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
- NCT06587685RecruitingPhase 3
PG-011 gel for eczema in teens and adults
This trial tests a new gel called PG-011 for people with mild to moderate eczema (atopic dermatitis). The gel is applied to the skin, and the study will check if it helps improve symptoms safely.
Shenyang, LiaoningAges 12–75 - NCT06588426Recruiting
Skin punch biopsy collection for heart disease research
This trial collects a small skin sample (like a biopsy) from people with congenital heart disease. The samples help prepare for future clinical trials that may test new heart treatments.
Rochester, MinnesotaAges 18 years+ - NCT06588543Recruiting
Planning brain blood vessel procedures using patient scans
This trial uses your medical images (like MRI or angiography) to create a personalized map of your brain's blood vessels. The goal is to help doctors better plan minimally invasive treatments for aneurysms or arteriovenous malformations.
GhentAges Any age - NCT06588764Recruiting
Modified eye surgery for moderate to severe Graves' eye disease
This trial tests a modified orbital decompression surgery to treat moderate-to-severe swelling and bulging of the eyes caused by Graves' disease. It aims to improve eye function and appearance for people whose thyroid condition has been stable for at least 6 months.
Guangzhou, GuangdongAges 18–80 - NCT06589245RecruitingPhase 1
Ciclesonide study for breathing support in preterm infants
This study tests an inhaled anti-inflammatory drug called ciclesonide for premature babies who need a breathing tube. It aims to see if the drug can help their lungs heal and reduce the need for steroids.
Kansas City, MissouriAges 1 week–1 month - NCT06590142Recruiting
Studying bowel tissue for future Hirschsprung's treatments
This trial collects leftover intestine tissue from children having bowel surgery to help develop future treatments using a child's own cells. It aims to find better ways to repair the bowel without needing donor tissue.
Liverpool, MerseysideAges birth–15 years - NCT06590220Recruiting
Predicting treatment needs for moderate to severe thyroid eye disease
This study aims to build a model that helps doctors predict how thyroid eye disease (TAO) will progress, specifically for patients with moderate to severe symptoms. If you have had TAO and meet the activity criteria, your participation could lead to better personalized care.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT06590974Recruiting
Study of human protein C concentrate for protein C deficiency
This trial is studying a medication called TAK-662 (freeze-dried human protein C concentrate) in people with congenital protein C deficiency. The goal is to see how well it works and how safe it is for patients who need this replacement therapy.
Tokyo, TokyoAges Any age - NCT06591793RecruitingPhase 1/Phase 2
Gene therapy for Usher syndrome type 1B vision loss
This trial tests an experimental gene therapy given as an injection under the retina. It aims to slow or stop vision loss in people with Usher syndrome type 1B, a rare genetic condition that causes both hearing and vision problems.
NaplesAges 18–50 - NCT06591936Recruiting
Genetic study of alpha thalassemia in children
This trial looks at the genetic profiles of children with suspected alpha thalassemia, a type of inherited anemia. It aims to better understand this condition in children treated at Sohag University Hospital.
SohagAges birth–18 years - NCT06593951Recruiting
EPM1 natural history study
This study will follow people with EPM1 over time to better understand the disease. It is a registry, meaning you will share information through video calls and questionnaires, with no experimental treatments involved.
Boston, MassachusettsAges Any age - NCT06594458Recruiting
New method to keep mom and baby together after C-section
This study tests a new procedure to reduce separation between mother and newborn after a planned C-section. It may help babies with mild breathing problems who need a little extra oxygen stay with their moms instead of going to a separate nursery.
RomaAges 1 minute–3 days - NCT06595407Recruiting
Loss of Y Chromosome in Aortic Stenosis Study
This study looks at how losing the Y chromosome in blood cells may be linked to aortic stenosis, a narrowing of the heart's aortic valve. It aims to understand why this condition may be more severe in men.
Charlottesville, VirginiaAges 40 years+ - NCT06595420Recruiting
Kidney health in people with cystic fibrosis on new treatments
This study looks at how the kidneys work in people with cystic fibrosis, especially those taking highly effective new treatments. It aims to understand kidney function in different groups, including those who are hospitalized frequently or rarely.
Birmingham, AlabamaAges 7 years+ - NCT06596746Recruiting
Tracking markers for neurodegenerative diseases
This study tracks changes in people with Parkinson's disease, other movement disorders, or cognitive decline to find better ways to measure disease progression.
Cassino, FrosinoneAges 10 years+ - NCT06597019RecruitingPhase 3
Study of inclisiran for children with high cholesterol
This trial tests a medicine called inclisiran to lower high cholesterol in children ages 6 to 12 with a genetic condition called heterozygous familial hypercholesterolemia (HeFH). It may help children who still have high cholesterol despite taking other treatments.
San Francisco, CaliforniaAges 6–11 - NCT06599411Recruiting
Skin biomarkers for inflammatory disease treatment
This study looks for clues in your skin and blood that could predict how well you'll respond to treatments for conditions like eczema, psoriasis, or lupus. It aims to help doctors choose the best therapy for you.
ParisAges 18 years+ - NCT06599619Recruiting
Study of PD1 therapy on atypical moles in melanoma patients
This trial tests if an immunotherapy drug (anti-PD1) can change atypical (dysplastic) moles in people who have had melanoma. It may help understand how these drugs affect mole cells.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06599892Recruiting
Cystic fibrosis care with highly effective modulators
This study looks at how cystic fibrosis care is organized when patients take highly effective CFTR modulator drugs. It aims to understand if care needs change for those on modulators compared to those who cannot take them.
LyonAges 6 years+ - NCT06600269Recruiting
Cycling training for ataxia recovery in SCA
This study tests whether cycling-based coordination training can help people with spinocerebellar ataxia (SCA) improve balance and movement by priming the brain for recovery.
TaoyuanAges 18 years+ - NCT05518188RecruitingPhase 1/Phase 2
Gene therapy trial for a childhood AP4M1 brain disorder
This trial studies a one-time gene therapy meant to help children with SPG50 disease caused by changes in the AP4M1 gene. Researchers will give the treatment and closely monitor safety and early signs of benefit.
Dallas, TexasAges 4 months–10 years - NCT04509050Recruiting
Testing a highly effective CF medicine in young children
This study looks at how well a highly effective cystic fibrosis (CF) medicine works in infants and young children, and what biological changes it causes. It may help families understand whether the medicine improves CF function and related measures early in life.
Birmingham, AlabamaAges Up to 10 years - NCT07689331Recruiting
Hungarian national amyloidosis study
This study creates a registry for people diagnosed with systemic amyloidosis in Hungary. It helps doctors better understand the condition by collecting medical history and tracking outcomes over time.
BalatonfüredAges 18 years+ - NCT03937661RecruitingPhase 2/Phase 3
Platelet-rich plasma injected into ovaries for poor egg responders
This trial studies whether an injection of your own “platelet-rich” plasma into the ovaries can improve egg production in people who respond poorly to fertility treatment. It targets women with low egg reserve and tries to see if this approach helps them get better results from stimulation.
AthensAges 35–47
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.