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 2283
- Phase 1235
- Phase 3216
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome54
- 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
- California340
- Maryland161
- Alabama132
- Massachusetts116
- New York84
- Arizona78
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT05548283RecruitingPhase 4
Standard IV antibiotic timing for cystic fibrosis lung flare-ups
This study standardizes how IV aminoglycoside antibiotics are given during a 14-day treatment for cystic fibrosis (CF) lung flare-ups. It may help make treatment more consistent and easier to manage for people who need IV antibiotics.
Birmingham, AlabamaAges 6 years+ - NCT05181748RecruitingPhase 2/Phase 3
Platelet-rich plasma into ovaries for low egg response
This trial tests whether injecting your own platelet-rich plasma (PRP) into the ovaries can improve egg production in people who don’t respond well to fertility stimulation. It may help some “poor responders” get more usable eggs for IVF.
Athens, AtticaAges 35–47 - NCT05670132Recruiting
Heart and brain rehab for youth with congenital heart disease
This trial tests a combined heart and brain rehabilitation program for young people with moderate to complex congenital heart disease. The goal is to improve both heart health and thinking skills, such as memory and attention.
BrusselsAges 8–25 - NCT06971731RecruitingPhase 3
JNT-517 pill for adults with PKU
This trial tests a new pill called JNT-517 to see if it can lower high blood phenylalanine (Phe) levels in people with PKU. You may be a good fit if you have PKU, are at least 18, and can stick to a steady diet.
Los Angeles, CaliforniaAges 18 years+ - NCT07101640RecruitingPhase 1/Phase 2
Montelukast study for breathing problems in premature infants
This trial tests if the medicine montelukast can help very premature babies on breathing machines. It aims to see if it improves lung development and makes it easier for them to breathe on their own.
Little Rock, ArkansasAges 1 week–4 weeks - NCT03121001RecruitingPhase 2
Transplant from a half-matched donor for aggressive sickle cell
This Phase 2 study tests a “half-matched” stem cell transplant to treat people with aggressive sickle cell disease. It may help by replacing the blood system using stem cells from a related donor who is close but not fully matched.
Chicago, IllinoisAges 16–60 - NCT07690930Enrolling by invitation
Hat glasses for eye protection during jaundice light therapy
This trial tests a special hat-like glasses to protect babies' eyes during phototherapy (light treatment for jaundice). It is for healthy full-term newborns who are in a regular hospital room.
KaramanAges birth–4 weeks - NCT07109375Recruiting
Long-term study of pegunigalsidase alfa for Fabry disease
This study observes how well the medication pegunigalsidase alfa works over a long period in people with Fabry disease. It helps doctors learn more about managing the condition in everyday life.
ArezzoAges 18 years+ - NCT06602869Recruiting
Cellular markers in cystic fibrosis with a certain lung infection
This study looks at special blood markers in people with cystic fibrosis who have or haven't been treated for a lung infection caused by nontuberculous mycobacteria. The goal is to better understand the body's response, which could lead to better treatments.
MontpellierAges 18 years+ - NCT06603194Recruiting
Collagen for skin, hair, and nails in healthy women
This study tests whether collagen from cows or fish can improve skin, hair, nails, and overall body health. It compares collagen to a placebo to see if it makes a real difference for healthy adults.
Chelmsford, EssexAges 35–55 - NCT06603831Recruiting
3D digital surgery study for bone and joint procedures
This study tests whether 3D digital planning tools make surgeries for certain bone and joint problems more effective and cost-efficient. If you need a specific type of surgery on your forearm, pelvis, or spine, this trial could help determine if digital surgery improves your recovery.
Sabadell, BarcelonaAges 18–80 - NCT06607471Recruiting
Study on non-ischemic heart muscle diseases
This study looks at different types of heart muscle diseases that are not caused by blocked arteries (non-ischemic cardiomyopathies). It aims to better understand how to diagnose, predict outcomes, and treat these conditions by gathering information from many patients over time.
Milan, MilanoAges 18 years+ - NCT06609694Recruiting
Comparing oxygen delivery methods for children with sleep apnea
This study tests a new way to give oxygen to children aged 5-17 who have obstructive sleep apnea (a condition where breathing stops briefly during sleep). It aims to see if a demand oxygen delivery system works better than standard care. This could help children with Down syndrome who often have untreated sleep apnea.
Cincinnati, OhioAges 5–17 - NCT06609889RecruitingPhase 1/Phase 2
Testing a new drug for Lafora disease in young people
This trial tests an experimental drug called ION283, given by a lumbar puncture (spinal tap), to see if it is safe and can help with Lafora disease symptoms. It is for patients aged 10 to 18 with a confirmed genetic diagnosis.
Dallas, TexasAges 10–18 - NCT06610019Recruiting
Heart imaging study using MRI
This study uses heart MRI scans to learn more about cardiovascular health in adults. If you are 18 or older and have been referred for a heart MRI at Montefiore, you may be able to join.
The Bronx, New YorkAges 18 years+ - NCT06610201Recruiting
Study of a new treatment for Von Willebrand disease bleeding
This study is testing a new treatment for people with certain types of Von Willebrand disease (Type 1 or 2A) who have frequent bleeding problems. It aims to see if the treatment can help reduce these bleeding episodes.
Phoenix, ArizonaAges 16 years+ - NCT06610864Recruiting
Corrective surgery for womb shape issues and pregnancy outcomes
This study looks at how women with certain womb shape problems (like a septum or unusual shape) do during and after pregnancy once they have a minor surgery to fix the shape. Researchers want to see if the surgery helps improve pregnancy and birth results.
Rome, RMAges 18 years+ - NCT06611787Recruiting
Registry study for congenital heart disease
This study collects information from people with congenital heart disease (a heart problem you're born with) over their entire life to better understand the condition and improve future care.
Beijing, Beijing MunicipalityAges Any age - NCT06613126Recruiting
App to help parents manage Tourette symptoms in children
This trial tests a smartphone app that helps parents manage their child's Tourette Syndrome symptoms. It may improve how parents care for their child and reduce stress.
Taoyuan CityAges 20–60 - NCT06614738Recruiting
Microbes and environment in polyps after FAP surgery
This study looks at how bacteria and your environment might affect polyp growth in people with familial adenomatous polyposis (FAP) after colon removal. By collecting stool samples and medical information, researchers hope to find ways to reduce polyp risk.
Lyon, FranceAges 18 years+ - NCT06614777Recruiting
Darier's disease immune and microbiome study
This study looks at immune markers and gut/skin bacteria in people with Darier's disease. It aims to better understand the condition, which could lead to better treatments in the future.
Toulouse, Centre Hospitalier Universitaire de ToulouseAges 18 years+ - NCT06614894RecruitingPhase 2/Phase 3
High dose ambroxol for adults with Sanfilippo syndrome
This trial tests a high dose of a medicine called ambroxol in adults with MPS III (Sanfilippo syndrome). It aims to see if the drug is safe and can help symptoms.
Fairfax, VirginiaAges 18–99 - NCT06616545Recruiting
French study on type 3 Glycogen storage disease
This study tracks the health of people with type 3 Glycogen storage disease. It may help doctors understand the disease better and plan better care.
ClamartAges Any age - NCT06616857Recruiting
Smartphone app to boost activity in teens with CF
This study tests a personalized smartphone app to help teens and young adults with cystic fibrosis be more active. The app provides tailored tips and support to encourage daily physical activity.
Jacksonville, FloridaAges 13–25
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 5,779 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.