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
- NCT06705816Recruiting
Cognitive function in Rett syndrome during trofinetide treatment
This study looks at how trofinetide (a medicine for Rett syndrome) affects thinking and memory in people with Rett syndrome. It will follow participants who are starting this treatment through a specific clinic.
Minneapolis, MinnesotaAges 18–99 - NCT06007547RecruitingPhase 4
Preventive surfactant therapy for preterm babies
This trial tests whether giving surfactant (a foam that helps lungs stay open) before breathing problems start can help very premature babies (born before 30 weeks) breathe better. It might reduce the need for a breathing tube.
Evanston, IllinoisAges Up to 15 minutes - NCT03266653RecruitingPhase 2
T-cell therapy for stubborn Epstein-Barr virus after transplants
This trial tests an immune therapy made from EBV-targeted T-cells to treat EBV infections that do not improve with standard anti-viral medicines. It may help by strengthening the body’s ability to fight EBV after certain transplants or immune problems.
Los Angeles, CaliforniaAges 1 month–79 years - NCT03266640RecruitingPhase 2
CMV cell therapy for difficult-to-treat viral infection
This trial tests an immune cell treatment (CMV-specific cytotoxic T cells) for people with a CMV infection that is not improving despite standard antivirals, or who cannot tolerate them. If eligible, the treatment aims to help the body control the virus after transplant-related immune problems.
Los Angeles, CaliforniaAges 1 month–79 years - NCT03266627RecruitingPhase 2
Adenovirus T-cell therapy for hard-to-treat virus after transplants
This trial tests a special immune-cell treatment made to fight adenovirus in people whose adenovirus infection keeps coming back or won’t improve after transplant. It may help control infection when standard antiviral medicines are not working or can’t be tolerated.
Los Angeles, CaliforniaAges 1 month–79 years - NCT04099966RecruitingPhase 2
Stem cell transplant to treat high-risk blood cancers
This trial tests a specialized donor stem cell transplant to treat high-risk problems in the blood and bone marrow, including some cancers and non-cancers. It may help by using donor immune cells designed to better control the disease after transplant.
Valhalla, New YorkAges 1 day–30 years - NCT06702241RecruitingPhase 2/Phase 3
Light therapy for newborn jaundice
This trial tests different ways to give light therapy to newborns with jaundice. It compares different approaches to see which is best, and may help parents understand the most effective treatment for their baby.
AalborgAges 1 day–2 weeks - NCT06700096RecruitingPhase 3
Testing a new drug for adults with severe hemophilia B
This trial tests a single dose of ANB-002, an experimental therapy for hemophilia B. It aims to see if the drug is safe and effective for people with severe hemophilia B who have been treated with factor IX before and do not have inhibitors.
ChelyabinskAges 18 years+ - NCT06699849RecruitingPhase 2
CSL889 for sickle cell pain crisis
This trial tests a new medicine, CSL889, for people with sickle cell disease who are currently having a pain crisis (vaso-occlusive crisis). The goal is to see if it safely helps reduce the pain and speed recovery.
Oakland, CaliforniaAges 12 years+ - NCT06699056Recruiting
AI heart monitor study for ejection fraction
This study uses an AI-powered wearable monitor to check how well your heart pumps blood (ejection fraction) from a simple EKG recording. It may help doctors quickly assess heart function without extra tests.
Orange, CaliforniaAges 18 years+ - NCT06693817Recruiting
Wireless sensors for newborns in the delivery room
This trial tests advanced wireless sensors to monitor babies born at 35 weeks or later who are stable at delivery. The goal is to see if these sensors can improve care right after birth.
Londrina, ParanáAges 8 months–10 months - NCT06701084Recruiting
Genetic Causes of Infant Epilepsy at Boston Children's
This study looks at the genetic causes of epilepsy that starts in babies under 1 year old. It aims to learn how a genetic diagnosis might help guide care.
Boston, MassachusettsAges Any age - NCT06693856Enrolling by invitation
Steroid pulse therapy for active Graves' eye disease
This study tests whether high-dose steroid pulses (given through a vein) can help people with moderate to severe Graves' eye disease that is still active. It is for people who have not yet been treated with steroids and whose eye problems began within the past year.
Shanghai, Shanghai MunicipalityAges 25–70 - NCT06703736Recruiting
Understanding leg function in multiple osteochondromas
This study aims to understand how multiple osteochondromas (bony growths that can form near joints) affect walking and leg function. It compares people with the condition to healthy volunteers, using motion analysis and imaging.
BolognaAges 6–40 - NCT06693674RecruitingPhase 3
Sacubitril-valsartan for heart health in congenital heart disease
This trial tests if a drug called sacubitril-valsartan can improve heart structure and function in adults with congenital heart disease. You will take the drug and have heart imaging to see changes.
Rochester, MinnesotaAges 18 years+ - NCT05135975RecruitingPhase 2
Cabozantinib maintenance to help stop pediatric cancer from growing
This trial studies cabozantinib given after standard treatment to try to keep high-risk pediatric solid tumors from coming back or getting worse. It is offered to children and young adults whose cancer has not fully disappeared but is not progressing at the start of this maintenance phase.
Birmingham, AlabamaAges 1.5 years–40 years - NCT06692049Recruiting
Freezing gonadal tissue to preserve fertility in children with a disorder of sex development
This study tests a procedure that freezes and stores gonadal tissue (tissue that makes eggs or sperm) to help preserve the chance of having biological children in the future. It is for children and young adults under 30 who have a disorder of sex development and either have a risk of cancer in that tissue or will have it removed for another medical reason.
Chicago, IllinoisAges Up to 30 years - NCT06683560Recruiting
3D-printed aligners for cleft lip and palate repair
This study tests whether custom 3D-printed aligners can help shape a baby's cleft lip, gum, and palate before surgery. It aims to make traditional molding treatment more precise and easier for families.
Cairo, AbbasyaAges Up to 1 month - NCT06691867Recruiting
Integrative training for sickle cell pain in teens
This study tests an integrative training program designed to help adolescents with sickle cell disease manage their pain better. It combines physical and mental techniques to reduce pain and improve quality of life.
Hartford, ConnecticutAges 12–18 - NCT06689410Recruiting
Brain aneurysm research registry and biobank
This study is collecting medical information and samples from people with brain aneurysms to learn more about the condition. If you have a recently found aneurysm, you may be able to help researchers understand how aneurysms form and how to better treat them in the future.
Nantes, Loire-AtlantiqueAges 18 years+ - NCT06690151Recruiting
Babies with heart defects and development support
This study looks at how a serious heart condition before birth may affect a baby's brain development. It follows babies and their parents to better understand and support these children as they grow.
Nantes, Loire AtlantiqueAges Any age - NCT06682208Recruiting
Pain relief catheter vs freeze ablation for pectus surgery recovery
This trial compares two ways to manage pain after the Nuss procedure for pectus excavatum. One uses a special catheter that numbs nerves, the other freezes them. The goal is to see which works better for teens and young adults.
Cincinnati, OhioAges 12–21 - NCT06689800Recruiting
Family communication tool for kids getting transplant or cell therapy
This study looks at a family communication tool for children and teens getting stem cell transplants or cellular therapy. It helps families talk about treatment together.
St Louis, MissouriAges 8 years+ - NCT06692426RecruitingPhase 1
Cell therapy for Duchenne muscular dystrophy
This trial tests a new cell-based treatment for Duchenne muscular dystrophy in adults who can no longer walk. The goal is to see if the therapy can help improve muscle function.
Minneapolis, MinnesotaAges 18 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.