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
- NCT06072976Recruiting
How feeding affects gut health in babies with bowel conditions
This study looks at whether breast milk or formula helps babies with certain bowel problems heal faster and reach full feeds sooner. It compares how different milk affects the bacteria in a baby's gut.
Seattle, WashingtonAges birth–55 years - NCT06073860Recruiting
Safety study of Luspatercept for MDS or thalassemia in Korea
This study is checking the safety of a drug called Luspatercept (brand name Reblozyl) for people in Korea who have myelodysplastic syndrome (a blood disorder) or beta-thalassemia (a type of inherited anemia). It is designed to monitor how safe the drug is when used as prescribed by your doctor.
SeoulAges 19 years+ - NCT06074276Recruiting
Almonds for facial skin collagen and wrinkles
This study tests whether eating almonds can improve collagen and reduce wrinkles on your face. If you are between 35 and 70, maintain your usual diet and skincare, and don't have a nut allergy, you may be eligible.
Sacramento, CaliforniaAges 35–70 - NCT06075693Recruiting
Spinal fluid markers for myotonic dystrophy
This study looks for markers in spinal fluid that could help doctors better understand myotonic dystrophy type 1 (DM1). It involves a spinal tap and MRI scan. It may help people with DM1 and healthy volunteers learn more about the condition.
Boston, MassachusettsAges 18 years+ - NCT06075251Recruiting
Online support for parents of kids with heart conditions
This study provides an online support program for parents or caregivers of children (ages 3 to 9) who have a congenital heart condition (a heart problem present at birth). The goal is to help you and your child cope and thrive, and you can join from home if you live in Ontario.
Toronto, OntarioAges 3–9 - NCT06076200Recruiting
How Piperacillin/Tazobactam works in pregnant women and newborns
This study looks at how the antibiotic Piperacillin/Tazobactam moves through the body of pregnant women and their newborns. It may help doctors decide the best dose to prevent infections in babies at risk.
JinanAges 18 years+ - NCT06079398RecruitingPhase 2
Weekly injection for infants with achondroplasia
This trial tests a new weekly injection called TransCon CNP for infants with achondroplasia (the most common form of dwarfism). The goal is to see if it can help children grow better than a placebo (a shot with no medicine).
Saint Paul, MinnesotaAges birth–2 years - NCT06081036Recruiting
Normal brain volumes from fetal MRI scans
This study looks at past fetal MRI scans to create a map of normal brain growth in babies before birth. It may help doctors better understand what healthy brain development looks like.
MarseilleAges 5 months–9 months - NCT06081062RecruitingPhase 3
Testing Fabagal in people with Fabry disease
This trial is testing a new enzyme replacement therapy called Fabagal for people with Fabry disease who have not had prior treatment. It aims to see if it helps with kidney, heart, or brain problems caused by the disease.
ManilaAges 8 years+ - NCT06081075Recruiting
Newborn Genomics Program for Sick Infants
This study looks at using rapid DNA testing (genomics) in newborns and children who are very sick in the hospital (NICU or PICU). The goal is to see if finding a genetic cause early can help guide treatment and improve outcomes.
AucklandAges birth–2 years - NCT06082713Recruiting
Extracellular Vesicles Study for Huntington's Disease
This study is collecting tiny particles called extracellular vesicles from blood and spinal fluid to learn more about Huntington's disease. It aims to find better ways to measure the disease in people who have HD and in those who do not.
Orlando, FloridaAges 18–75 - NCT06087315Recruiting
Study of oxygen use in children's hospitals
This study looks at how hospitals in six countries use pulse oximeters and oxygen therapy to treat children. It aims to find ways to improve oxygen delivery and care for children with serious illnesses.
Phnom PenhAges birth–15 years - NCT06087367Recruiting
Database study of KCNH2 gene variants
This study is building a database to better understand how different changes in the KCNH2 gene affect health. It may help doctors make more accurate diagnoses and predictions for people with this gene variant.
Nantes, Loire-atlantiqueAges Any age - NCT06088030RecruitingPhase 2
Arsenic trioxide plus chemo for p53-mutated childhood cancer
This trial tests if adding arsenic trioxide to standard chemotherapy can help children with cancers that have a certain p53 gene change. The goal is to see if the drug can reactivate the broken p53 protein to fight the tumor.
Guangzhou, GuangdongAges Up to 18 years - NCT06088589Recruiting
Speech-in-noise in autism and fragile x
This study measures how well people with autism or fragile X syndrome perceive speech in noisy settings, compared to typically developing individuals. It uses EEG to understand brain activity during listening tasks.
Cincinnati, OhioAges 15–35 - NCT06089902Recruiting
European registry for anomalous aortic origin of coronary arteries
This is a European registry for people with an anomalous origin of a coronary artery from the aorta (AAOCA). It will track how the condition is treated—whether with surgery or monitoring—to learn what works best.
Padova, PDAges Any age - NCT06090669RecruitingPhase 1
Trial of imatinib for people with RUNX1 deficiency
This trial tests if imatinib can help people with a genetic condition called RUNX1 deficiency, which causes bleeding problems. It aims to increase the activity of the RUNX1 gene to improve blood clotting.
Bethesda, MarylandAges 18–120 - NCT06091371Recruiting
Using a small implant for flatfoot in growing kids
This trial tests a new implant to treat flexible flatfoot in children. It may help reduce pain and improve foot function during growth.
BolognaAges 8–14 - NCT06091475Recruiting
Maintaining remission in improved dilated cardiomyopathy
This study tests whether people with dilated cardiomyopathy whose heart function has already recovered to normal can safely stop or reduce one of their heart medications while staying in remission. It aims to find the best way to keep the heart healthy without extra medicines.
LondonAges 18–85 - NCT06092034RecruitingPhase 2
Gene therapy for Danon disease in males
This trial tests a gene therapy called RP-A501 for men with Danon disease, a heart condition. It aims to replace the faulty gene that causes the disease to improve heart function.
La Jolla, CaliforniaAges 8 years+ - NCT06092346Recruiting
Understanding rare purine and pyrimidine metabolism disorders
This study aims to learn more about rare disorders of purine and pyrimidine metabolism (DPPM), which can affect the brain, immune system, kidneys, and muscles. Researchers will study people with these disorders, their family members, and healthy volunteers to better understand the condition over time.
Bethesda, MarylandAges 1 month–100 years - NCT06092463Recruiting
Immune system study in newborn intestines
This study looks at how the immune system develops in the intestines of newborns, including those born early or with a blocked intestine (atresia). It may help doctors understand inflammation and improve care for sick babies.
CopenhagenAges Up to 1 year - NCT06092528Recruiting
Pulmonary rehab for kids with primary immunodeficiency
This study tests whether a pulmonary rehabilitation program helps children aged 6–18 with primary immunodeficiency improve their breathing and physical fitness. If your child has this condition and is not currently having a lung infection or other serious health issues, they may be able to join.
Ankara, ÇankayaAges 6–18 - NCT06093100Recruiting
Wearable device study for Duchenne muscular dystrophy
This study uses a wearable device to track blood sugar levels and heart rate in people with Duchenne muscular dystrophy. It aims to understand how these factors relate to heart health over time.
Nashville, TennesseeAges 10 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.