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 Disease84
- 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
- California325
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York85
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT05997680Recruiting
Yoga for attention in kids with heart disease
This study tests if a parent-child yoga program can help improve attention in children aged 4 to 6 with congenital heart disease who have attention difficulties. You and your child would attend yoga sessions together for 8 weeks.
Montreal, QuebecAges 4–6 - NCT06709690Recruiting
AI-powered PET/MR scanner for children with suspected cancer
This study tests a new, fast, and quiet AI-powered PET/MR scanner to take detailed pictures of children with suspected cancer. It aims to make the imaging experience easier and more comfortable for kids.
Shanghai, Shanghai MunicipalityAges Up to 18 years - NCT07609108RecruitingPhase 3
Pridopidine to slow Huntington's disease progression
This study tests whether a medication called pridopidine can help slow the progression of Huntington's disease symptoms in adults. Huntington's is a genetic disorder affecting movement, mood, and thinking, and this trial aims to see if pridopidine improves daily functioning and quality of life.
Birmingham, AlabamaAges 23–65 - NCT07604324RecruitingPhase 1
New Drug for Moderate to Severe Eczema Treatment
This study tests a new investigational drug called DCY636 to see if it can help people with moderate to severe atopic dermatitis (eczema) that hasn't improved with topical creams. The trial starts with safety testing in healthy volunteers, then moves to people with active eczema.
FukuokaAges 18–55 - NCT07606274RecruitingPhase 1/Phase 2
Study of NVC-001 for heart muscle disease caused by LMNA gene change
This trial tests an experimental drug called NVC-001 for people with a specific genetic heart condition (LMNA-related dilated cardiomyopathy) that causes the heart to become weak and enlarged. The goal is to see if the drug can help improve heart function.
Birmingham, AlabamaAges 18 years+ - NCT07608432RecruitingPhase 3
Testing a New DMD Treatment Given Every 4 Weeks
This study tests whether a new medicine called zeleciment rostudirsen can help ambulatory (walking) males with Duchenne muscular dystrophy (DMD). The medicine is given as an intravenous infusion (through a vein) every 4 weeks and works by helping the body produce a protective muscle protein.
Hillsborough, North CarolinaAges 4–18 - NCT07610590Recruiting
Understanding Genetic Test Results During Pregnancy
This research study helps pregnant people understand the results of genetic tests done during pregnancy (like CVS or amniocentesis). The study follows families for one year after birth to learn how genetic counseling and test results affect decision-making and family wellbeing.
Boston, MassachusettsAges 18 years+ - NCT07607730Recruiting
Group therapy for emotion control in teens with Prader-Willi syndrome
This study tests a group therapy program designed to help teenagers with Prader-Willi syndrome (a genetic condition) learn better emotional control and regulation skills. Your teen and family would attend weekly group sessions to practice tools for managing emotions like anger or frustration.
Kansas City, MissouriAges 13–17 - NCT07610811Recruiting
Foot Reflexology and Massage for Jaundiced Newborns
This study tests whether gentle foot reflexology and massage can help newborns with jaundice (high bilirubin levels in the blood) recover faster during light therapy treatment. Jaundice is common in newborns and is usually treated with special lights; this trial explores whether touch therapy might improve outcomes.
Kusadasi, AydınAges 2 days–1 week - NCT07609394Recruiting
Duchenne and Becker Muscular Dystrophy Health Records Study
This study collects electronic health records (medical information already in your doctor's computer system) from people with Duchenne or Becker muscular dystrophy, or female carriers of these conditions. Researchers will use this data to better understand the diseases and improve care over time.
Little Rock, ArkansasAges Any age - NCT07610239Recruiting
Newborn Sickle Cell Disease Screening System in Uganda
This study tests a new computer system to help hospitals quickly identify babies with sickle cell disease (an inherited blood disorder) right after birth or at early health visits. Finding sickle cell disease early helps prevent serious health problems and saves lives.
JinjaAges 18 years+ - NCT07606989Recruiting
Genetic testing to understand fetal birth defects
This study uses advanced genetic testing (whole genome sequencing) on tissue samples from fetuses with structural abnormalities detected on ultrasound. The goal is to identify genetic causes of birth defects to help with diagnosis and understanding of your baby's condition.
Hangzhou, ZhejiangAges 18 years+ - NCT06518005RecruitingPhase 2
Gene therapy for severe Crigler-Najjar syndrome
This trial tests a new gene therapy called GNT0003, given after a pre-treatment to help it work, for people with severe Crigler-Najjar syndrome. The goal is to see if it can reduce the need for daily light treatments.
ClamartAges 18 years+ - NCT05942716RecruitingPhase 2
Testing how serotonin changes impulsivity in Tourette
This Phase 2 study looks at whether changing serotonin activity can affect impulsivity and related brain function in people with Tourette disorder. You may be asked to continue aripiprazole and complete brain scans that test how your brain responds during tasks.
BronAges 18–65 - NCT06392906RecruitingPhase 3
Radiation for Graves' Eye Disease: 1 vs 2 Weeks
This trial tests if a shorter course of radiation (1 week) works as well as a standard longer course (2 weeks) for treating active thyroid eye disease that did not improve enough with steroid medicine. It may be an option if you still have eye symptoms after steroid treatment.
Bangkok NoiAges 18–70 - NCT07599176RecruitingPhase 1/Phase 2
Partial stem cell transplant for sickle cell disease from matched donors
This study tests a partial stem cell transplant from a matched donor to treat sickle cell disease. It may help reduce complications like strokes, organ damage, or pain crises.
Bethesda, MarylandAges 4–65 - NCT06694012Recruiting
Ohtori Study Part 2 heart health
This study looks at heart and metabolic health in the general population. It helps researchers understand how to prevent conditions like diabetes and heart disease.
Sakai, OsakaAges 20 years+ - NCT07598383RecruitingPhase 2
Gel treatment for mild-to-moderate atopic dermatitis
This study tests a new gel called VC005 to see if it safely reduces itching, redness, and irritation in people with atopic dermatitis (a chronic skin condition). You'll be randomly assigned to either the new gel or a placebo (inactive gel) and apply it to affected areas over several weeks.
Hangzhou, ZhejiangAges 18 years+ - NCT07597200RecruitingPhase 1
Cizutamig treatment for active thyroid eye disease
This study tests a new drug called cizutamig for thyroid eye disease (TED), a condition where the eye muscles and tissues swell due to Graves' disease. The drug is designed to reduce inflammation and may help improve symptoms like eye bulging, double vision, and discomfort.
Hefei, AnhuiAges 18–75 - NCT07601425RecruitingPhase 1/Phase 2
ATV-1601 Treatment for Hereditary Hemorrhagic Telangiectasia
This trial tests a new drug called ATV-1601 in people with HHT, a genetic condition that causes abnormal bleeding and blood vessel problems. The drug aims to reduce bleeding and anemia caused by HHT.
Phoenix, ArizonaAges 18 years+ - NCT07599813RecruitingPhase 2
Testing camoteskimab for moderate-to-severe eczema
This study tests a new medication called camoteskimab for adults with moderate-to-severe atopic dermatitis (eczema) that hasn't responded well to creams alone. The drug works by targeting a protein in the body that causes eczema symptoms like itching and inflammation.
Birmingham, AlabamaAges 18–65 - NCT07603466Enrolling by invitationPhase 4
Two drugs combined to treat Cushing's disease
This trial tests whether taking two medications together—osilodrostat and cabergoline—can help control Cushing's disease by lowering excess cortisol levels. Cushing's disease is caused by a pituitary tumor that makes too much of a hormone called ACTH, which drives cortisol overproduction.
BasraAges 18–70 - NCT07598643RecruitingPhase 2
Study of tofacitinib for immune function in Down syndrome
This study tests if an oral medication called tofacitinib can help the immune system work better in children and young adults with Down syndrome, which may improve learning and development. The study will follow participants for 6 months.
Aurora, ColoradoAges 6–22 - NCT07603232Enrolling by invitation
Long-Term Health Outcomes in Children With Hirschsprung's Disease or Anorectal Malformations
This study follows children who were born with Hirschsprung's disease (a condition affecting the nerves in the colon) or anorectal malformations (differences in how the rectum or anus formed) to understand how they do over time. Researchers will track health outcomes and quality of life to help improve care for future children with these conditions.
Montreal, QuebecAges Up to 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 5,456 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.