Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,756 recruiting studies in this area right now.
By phase
- Not applicable2,677
- Phase 2277
- Phase 1238
- Phase 3210
- Phase 1/Phase 2208
- Phase 497
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease134
- Atopic Dermatitis125
- Congenital Heart Disease84
- Cystic Fibrosis76
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy37
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)28
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
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
- Massachusetts General Hospital22
Where studies are running
- California325
- Maryland161
- Alabama133
- Massachusetts107
- Arizona86
- New York85
- Texas70
- Minnesota68
- Pennsylvania62
- Florida59
Recruiting studies3,756 total
- NCT07776444Recruiting
Measuring brain blood flow with light and MRI
This study uses a special light-based device and MRI to measure how blood vessels in the brain respond to changes. It may help researchers better understand blood flow in people with and without sickle cell disease.
St Louis, MissouriAges 6–70 - NCT07781267Recruiting
Long-term outcomes of congenital adrenal hyperplasia
This study looks at the long-term experiences of adults with classic congenital adrenal hyperplasia (CAH), a condition affecting hormone production. It aims to understand patient satisfaction and health outcomes over time.
LilleAges 18–65 - NCT07781566Recruiting
Speech therapy intensity for cleft palate speech errors in children
This study looks at how well two different intensities of speech therapy work for children with a cleft palate who have speech sound errors. It aims to find the best way to help your child speak more clearly.
Ghent, East-FlandersAges 4 years+ - NCT06555237RecruitingPhase 2
MEK inhibitors for heart thickening in RASopathies
This trial tests a targeted drug (MEK inhibitor) to treat thickened heart muscle caused by a group of genetic conditions called RASopathies. It may help improve heart function in people with hypertrophic cardiomyopathy.
WarsawAges 1 day–18 years - NCT02098252Recruiting
Study for treating brain blood vessel malformations (AVMs)
This study is looking at treatment options for people who have an abnormal cluster of blood vessels in the brain (an AVM). It may help participants by offering a structured treatment approach, depending on the AVM type and recent symptoms.
Jacksonville, FloridaAges 5 years+ - NCT06553339RecruitingPhase 1
A study of HS-10516 for VHL syndrome tumors
This trial tests a new drug called HS-10516 in people with VHL syndrome or advanced kidney cancer. The goal is to see if the drug can shrink tumors and what side effects it might have.
Beijing, Beijing MunicipalityAges 18–80 - NCT05954455Recruiting
Studying speech and swallowing development in children with SMA type 1
This study looks at how speech, communication, and swallowing (“bulbar function”) develop in babies and children with SMA type 1. It aims to better understand what to expect over time for children who start approved SMA treatments early.
LondonAges birth–18 years - NCT03764748RecruitingPhase 1
Washed stool transplant for Tourette syndrome in kids
This Phase 1 study tests whether a “washed” stool microbiota transplant can reduce Tourette syndrome tics in children. It aims to see if changing gut bacteria is safe and may improve tic severity.
Nanjing, JiangsuAges 6–17 - NCT05845723RecruitingPhase 2
Study drugs for blood-vessel Behçet’s to reduce inflammation
This Phase 2 trial tests tocilizumab and tofacitinib to see if they can better control vascular (blood-vessel) Behçet’s syndrome. It may help by lowering inflammation and reducing complications from aneurysms in the aorta or peripheral arteries.
Beijing, Beijing MunicipalityAges 18–65 - NCT06157268Recruiting
Study of muscle tiredness in congenital myopathies
This study follows patients with congenital myopathies to understand how their muscles get tired over time. It may help develop better treatments.
Nijmegen, GelderlandAges 2 years+ - NCT05098821Recruiting
Study factors that affect eczema control on dupilumab
This trial studies why eczema (“atopic dermatitis”) varies in severity even when people receive the medicine dupilumab. It may help doctors better predict who responds well and how to monitor treatment.
BerlinAges 18–75 - NCT07769957Recruiting
Brain scans for babies with slow growth in the womb
This study uses a special type of MRI (magnetic resonance imaging) to look at the brain development of babies who are growing slowly in the womb. The goal is to see if this scan can help doctors better understand this condition and how to care for these babies.
IstanbulAges 18–45 - NCT06544473RecruitingPhase 4
Estrogen pill vs. patch for Turner syndrome
This trial compares oral and transdermal (patch) estrogen to find the right dose for women with Turner syndrome. It aims to see which method works best for hormone replacement.
Aarhus, Aarhus NAges 18–50 - NCT07773246RecruitingPhase 1/Phase 2
Study of KRRO-121 in healthy adults and people with urea cycle disorder
This study tests an investigational drug called KRRO-121. It is first tested in healthy volunteers to check safety, and then in patients with urea cycle disorder (a condition where the body can't remove ammonia properly) to see if it helps.
Melbourne, VictoriaAges 18–65 - NCT07771465RecruitingPhase 3
Long-term safety of danicamtiv for genetic dilated cardiomyopathy
This study checks the long-term safety and effectiveness of the drug danicamtiv in people with a genetic form of dilated cardiomyopathy, a condition where the heart muscle becomes weak and enlarged. If you've already completed the earlier DAN-301 study, this trial continues to monitor you while you keep taking the medication.
Birmingham, AlabamaAges 18 years+ - NCT07767253RecruitingPhase 3
New injectable medicine for children with moderate to severe eczema
This study tests a new medicine (called 611) given as an injection to children with moderate-to-severe eczema that hasn't improved with creams. The goal is to see if it can reduce itching and clear the rash.
Beijing, Beijing MunicipalityAges 2–11 - NCT07774975RecruitingPhase 1
Safety study of pitavastatin nanoparticles for retinitis pigmentosa
This trial tests a new form of the drug pitavastatin, which is usually used for cholesterol, but here it's given as tiny particles injected into the eye. The goal is to see if it's safe and might help people with retinitis pigmentosa, a condition that causes vision loss.
FukuokaAges 18–70 - NCT07771023Enrolling by invitation
Exercise for people who had aortic repair
This trial looks at whether a supervised exercise program can improve fitness and quality of life in adults who were born with a narrowed aorta (coarctation) and had surgery to fix it as a baby or child. It aims to see if exercise is safe and helpful for heart health and daily energy, without placing extra strain on the repaired area.
LjubljanaAges 18 years+ - NCT07771621Enrolling by invitationPhase 1
PET scan for detecting heart amyloidosis
This study tests a new PET imaging agent that may help doctors see amyloid buildup in the heart more clearly. It may help people with heart amyloidosis or those suspected of having it.
St Louis, MissouriAges 18 years+ - NCT06566066Recruiting
Thyroid hormone resistance patient registry
This study creates a registry for people with thyroid hormone resistance caused by specific genetic mutations. It helps doctors collect data to better understand and treat the condition.
BerlinAges Any age - NCT05924555Recruiting
Exercise testing in inherited fatty acid breakdown disorders
This study checks how well people with long-chain fatty acid breakdown disorders tolerate exercise. It may help doctors better understand exercise limits and how to manage symptoms during activity.
Amsterdam, North HollandAges 18 years+ - NCT06570460RecruitingPhase 4
Estrogen pills vs. patch for Turner syndrome
This study compares two ways of taking estrogen (pills or a skin patch) in women with Turner syndrome to see which works better over time. It also includes healthy women for comparison.
Aarhus, Aarhus NAges 18–50 - NCT03336008Recruiting
Registry for certain inherited spinocerebellar ataxias
This study is a registry that collects information from adults with certain types of inherited ataxia caused by specific genetic diagnoses. It may help doctors better understand these conditions and support future research.
Hong Kong, ShatinAges 18–90 - NCT06057415Recruiting
Gut nerve stimulation study for high-risk newborns
This study tests whether stimulating the nerves in the gut can help improve digestion in high-risk newborns, specifically those born very early or with a diaphragmatic hernia. It takes place at Amalia Children's Hospital and aims to find a way to help these babies' intestines work better.
NijmegenAges Up to 2 days
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,756 recruiting medical genetics studies from the U.S. registry right now, out of 5,439 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.