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
- NCT06636344Recruiting
Better care for people with pseudoxanthoma elasticum
This study looks at whether a new way of organizing care helps patients with pseudoxanthoma elasticum (PXE). It also asks some patients about their experience with the new care setup.
AngersAges 6 years+ - NCT06638463Recruiting
My Health Coach App Study
This trial tests whether the My Health Coach app can help adults with fetal alcohol spectrum disorder or prenatal alcohol exposure. It is for people who own a smartphone and can read English.
Rochester, New YorkAges 18 years+ - NCT06641011Recruiting
Sealing molars with enamel spots: rinsing vs blotting
This study tests whether rinsing off or blotting dry a special silver fluoride liquid before placing a tooth sealant helps the sealant last longer on molars affected by a common condition called molar-incisor hypomineralization. The goal is to find the best way to protect these teeth from cavities.
AnkaraAges 6–14 - NCT06641154RecruitingPhase 1/Phase 2
Gene therapy for severe Crigler-Najjar syndrome
This trial tests a gene therapy for children with severe Crigler-Najjar syndrome type I. It aims to reduce the need for daily phototherapy by fixing the faulty gene.
MoscowAges 3 months–10 years - NCT06641310Recruiting
Exercise therapy trial for people with familial adenomatous polyposis
This trial tests whether a home-based exercise program can help people with familial adenomatous polyposis (FAP) who have rectal polyps. You may qualify if you have FAP, have not had your rectum removed, have at least 5 rectal polyps larger than 2 mm, and are currently inactive.
Ann Arbor, MichiganAges 18 years+ - NCT06641895RecruitingEarly Phase 1
Testing BBM-D101 gene therapy for Duchenne muscular dystrophy
This early-phase study tests a new gene therapy called BBM-D101 for boys with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and helps improve muscle function in young boys.
Shanghai, Shanghai MunicipalityAges 4–8 - NCT06642610Recruiting
Using CGM to predict prediabetes in cystic fibrosis
This study uses a continuous glucose monitor (CGM), a small sensor worn on the skin, to track blood sugar levels in people with cystic fibrosis (CF) who do not have diabetes or have prediabetes. The goal is to find patterns that might predict who will develop diabetes, so it can be caught earlier.
Indianapolis, IndianaAges 12–55 - NCT06644742Recruiting
PKP2 heart condition natural history study
This study is for people with a specific genetic heart condition (PKP2-related arrhythmogenic cardiomyopathy) who already have an ICD. It does not test a new treatment but instead follows participants over time to learn more about the condition.
Boston, MassachusettsAges 12 years+ - NCT06645899Recruiting
Italian database for parathyroid conditions
This study is creating a database of people with hypoparathyroidism or pseudohypoparathyroidism in Italy. It may help researchers learn more about these conditions and improve future care.
AlessandriaAges Any age - NCT06646250Recruiting
New ultrasound for steady brain monitoring in newborns
This trial uses a new ultrasound device (NeoDoppler) to watch blood flow in a baby's brain continuously. It aims to see if this gentle, bedside tool can help doctors monitor babies—especially those born early or with certain health concerns—without moving them.
TrondheimAges birth–1 year - NCT06646640Recruiting
Long-term follow-up for hemoglobin disorder treatments
This study tracks people with sickle cell disease or thalassemia who have had a bone marrow transplant or gene therapy in the past 15 years. It aims to monitor their health over time to understand long-term effects.
Memphis, TennesseeAges Any age - NCT06647927Recruiting
Genetic study for better Brugada diagnosis
This study looks at the genes of people with Brugada Syndrome to find new markers that can help doctors diagnose it more accurately. If you have Brugada Syndrome, joining could help improve understanding and care for this condition.
San Donato Milanese, MilanAges 18 years+ - NCT06647979RecruitingPhase 1
Gene editing for severe sickle cell or thalassemia
This trial tests a new gene therapy that edits your own blood stem cells to help them make more fetal hemoglobin, which can prevent sickling and reduce the need for transfusions. It is for people with severe sickle cell disease or beta thalassemia who do not have a matched sibling donor.
Boston, MassachusettsAges 13–40 - NCT06648096RecruitingPhase 1/Phase 2
Testing afatinib for head and neck cancer in Fanconi anemia patients
This trial tests whether the targeted drug afatinib can shrink advanced head and neck cancers in people who also have Fanconi anemia. It is for patients whose tumors cannot be surgically removed and have not responded to or cannot have other standard treatments.
HanoverAges 18 years+ - NCT06650319RecruitingEarly Phase 1
Testing LY-M003 injection for Wilson disease
This study tests an injection called LY-M003 for people with Wilson disease who have already been treated with standard medicines. The goal is to see if it is safe and works for them.
Hangzhou, ZhejiangAges 10–60 - NCT06650683Recruiting
Nursing support for parents of babies with Hirschsprung disease
This trial is for parents of babies with Hirschsprung disease who need daily nursing care before surgery. It studies whether extra nursing support can help reduce parents' stress while taking care of their baby at home.
MarseilleAges Any age - NCT06650813Recruiting
Adapting a fatigue program for sickle cell disease
This trial adapts the FACETS program, a fatigue-management course, for people with sickle cell disease. It aims to help participants better cope with fatigue and improve daily life.
GrenobleAges 18 years+ - NCT06651255Recruiting
Algorithm-guided care for bleeding in Von Willebrand disease
This trial tests whether a special algorithm can reduce repeat bleeding from the gut or nose in people with inherited Von Willebrand disease. If you have had serious nosebleeds or digestive bleeding, this study may help find a better way to manage your condition.
Lille, NordAges 18 years+ - NCT06651489RecruitingPhase 2
Guselkumab for Hailey-Hailey disease
This study tests the drug guselkumab to see if it can help control Hailey-Hailey disease, a rare skin condition. If you have moderate-to-severe HHD affecting multiple body areas, you may be able to join.
New Haven, ConnecticutAges 18–90 - NCT06652321Recruiting
New laser method for corneal transplant in Fuchs' dystrophy
This trial tests a new way to perform a corneal transplant (DSAEK) using a laser to prepare the donor tissue. It may help people with Fuchs' dystrophy or certain types of corneal swelling, but not those with glaucoma, scarring, or other eye diseases.
GizaAges Any age - NCT06654752Recruiting
Simpler treatment for lung flare-ups in kids with CF
This study tests a streamlined approach to managing sudden lung symptoms (pulmonary exacerbations) in children with cystic fibrosis. It aims to see if a simpler treatment plan works as well as standard care, which could mean less time in the hospital and fewer disruptions to daily life.
Birmingham, AlabamaAges 3–18 - NCT06655662RecruitingPhase 1
Gene therapy for severe beta-thalassemia
This trial tests a one-time gene therapy using your own blood stem cells to treat severe beta-thalassemia. It may help you stop needing regular blood transfusions.
GuangxiAges 6–35 - NCT06657911Recruiting
Studying a new sizing method for bicuspid aortic valves
This trial looks back at people with a specific type of bicuspid aortic valve (a valve with two flaps instead of three) who had a transcatheter aortic valve replacement. Researchers want to see how well a special sizing method called LIRA worked for choosing the right valve size.
Brescia, ItalyAges 18 years+ - NCT06658496Recruiting
Studying kidney health in very premature newborns
This trial looks at how well kidneys work in very premature babies (born before 32 weeks). It uses special sensors to monitor kidney health in the first few days of life.
Madison, WisconsinAges 1 minute–1 week
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.