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
- NCT06816953Enrolling by invitation
Joint health in severe hemophilia A with prophylaxis
This study is looking at how well joint health can be tracked in people with severe hemophilia A who are receiving regular factor VIII treatment. It uses special markers in the blood and ultrasound to check for early joint changes.
Bangkok, PatumwanAges Any age - NCT06817590RecruitingPhase 1
Nucleoside therapy for telomere disorders
This trial tests a nucleoside therapy (dC/dT) for people with telomere biology disorders, which cause premature aging and problems like low blood counts, lung or liver issues. The goal is to see if the treatment can help manage these symptoms.
Boston, MassachusettsAges 1–70 - NCT06816095Recruiting
Ozone therapy for pelvic pain after gynecological cancer treatment
This study is looking at whether ozone therapy can help relieve chronic pelvic pain caused by gynecological cancer treatments like radiation and chemotherapy. It aims to understand how the treatment works at a molecular level.
Las Palmas, Las PalmasAges 18 years+ - NCT06822400Recruiting
Study of Tetralogy of Fallot in Newborns
This trial looks at newborns with Tetralogy of Fallot, a heart condition present at birth. It aims to study the best way to treat these babies in the first month of life.
Beijing, Beijing MunicipalityAges 1 minute–6 months - NCT06818760Recruiting
Remote monitoring for pregnant women with heart conditions
This study tests if a wrist wearable can safely monitor pregnant women with congenital heart disease. It aims to improve care during pregnancy, delivery, and after birth by tracking health data remotely.
Cleveland, OhioAges 18 years+ - NCT06818266RecruitingPhase 3
Tocilizumab for severe chest crisis in sickle cell disease
This trial tests if the drug tocilizumab can help treat acute chest syndrome, a serious lung complication of sickle cell disease. It is for people with sickle cell disease who are hospitalized and need oxygen or breathing support. The study compares tocilizumab to a placebo to see if it speeds up recovery.
ParisAges 2 years+ - NCT06657846Recruiting
Quality of life and costs for people with VEXAS
This study looks at how VEXAS syndrome affects your daily life and finances. It aims to understand the challenges you face so doctors can better support you.
AnconaAges 18–99 - NCT06554275Recruiting
CCHS research health hub
This study creates a secure health information hub for people with Congenital Central Hypoventilation Syndrome (CCHS) to help researchers better understand the condition and find new ways to treat it. Being part of this hub may help advance future studies and care options for CCHS.
Chicago, IllinoisAges Any age - NCT06808880Recruiting
Expanding prenatal screening for single-gene disorders
This study looks at a new blood test to screen for single-gene disorders (conditions caused by changes in one gene) during pregnancy. It may help if you or your baby's father carry a genetic disorder, or if an ultrasound shows signs of a possible genetic condition.
Glendale, ArizonaAges 18 years+ - NCT06807099Recruiting
Stent graft for dialysis access problems
This trial tests a special stent graft called WRAPSODY CIE to treat narrowed or blocked veins in the arm used for dialysis. It aims to keep your access working well so you can continue receiving hemodialysis.
Dothan, AlabamaAges 18 years+ - NCT06805695Recruiting
Long-term follow-up for iECURE trial participants
This study follows up with people who took part in an earlier iECURE trial (such as a gene therapy study) to check on their health over time. It collects information to see how safe and lasting the treatment effects are.
Aurora, ColoradoAges 7 months–1.3 years - NCT06807281RecruitingPhase 3
Abrocitinib for children with eczema: long-term study
This study tests the long-term safety and effectiveness of abrocitinib (Cibinqo) for children aged 6 to 12 with moderate to severe eczema that hasn't improved with creams. It may help their skin clear and reduce itching.
Birmingham, AlabamaAges 2–11 - NCT06805825RecruitingPhase 1
Study of NN3201 for advanced cancers with c-Kit
This trial tests a new drug, NN3201, that targets a protein called c-Kit found on some cancer cells. It may be an option for people with certain advanced solid tumors, including GIST, small cell lung cancer, or other c-Kit-positive cancers, who have already tried standard treatments or cannot take them.
Ann Arbor, MichiganAges 18 years+ - NCT06808477RecruitingPhase 1
BBT001 for moderate to severe eczema
This trial tests an experimental drug called BBT001 for people with moderate to severe eczema (atopic dermatitis) that hasn't been helped enough by creams. The study is in early phases, so it will check how safe the drug is and how well it might work.
Irvine, CaliforniaAges 18–72 - NCT06809972Recruiting
Understanding joint lining changes on ultrasound in hemophilia
This study uses ultrasound to check if thickened joint lining (synovium) in people with severe hemophilia is a sign of active inflammation or just a harmless leftover. It may help doctors decide whether to treat or watch these joints.
UtrechtAges 12 years+ - NCT06812507Recruiting
Umbilical cord blood transfer in very premature babies
This trial tests different ways of transferring blood from the umbilical cord to very premature babies (born at or before 32 weeks) to see if it can prevent complications of prematurity and improve their blood counts. It aims to find the best method for helping these vulnerable newborns.
AlexandriaAges 1 minute–2 minutes - NCT06805734Recruiting
Tracking health in children with PTEN gene changes
This study follows children and their affected relatives who have a harmful change in the PTEN gene. The goal is to understand the health risks, especially the chance of developing cancer, over time.
MilanAges Up to 17 years - NCT06813625Enrolling by invitation
Shared reading for mental well-being in young people
This trial tests whether reading books together can improve mental well-being in young people with and without Down syndrome. It involves children with Down syndrome and college students from the University of Macau reading together.
MacauAges 2–35 - NCT06800599Enrolling by invitation
Cancer genetic risk assessment registry
This study collects information from people who receive genetic counseling for cancer risk. Joining the registry helps researchers learn more about cancer genetics and improve future care.
Bologna, ItalyAges Any age - NCT06801535Recruiting
3D-printed splint for burn scars on mouth
This trial tests a custom 3D-printed mouth splint to help people who have a tight, small mouth opening after a burn. It aims to see if the splint can improve how wide you can open your mouth.
Winston-Salem, North CarolinaAges 2–99 - NCT06797206Recruiting
CF in pregnancy and child health study
This study follows women with cystic fibrosis (CF) during pregnancy and after birth, and checks on their babies and young children. The goal is to learn more about how CF affects pregnancy and child development.
LondonAges Any age - NCT06801821Recruiting
Stigma study on skin conditions
This study looks at how people view those with certain skin conditions. It aims to understand stigma, which may help improve awareness and support.
Roma, ItaliaAges 18 years+ - NCT06795152Recruiting
Rare GSD Natural History Study
This study follows people with rare types of glycogen storage disease (GSD) over time to learn more about how the disease progresses. It does not test a new treatment but will collect information to help future research.
Durham, North CarolinaAges birth–90 years - NCT06794489Recruiting
Tracking CMT with biomarkers over time
This study is looking for people with Charcot-Marie-Tooth disease (a nerve condition) and healthy volunteers to track long-term changes in the body. Researchers want to see if certain biomarkers (measurable signs) can help predict how CMT progresses over time.
Göttingen, Lower SaxonyAges 18–65
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.