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 2284
- Phase 1235
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome55
- 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
- California341
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona78
- Texas72
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT06439654Recruiting
Optimizing cholesterol treatment for heart health
This trial helps people with high cholesterol or a history of heart disease find the best treatment to lower their risk of future heart problems. It tests a program to optimize lipid-lowering therapy.
Clark, New JerseyAges 18–120 - NCT06442592Recruiting
Understanding development in kids with heart defects
This study looks at how children with complex congenital heart defects develop, especially their brain and learning skills. It will help doctors provide better support for these children.
Brest, Brittany RegionAges 3–11 - NCT06442761RecruitingPhase 1
Collecting stem cells for gene therapy in sickle cell disease
This study tests a new drug called motixafortide to help collect your own blood stem cells for future gene therapy. It is for adults with severe sickle cell disease who are willing to donate their stem cells and meet certain health requirements.
Birmingham, AlabamaAges 18 years+ - NCT06445322Recruiting
Finding people with Stargardt disease for ACDN-01
This prescreening study looks for people with a specific genetic eye condition called Stargardt disease to see if they might be able to join a future trial for a new treatment called ACDN-01.
San Francisco, CaliforniaAges 5 years+ - NCT06447025RecruitingPhase 2
Study of CTI-1601 for people with Friedreich's ataxia
This trial tests an injectable medicine called CTI-1601 for Friedreich's ataxia (FRDA). It is an open-label study, meaning everyone knows they are getting the drug. The study is for people who have taken CTI-1601 before (without serious side effects) or are new to the drug.
Los Angeles, CaliforniaAges 2–60 - NCT06447961Recruiting
Psychological impacts of living with inherited colorectal cancer risk
This study explores how people feel emotionally and mentally when they have a genetic condition that raises their risk of colorectal cancer. It aims to understand the psychological effects, which could help improve support for others in the future.
LondonAges 18–99 - NCT06448546Recruiting
Gut bacteria in Huntington's disease
This study looks at how gut bacteria are different in people with Huntington's disease who have unusual weight changes. You'll provide stool and blood samples to help researchers understand gut health in HD.
Orlando, FloridaAges 18 years+ - NCT06448689Recruiting
Wrist monitor study for itch in eczema and psoriasis
This study tests whether a wrist-worn device can accurately measure scratching during sleep in people with eczema (atopic dermatitis) or psoriasis. It may help doctors better understand and treat nighttime itching.
ParisAges 14–70 - NCT06451575Recruiting
Blood Clots and Behçet's Disease Study
This study looks at how Behçet's disease affects blood clotting. If you have Behçet's disease and are between 18–70, you may help doctors understand why some people with Behçet's develop dangerous blood clots.
Erzurum, Üniversite MahallesiAges 18–70 - NCT06452199Recruiting
Bifidobacterium infantis for newborns study (BEGIN)
This study gives healthy newborns a probiotic supplement called Bifidobacterium infantis to see if it helps with growth, immune health, and reducing inflammation. It may be a safe way to support your baby's early development.
Herning, Central JutlandAges 1 day–1 week - NCT06453512Recruiting
Testing Doublebase Once Moisturizer for Atopic Eczema
This study tests a moisturizer called Doublebase Once for people with atopic eczema. You apply it once a day for 4 weeks to see how well it improves skin moisture and eczema symptoms.
Nottingham, NottinghamshireAges Any age - NCT06454942Recruiting
Eggs and eczema: a diet study for mild to moderate cases
This trial tests if eating regular eggs or nutrient-fortified eggs can improve eczema in people with mild to moderate symptoms. It looks for healthy adults aged 21-59 with a BMI in the normal range.
SingaporeAges 21–59 - NCT06456827Recruiting
Bite force and bone changes after braces in different face types
This trial compares how bite force and bone turnover markers change during the retention phase (after braces) in people with high-angle and low-angle face types. It may help understand why some people have more relapse after braces.
Rohtak, HaryanaAges 18–45 - NCT06457451Recruiting
Diet and exercise program for cystic fibrosis patients on Trikafta
This study tests whether a program combining diet advice and guided exercise can help people with cystic fibrosis who are already on Trikafta improve their lean muscle mass. Participants will be randomly assigned to receive the program or continue their usual care.
AngersAges 18 years+ - NCT06458010RecruitingEarly Phase 1
Gene therapy trial for inherited high cholesterol
This trial tests a new gene therapy called YOLT-101 for people with familial hypercholesterolemia, a genetic condition that causes very high cholesterol. The treatment aims to lower cholesterol levels and reduce heart risks.
Shanghai, Shanghai MunicipalityAges 18–75 - NCT06458374Recruiting
Heart Amyloidosis in Carpal Tunnel Surgery Patients
This study looks for people who had carpal tunnel surgery at Ålesund Hospital to check for a rare heart condition called ATTR amyloidosis. It aims to find the disease early and see how common it is.
Ålesund, Møre og RomsdalAges 50 years+ - NCT06458478Recruiting
Gel to help healing after wisdom tooth removal
This trial tests a special gel applied after removing lower wisdom teeth to see if it helps healing. It is for healthy people who need both bottom wisdom teeth taken out.
PerugiaAges 18–35 - NCT06462430Recruiting
Registry for children with PTEN Hamartoma Tumor Syndrome
This study is creating a registry (a database) of children with PTEN Hamartoma Tumor Syndrome, a condition that can cause growths, large head size, and other health issues. By joining, you help doctors learn more about the condition and how to better care for kids like yours.
IstanbulAges 1 year+ - NCT06463028RecruitingPhase 2
Targeted pills plus chemo for advanced endometrial cancer
This trial tests a combination of two targeted pills (sapanisertib and serabelisib) with standard chemo (paclitaxel) for endometrial cancer that has come back or spread. A substudy also looks at combining these drugs with a special diet that lowers insulin levels.
La Jolla, CaliforniaAges 18 years+ - NCT06464458Recruiting
Better hydroxyurea dosing for sickle cell patients
This trial checks whether monitoring hydroxyurea drug levels in the blood can help doctors find the best dose for sickle cell patients. It focuses on people with HbSS who have recently had a pain crisis or whose current dose isn't working.
StrasbourgAges 2–35 - NCT06464588RecruitingPhase 1
Testing safety of newborn stem cells for dilated cardiomyopathy in young adults and children
This study tests whether a single intravenous infusion of stem cells from donated newborn tissue is safe for people aged 4 to 39 with dilated cardiomyopathy (a weakened, enlarged heart). It aims to find a new option for those who haven't improved enough with standard heart failure medications.
Atlanta, GeorgiaAges 4–40 - NCT06464757Recruiting
Laryngomalacia and breathing in children: before and after treatment study
This trial is for children with laryngomalacia (a floppy voice box) who have noisy breathing and trouble breathing. It will check how they are doing before and after treatment, and follow up for one year to see how their quality of life improves.
OsloAges 1 week–12 months - NCT06465823RecruitingPhase 2
Bumetanide for thinking skills in Down syndrome
This trial tests whether a medication called bumetanide can improve thinking and learning skills in teenagers with Down syndrome. It is a 10-week study that compares bumetanide to a placebo (a pill with no medicine).
RomeAges 10–17 - NCT06466291Recruiting
Spanish Wilson Disease Registry
This study is a registry that collects information about people with Wilson disease. By joining, you can help researchers learn more about the condition and improve care for others.
BarcelonaAges 18 years+
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 5,764 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.