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
- NCT07241169RecruitingEarly Phase 1
Gene therapy trial for ABCA4-related retinal disease
This trial tests a new gene therapy called ZVS106e for people with inherited retinal degeneration caused by changes in both copies of the ABCA4 gene. The therapy aims to slow or stop vision loss, and the study will check if it is safe and if it works.
Beijing, Beijing MunicipalityAges 8 years+ - NCT07248449Recruiting
Exercise study for children with Down syndrome ages 3-8
This trial tests two types of exercise—Cuevas Medak and rebound exercises—to see which one improves physical fitness in children with Down syndrome. It may help your child get stronger and move better.
Lahore, Punjab ProvinceAges 3–8 - NCT07240844RecruitingPhase 4
Enavogliflozin for amyloid heart disease
This study tests a drug called enavogliflozin to see if it helps people with a specific type of heart failure caused by amyloid buildup. It may improve symptoms and daily function.
SeoulAges 19 years+ - NCT07249112Recruiting
Genomic study of metabolic fatty liver disease
This study aims to understand the genetic factors behind metabolic fatty liver disease (MASLD) by comparing patients with advanced liver scarring or liver cancer to healthy blood donors. It may help doctors better predict and treat the disease in the future.
Milan, MilanoAges 18–90 - NCT07248267Recruiting
Vojta therapy for children with Down syndrome
This study is testing a therapy called Vojta therapy to see if it helps children with Down syndrome move better and walk more easily. It compares the therapy to other supportive care.
Lahore, Punjab ProvinceAges 2–10 - NCT07240259Enrolling by invitation
Using a donated aortic patch to rebuild a child's windpipe
This trial tests a new way to repair a child's windpipe using a piece of donated aorta (a blood vessel) as a patch. It's for children who have severe breathing problems from a narrowed or damaged windpipe and cannot have standard surgery.
TaipeiAges birth–18 years - NCT07246265Recruiting
Morphine clearance study for sickle cell crisis in ICU
This study looks at how the body clears morphine in adults with sickle cell disease who are in the intensive care unit (ICU) for a pain crisis or acute chest syndrome and are receiving morphine through a patient-controlled analgesia (PCA) pump. The goal is to understand how kidney function affects morphine breakdown.
Tours, FranceAges 18 years+ - NCT07244133Recruiting
Powerball exercise for kids with Down syndrome
This trial tests whether using a Powerball (a small exercise ball) can improve hand strength and coordination in children with Down syndrome. It may help with daily tasks and motor skills.
Lahore, Punjab ProvinceAges 6–10 - NCT07247058RecruitingPhase 4
Testing Isturisa for mild cortisol excess from adrenal tumors
This study tests whether a drug called Isturisa (osilodrostat) can safely lower mild cortisol excess in people with adrenal tumors. It aims to help patients avoid the long-term effects of too much cortisol.
Baltimore, MarylandAges 18 years+ - NCT05674487Recruiting
Balloon vs prostaglandin medicine to start labor in small babies
This trial compares two ways to help start labor by the vagina in pregnancies where the baby is measuring small. It aims to see which method works better and is safer for getting birth when needed.
BordeauxAges 18 years+ - NCT06735625Recruiting
AHSP as a biomarker for sickle cell disease
This study looks at a substance in the blood called AHSP to see if it can help track sickle cell disease. It compares people with sickle cell disease (SS or Sβ0 type) to people without it, to better understand the condition.
Saint-DenisAges 3 years+ - NCT06788314RecruitingPhase 2
Enalapril for painful venous malformations
This trial tests if the blood pressure drug enalapril can reduce pain from venous malformations (abnormal veins). It may help people whose pain limits daily activities or interrupts sleep.
Oslo, NorwayAges 18–70 - NCT06794008RecruitingPhase 2
CAR-T cell therapy for hard-to-treat autoimmune diseases
This trial tests a new treatment using your own immune cells (called CAR-T cells) designed to target and attack cells that cause autoimmune diseases. It is for people with lupus, Sjögren's syndrome, inflammatory myopathy, scleroderma, Behcet's disease, ANCA-associated vasculitis, IgG4-related disease, antiphospholipid syndrome, or thrombotic thrombocytopenic purpura who have not improved with standard therapies.
Beijing, Beijing MunicipalityAges 18–65 - NCT06907836Recruiting
Transthyretin cardiac amyloidosis in internal medicine
This study looks back at medical records of patients who were diagnosed with transthyretin cardiac amyloidosis (a type of protein buildup in the heart) while hospitalized in internal medicine at Strasbourg University Hospitals between 2013 and 2023. It aims to describe their health journey and outcomes.
StrasbourgAges 18 years+ - NCT06906406Recruiting
Quality of life in children with clubfoot
This study looks at how clubfoot affects the daily life and well-being of children aged 2 to 15. It uses questionnaires to understand their physical and emotional health, which can help improve care and support for kids with this condition.
Drammen, BuskerudAges 2–15 - NCT04507997Recruiting
Study of the natural course of Angelman syndrome
This study follows people with Angelman syndrome to understand how their symptoms and development change over time. It may help researchers better describe the condition and support future treatments.
Los Angeles, CaliforniaAges Any age - NCT07238426Enrolling by invitation
Heart screening for older adults in Ordu, Turkey
This study is a screening program in Ordu, Turkey, to find older adults (65+) who might have a heart condition called cardiac amyloidosis. It uses medical records and heart ultrasounds to check people who already have certain heart problems.
Ordu, OrduAges 65 years+ - NCT07235384RecruitingPhase 1
Zai Lab tests ZL-1503 for moderate to severe eczema
This study tests a new medication called ZL-1503 for people with moderate to severe atopic dermatitis (eczema) that hasn't improved enough with topical treatments. The trial will check if the drug is safe and how well it works.
Woolloongabba, QueenslandAges 18–65 - NCT07230483RecruitingPhase 2
Long-term safety of CM512 for atopic dermatitis
This study is for people who have already completed a previous study of CM512 for atopic dermatitis. It will check if the medicine is safe and works well over a longer period.
Beijing, Beijing MunicipalityAges 18 years+ - NCT06212947Recruiting
An observational study for children with hypochondroplasia
This study observes children with hypochondroplasia to learn more about the condition. It does not involve any treatment—just monitoring and data collection to help future research.
Phoenix, ArizonaAges Up to 15 years - NCT06863701RecruitingPhase 3
Combination therapy for acute gout flare
This trial tests a combination of two medicines, etoricoxib and betamethasone, for quickly treating a painful gout flare. It is for people who have had gout before and whose current attack started within two days.
Mexico CityAges 18 years+ - NCT06909656Recruiting
Brain Activity Study in Tourette Syndrome with Deep Brain Stimulation
This study looks at brain activity in people with Tourette syndrome who are already receiving deep brain stimulation (a treatment that uses electrical pulses to help control tics). The goal is to learn more about the brain signals linked to tics, which could help improve future treatments.
BordeauxAges 16 years+ - NCT07233421Enrolling by invitation
Blood sugar levels in children with Long QT syndrome on beta-blockers
This study looks at how often low blood sugar (hypoglycemia) happens in young children with Long QT syndrome who take beta-blocker medicine. It may help doctors understand if these children need extra monitoring.
HelsinkiAges Up to 7 years - NCT07237321Recruiting
Magnetic stimulation for flat feet: effects on arch and strength
This study tests whether magnetic stimulation of foot muscles can improve the arch of the foot and muscle strength in adults with flat feet. If you have flat feet without major leg or health problems, this trial may help find a new way to strengthen your feet.
Ljubljana, LjubljanaAges 18–50
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.