia B. It aims to see if the treatment is safe and can help the body make more clotting factor over time.
Tianjin, Tianjin MunicipalityAges 12–18 - NCT05629559RecruitingPhase 1/Phase 2
Study drug 4D-310 for Fabry disease with heart problems
This Phase 1/2 study tests how safe and potentially effective 4D-310 is for adults with Fabry disease who have heart involvement. It may help people who can’t tolerate standard treatments like enzyme replacement therapy (ERT) or migalastat, or who still have disease progression.
MelbourneAges 18–65 - 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+ - NCT06425666Recruiting
Cataract surgery with triple-DMEK for Fuchs dystrophy
This trial tests a combined surgery that treats both cataract and Fuchs endothelial corneal dystrophy at the same time. It compares the standard cataract surgery to one that also replaces the damaged cornea layer, aiming to improve vision with fewer steps.
Aarhus N, Central JutlandAges 18 years+ - NCT06840717Recruiting
Genetic links to type 2 asthma in kids
This study looks at how certain genes might be connected to type 2 asthma in children. It aims to find out which kids are more likely to have asthma so doctors can better treat it.
Guangzhou, GuangdongAges 3–14 - NCT06509984RecruitingPhase 2
Testing apremilast for genetic EBS with frequent blisters
This study tests whether a daily pill called apremilast can reduce the number of new blisters in people with a genetic form of EBS (epidermolysis bullosa simplex). If you get more than 3 new blisters per day and have a confirmed KRT5 or KRT14 mutation, this trial may be a good fit.
Nice, FranceAges 6–99 - NCT05641610RecruitingPhase 1/Phase 2
Study drug for adults with hemophilia B who still bleed
This Phase 1/2 study tests ZS801, a gene-related treatment, to see how safe it is and whether it helps prevent bleeding in adults with hemophilia B. You may be considered if you’ve had frequent bleeds or joint problems and meet several lab and health requirements.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT05630651Recruiting
ZS801 for adults with severe hemophilia B
This trial studies ZS801, a gene therapy, in adults with hemophilia B who have very low natural FIX (a clotting protein) and frequent bleeding or joint problems. It aims to see how well it works and whether it is safe, especially by monitoring blood counts, infections, and immune responses.
Tianjin, Tianjin MunicipalityAges 18–65 - NCT06065215Recruiting
Early-life MRI to Predict Breathing Problems in Extremely Preterm Infants
This study uses an MRI scan to look at how the lungs develop in babies born very early (before 29 weeks of pregnancy). The goal is to see if early MRI images can help predict future breathing problems, so doctors can better support these infants.
Ottawa, OntarioAges 8 months–1.8 years - NCT06316778Recruiting
Pelvic floor training for women with myotonic dystrophy
This study tests whether pelvic floor muscle training (exercises to strengthen the muscles that control urine flow) can help women with myotonic dystrophy type 1 who have bladder leakage. It's a gentle, non-surgical approach that may improve quality of life.
Jonquière, QuebecAges 18 years+ - NCT07374913Recruiting
Study of COL4A1 and COL4A2 gene mutations
This study looks at people with a mutation in the COL4A1 or COL4A2 genes, which can cause problems in small blood vessels throughout the body. It aims to learn more about the condition and may also include family members who carry the mutation or who can serve as healthy controls.
Florence, FIAges Any age - NCT07377162Recruiting
Hyperoxia effects on brain blood flow in ICU patients
This trial studies how giving extra oxygen (hyperoxia) affects blood flow in the brain for patients on a breathing machine in the intensive care unit. The goal is to see if short bursts of higher oxygen levels are safe and how they change brain activity.
BrusselsAges 18 years+ - NCT07374354Recruiting
Pain and anxiety control for kids with MIH
This study tests a pain-relieving medicine given before dental treatment to see if it helps reduce pain and anxiety in children with molar incisor hypomineralization (MIH), a condition that causes weak enamel on molars and front teeth.
GizaAges 6–12 - NCT07372898Recruiting
Helping parents create a calm home for preterm babies after surgery
This study tests a program called H-HOPE that helps parents create a calm, supportive environment for their baby after major surgery. The goal is to improve the baby's recovery and reduce stress for the whole family.
Chicago, IllinoisAges 1 week–2 months - NCT07371767RecruitingEarly Phase 1
Gene editing for high triglycerides in kids with FCS
This trial tests a new gene-editing treatment (CS-121) for children and teens with a rare inherited condition called Familial Chylomicronemia Syndrome (FCS). It aims to lower very high triglyceride levels and reduce the risk of pancreatitis.
Shanghai, Shanghai MunicipalityAges 4–18 - NCT07373821Recruiting
Lying face up after cornea transplant surgery
This study tests if lying face up after DMEK surgery (a cornea transplant for Fuchs dystrophy) helps the surgery work better. It may be an option for people with Fuchs dystrophy or bullous keratopathy who can lie flat on their back.
HeidelbergAges Any age - NCT07375602Recruiting
AI tool to predict complications after VSD closure in children
This study uses artificial intelligence to predict complications in children who have had a specific type of hole in the heart (called a perimembranous ventricular septal defect) repaired with a catheter-based procedure. The goal is to help doctors better identify which children might have problems after the procedure.
Shanghai, Shanghai MunicipalityAges Up to 18 years - NCT07378423Recruiting
Survey on signs of cancer at birth
This trial asks newly diagnosed children and young adults with certain types of cancer to fill out a questionnaire about signs of cancer present at birth. The answers may help improve early detection of childhood cancer.
BernAges Up to 21 years
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 5,482 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.