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
- NCT06915441RecruitingPhase 2
Lipid infusions to improve nutrition for premature babies
This trial tests whether giving fat (lipid) infusions can improve nutrition and growth in very premature or very low birth weight babies. It may be for babies born before 28 weeks or weighing 1,000 grams or less.
Houston, TexasAges 12 hours–6 months - NCT06921733Recruiting
Ultrasound study for children with kidney and urinary birth defects
This trial uses a special ultrasound technique to create detailed images of blood flow in the kidneys of children with congenital kidney and urinary tract conditions. It aims to improve how doctors assess blockages without needing radiation.
Erlangen, BavariaAges birth–6 years - NCT06918119Recruiting
Spinal cord stimulation for children with spina bifida
This study tests whether a special type of electrical stimulation applied to the skin over the lower spine can help improve function in children and teenagers with spina bifida. It aims to see if this treatment can make daily activities easier.
Rochester, MinnesotaAges 5–18 - NCT06919224Recruiting
Text message support for adults with sickle cell disease
This study uses text messages to help adults with sickle cell disease learn about the latest medical guidelines and tools to manage their health. The goal is to see if this mobile approach improves how people follow their care plan.
Chicago, IllinoisAges 18 years+ - NCT06911749Recruiting
Using MSOT and MRI to measure bowel transit in children
This trial tests whether two imaging techniques, MSOT and MRI, can safely and non-invasively measure how long it takes for food to move through the bowels in children with chronic constipation or other bowel emptying problems. It may help doctors better understand and treat these conditions.
Erlangen, BavariaAges 1 year+ - NCT06921213RecruitingPhase 2
Oral antibiotics for newborns exposed to syphilis
This study tests whether giving oral antibiotics at home is as safe and effective as the usual injections for newborns exposed to syphilis during pregnancy. It is for babies who appear healthy at birth.
JakartaAges birth–1 week - NCT06918392Recruiting
Testing a questionnaire for teens with pectus excavatum
This study tests a questionnaire about living with pectus excavatum (sunken chest). If you are a teen (12-18) scheduled for the Nuss procedure, you and your parent can help by filling out the questionnaire to see if it works well.
HeerlenAges 12–18 - NCT06921720Recruiting
Phosphate diabetes muscle study using phosphorus scan
This study uses a special MRI scan to look at phosphorus levels in the muscles of people with phosphate diabetes (a condition where the body loses too much phosphate through the kidneys). It aims to better understand how this condition affects muscles and may help guide future treatments.
BronAges 10 years+ - NCT06914726Enrolling by invitation
Cancer risk follow-up care for hereditary syndromes
This study tests a tool to help primary care doctors provide better cancer prevention care for people with hereditary breast and ovarian cancer (HBOC) or Lynch syndrome (LS). It aims to ensure you're up-to-date on recommended screenings and preventive steps.
Minneapolis, MinnesotaAges 18 years+ - NCT06919757Recruiting
Early Lung Health in Very Premature Infants
This study compares the early lung development of very premature babies who had growth problems in the womb to those who did not. The goal is to understand how growth in the womb affects lung health after birth.
RomaAges Any age - NCT06918795Recruiting
Fontan Fitness Trial: Home exercise for better heart health
This study tests whether a home-based exercise training program can improve fitness in people with Fontan circulation. Participants will do regular aerobic exercise and be monitored for changes in heart and lung function over time.
Philadelphia, PennsylvaniaAges 10–17 - NCT06590220Recruiting
Predicting treatment needs for moderate to severe thyroid eye disease
This study aims to build a model that helps doctors predict how thyroid eye disease (TAO) will progress, specifically for patients with moderate to severe symptoms. If you have had TAO and meet the activity criteria, your participation could lead to better personalized care.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT03676868Recruiting
Study of brain blood vessel malformations and outcomes
This study looks at how brain arteriovenous malformations (abnormal connections between arteries and veins in the brain) behave over time, and how different treatment choices affect outcomes. It may help researchers better predict prognosis and guide future care.
ParisAges birth+ - NCT06906367Recruiting
Long-term study of Fabry disease treatments in the US
This study looks at how well available Fabry disease treatments (like migalastat or ERT) work over time in real life. It may help doctors understand who benefits most from these medicines.
Birmingham, AlabamaAges 18 years+ - NCT06910813RecruitingPhase 1/Phase 2
Gene therapy trial for children with cystinosis
This trial tests a new gene therapy called DFT383 for children aged 2 to 5 with nephropathic cystinosis. It aims to see if the treatment is safe and can help improve kidney function.
Phoenix, ArizonaAges 2–5 - NCT06902558RecruitingPhase 2
Study of ABBV-CLS-628 for adults with polycystic kidney disease
This trial tests a new drug, ABBV-CLS-628, for people with autosomal dominant polycystic kidney disease (ADPKD). The goal is to see if it slows kidney function decline and is safe to use.
Birmingham, AlabamaAges 18–55 - NCT06908226Enrolling by invitationPhase 3
LP352 for children and adults with DEE
This trial tests a medication called LP352 for children and adults with certain types of epilepsy (Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathies). It's for people who already completed a previous study of LP352 and may benefit from continued treatment.
Little Rock, ArkansasAges 2–66 - NCT06904261RecruitingPhase 3
Study of migalastat for children with Fabry disease
This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.
Atlanta, GeorgiaAges 2–11 - NCT04173650RecruitingPhase 1/Phase 2
Testing stem cell “package” therapy for severe skin blistering
This early-stage study tests stem cell–derived “extra-cellular vesicles” (small natural cell packages) to help heal painful skin wounds in people with dystrophic epidermolysis bullosa (DEB). If you have suitable, untreated wounds and meet the safety requirements, you may be able to join and help researchers learn whether this treatment is beneficial.
Phoenix, ArizonaAges 6 months+ - NCT06336733RecruitingPhase 3
Trial of anakinra for FMF attacks in patients who refuse daily shots
This trial tests if taking anakinra (a targeted medicine) only when you have a painful FMF attack can help, instead of taking it every day. It is for people with Familial Mediterranean Fever (FMF) whose attacks continue despite taking colchicine, and who do not want daily injections.
ParisAges 6 years+ - NCT06588426Recruiting
Skin punch biopsy collection for heart disease research
This trial collects a small skin sample (like a biopsy) from people with congenital heart disease. The samples help prepare for future clinical trials that may test new heart treatments.
Rochester, MinnesotaAges 18 years+ - NCT06902896RecruitingPhase 1/Phase 2
FAP iCDC therapy for advanced heart failure from dilated cardiomyopathy
This trial tests a new treatment called FAP iCDC for people with end-stage dilated cardiomyopathy, a type of heart muscle disease. It aims to see if this therapy is safe and helps improve heart function when standard medicines are no longer enough.
Hangzhou, ZhejiangAges 18–75 - NCT06904027Recruiting
Glycerol phenylbutyrate for children with urea cycle disorders
This trial tests a medicine called glycerol phenylbutyrate in children with urea cycle disorders, which are genetic conditions that make it hard for the body to get rid of extra ammonia. The goal is to see if the medicine can help lower ammonia levels and manage the condition safely.
Beijing, Beijing MunicipalityAges birth–18 years - NCT06901245RecruitingPhase 4
Tirzepatide for young adults with obesity
This study tests the diabetes/weight-loss drug tirzepatide (Mounjaro) in young adults aged 18–26 who have obesity and either Prader-Willi syndrome, hypothalamic obesity, or general obesity. It looks at whether a weekly injection can help with weight management.
Saint Paul, MinnesotaAges 18–26
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.