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 applicable2,772
- Phase 2282
- Phase 1235
- Phase 3215
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 353
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis129
- 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
- Arizona79
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT06772402Enrolling by invitation
Gene therapy for children with type 2 SMA
This trial tests a new gene therapy called GCB-001 for children with type 2 spinal muscular atrophy who can sit but not walk. It aims to improve muscle function and overall health.
Hangzhou, ZhejiangAges 2–12 - NCT06763549Enrolling by invitation
AI-powered wearable patch for heart and lung monitoring
This study tests an AI-powered wearable patch (the COR ECG) to see if it can better track heart and lung health. It may help doctors find problems earlier without needing bulky equipment.
Melbourne, FloridaAges 18 years+ - NCT06747546Enrolling by invitation
Oxygen-based blood flow management after heart surgery
This study is testing a new way to manage blood flow after heart surgery in children with congenital heart disease. The goal is to make sure the body gets the right amount of oxygen to help recovery.
Minhang, Shanghai MunicipalityAges 1 week–18 years - NCT06744569Enrolling by invitationPhase 1
Testing a new ointment for atopic dermatitis in healthy adults and patients
This trial tests a new ointment called CU-10101. It will first be tested in healthy volunteers to check safety, and then in people with atopic dermatitis (eczema) to see how well it works for mild to moderate symptoms.
Shanghai, Shanghai MunicipalityAges 18–65 - NCT06726122Enrolling by invitation
Matrix Pro skin treatment study for ages 18-84
This study tests a new device called Matrix Pro that uses Profound Matrix technology to treat skin concerns. It aims to see if the treatment is safe and works well for people with different skin types.
Amsterdam, EKAges 18–84 - NCT06724497Enrolling by invitationPhase 1/Phase 2
Comparing 3D face scan methods for jaw surgery patients
This trial tests whether using a handheld 3D scanner is as accurate as a standard optical scanner for predicting soft tissue changes after corrective jaw surgery. It aims to find a more convenient and accessible way to plan surgery for patients with an underbite.
Cairo, Giza GovernorateAges 18–50 - NCT06717932Enrolling by invitation
Long-term safety study of CS-101 injection
This study follows up with people who already received the CS-101 injection in a previous trial to check its safety and how well it works over time.
NanningAges 12–35 - NCT06693856Enrolling by invitation
Steroid pulse therapy for active Graves' eye disease
This study tests whether high-dose steroid pulses (given through a vein) can help people with moderate to severe Graves' eye disease that is still active. It is for people who have not yet been treated with steroids and whose eye problems began within the past year.
Shanghai, Shanghai MunicipalityAges 25–70 - NCT06685536Enrolling by invitation
Long-term follow-up for CS-101 recipients
This study follows up on people who have already received the CS-101 infusion in a previous trial. It aims to check how participants are doing over time. There are no restrictions to join if you already received CS-101.
Nanning, GuangxiAges 6–35 - NCT06679972Enrolling by invitation
Impact of pregnancy liver condition on baby's breathing
This study looks at how a pregnancy liver condition (intrahepatic cholestasis) affects the baby's breathing after birth. It compares newborns whose mothers had this condition (and took medication for it) with newborns whose mothers did not have any liver issues during pregnancy.
Bucharest, BucharestAges 1 hour–4 weeks - NCT06679790Recruiting
Understanding changes in Tourette Syndrome over time
This study tracks how tics and related symptoms change over time in people with Tourette Syndrome. It aims to better understand the condition and could help develop better treatments.
Minneapolis, MinnesotaAges 12–17 - NCT06664541Enrolling by invitation
Gene therapy decisions and quality of life
This study looks at how people with blood diseases and their families make decisions about gene therapy, and how it affects their quality of life. It involves interviews or surveys to understand your experience.
Washington D.C., District of ColumbiaAges 18 years+ - NCT06649825Enrolling by invitation
Vitamin B1 for familial adenomatous polyposis
This trial tests whether taking vitamin B1 can help people with familial adenomatous polyposis (FAP) reduce the number of polyps in their colon or rectum. It is designed for people who still have most of their bowel and have more than 10 polyps.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT06642662Enrolling by invitation
Finding biomarkers in children after heart surgery
This study looks at proteins in the blood that may help doctors understand how children are recovering after heart surgery. It enrolls children under age 5 having certain types of heart repairs at Boston Children's Hospital.
Boston, MassachusettsAges 2 days–5 years - NCT06640998Enrolling by invitation
Matrix pro for eyebrow and neck lift
This trial tests a radiofrequency microneedling device called Matrix Pro to lift droopy eyebrows and tighten loose skin under the chin and neck. It may help if you have mild to moderate skin laxity and are willing to avoid other cosmetic treatments during the study.
Marlborough, MassachusettsAges 18–75 - NCT06627543Enrolling by invitation
Enzyme replacement therapy for heart health in Gaucher type 3
This study looks at how enzyme replacement therapy affects heart function in children with Gaucher disease type 3. It aims to see if the therapy helps keep the heart healthy.
AsyutAges Any age - NCT06614582Enrolling by invitation
Long-term nose tube study for children with weak muscle tone
This study looks at the long-term safety and effectiveness of a nose tube (nasopharyngeal airway) for children who have obstructive sleep apnea due to weak muscle tone. It's for children who already used this device in a previous study.
Ann Arbor, MichiganAges 3–23 - NCT06606340Enrolling by invitation
Long-term study of eteplirsen, golodirsen, or casimersen for DMD
This study follows people with Duchenne muscular dystrophy (DMD) who are taking eteplirsen, golodirsen, or casimersen. It aims to understand how these medicines work in everyday life over a long period.
Little Rock, ArkansasAges Any age - NCT06605612Enrolling by invitation
Falls risk test for people with muscle and nerve conditions
This study is creating a test to predict falls in people with neuromuscular disorders. You will fill out a questionnaire about your fall risk.
München, BavariaAges 18–65 - NCT06597188Enrolling by invitation
TAVR vs. Surgery for severe bicuspid aortic valve stenosis
This trial compares a less invasive valve replacement (TAVR) with traditional open-heart surgery for people with a specific type of severe aortic valve narrowing (bicuspid valve). It aims to see which approach works better for those at intermediate or low surgical risk.
Chengdu, SichuanAges 18–75 - NCT06584877Enrolling by invitation
Understanding how childhood tumors and birth conditions start
This study looks at leftover tissue samples from children who have had tumors or birth conditions. Researchers want to learn more about how these conditions develop, and you may help if you or a relative fits the criteria.
CambridgeAges Any age - NCT06559176Enrolling by invitationPhase 1/Phase 2
Gene editing trial for chronic granulomatous disease
This trial tests a new gene editing treatment called PM359 for people with a specific type of chronic granulomatous disease (CGD) caused by a delGT mutation in the NCF1 gene. The goal is to see if it's safe and helps fight infections and inflammation.
Los Angeles, CaliforniaAges 6 years+ - NCT06549400Enrolling by invitationPhase 3
Study on long-term safety of mexiletine for myotonic dystrophy
This study tests if a daily capsule of mexiletine is safe and helpful over 26 weeks for people with myotonic dystrophy type 1 or 2. It's for people who finished a previous related study and want to continue treatment.
LeuvenAges 16 years+ - NCT06545305Enrolling by invitation
Eye problems in homocystinuria patients, 2018–2022
This study looks at eye problems in people with homocystinuria. It will review medical records from 2018 to 2022 to find out how common eye issues are and what might increase the risk.
JeddahAges 20–30
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 5,795 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.