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
- NCT06136572Recruiting
Study on assertiveness and emotions in Tourette's
This study looks at how people with Tourette's syndrome manage emotions and assertiveness. It may help doctors understand the challenges patients face.
Clermont-Ferrand, FranceAges 18 years+ - NCT06136767Recruiting
Registry for systemic eczema treatments
This study is a registry for people with eczema (atopic dermatitis) who are under 26 and have taken or plan to take body-wide treatments like pills or injections. By joining, you help researchers track how these treatments work over time.
Baltimore, MarylandAges 1–26 - NCT06138509Recruiting
Serotonin levels in children with albinism
This study measures blood serotonin levels in children with albinism compared to children without the condition. It aims to learn if serotonin plays a role in albinism, which could help future treatments.
ParisAges 2–17 - NCT06138639RecruitingPhase 1/Phase 2
Gene therapy SGT-003 for Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
Little Rock, ArkansasAges birth–17 years - NCT06139510Recruiting
Understanding Pain in Sickle Cell Disease
This study looks at what causes pain in people with sickle cell disease. They hope to learn more about pain patterns to better help patients manage it.
Durham, North CarolinaAges 15–40 - NCT06142552RecruitingPhase 3
Study of a longer-lasting hemophilia A treatment for men
This trial tests a new form of factor VIII that lasts longer in the body, so you may need fewer infusions. It is for men with severe hemophilia A who have already used factor VIII products for at least 150 days.
BeijingAges 12–65 - NCT06143254Recruiting
Infant sign training for speech development
This trial tests whether teaching infants with cleft palate simple signs can help them communicate before they can speak. It may support their speech and language development.
Ghent, East FlandersAges 1–2 - NCT06144125RecruitingPhase 4
Blood management trial for heart surgery patients
This trial compares two treatments (prothrombin complex concentrate and fresh frozen plasma) for managing bleeding during heart surgery. It aims to find which approach works better and is safer for patients.
Seoul, SeoulAges 20–75 - NCT06145373RecruitingPhase 4
Fitusiran for preventing bleeds in severe hemophilia A
This study tests a new medicine called fitusiran to help prevent bleeding episodes in people with severe hemophilia A who are already receiving emicizumab. It may offer an additional treatment option for those with or without inhibitors.
Los Angeles, CaliforniaAges 12 years+ - NCT06145841Recruiting
Gene test to guide infection treatment in AIDS
This study is for people with HIV/AIDS who develop severe pneumonia or septic shock. It uses a special genetic test (metagenomic next-generation sequencing) to find the exact germ causing the infection so doctors can choose the right antibiotic.
Guangzhou, GuangdongAges 18–65 - NCT06145893RecruitingPhase 3
Hemay005 for Behçet's disease mouth sores
This study tests a new pill called Hemay005 for adults with Behçet's disease who have at least two mouth sores that need treatment beyond creams or rinses. It aims to see if the drug safely reduces mouth ulcers.
Beijing, Beijing MunicipalityAges 18–75 - NCT06146231Recruiting
Breast reconstruction with Motiva implants and fat grafting
This study tests a new breast reconstruction method using Motiva Flora implants and your own fat to rebuild the breast after a mastectomy. It is for women who need tissue expansion as part of their reconstruction and have completed cancer treatments at least one year earlier.
GhentAges 18 years+ - NCT06147414Recruiting
Blood test for genetic disorders in pregnancy
This study tests a safer blood test to check for certain genetic disorders during pregnancy, instead of an invasive procedure like amniocentesis. It's for pregnant women who are at risk of passing on a known genetic condition.
ParisAges 18 years+ - NCT06148051Recruiting
Australian CADASIL study: learning more about this condition
This study is building a group of Australians with CADASIL (a genetic condition that affects blood vessels in the brain) to learn more about the disease. As a participant, you will have a medical exam, a blood test, and a short memory test.
Newcastle, New South WalesAges 18 years+ - NCT06150716RecruitingPhase 1
Study of a new drug for Pelizaeus Merzbacher disease in boys
This trial is testing an experimental drug (ION356) given by a lumbar puncture to see if it is safe and how it works in boys with Pelizaeus Merzbacher disease (PMD). The drug is designed to target the genetic cause of PMD.
Atlanta, GeorgiaAges 2–17 - NCT06150950Recruiting
Exercise rehab for Fontan failure
This trial tests if a supervised exercise program (cardiac rehabilitation) can help people who have had a Fontan procedure and are now experiencing heart failure symptoms. It aims to improve your strength, stamina, and overall well-being.
Stanford, CaliforniaAges 18 years+ - NCT06152302Recruiting
Water mobility test for infants with SMA
This study tests how well infants with spinal muscular atrophy (SMA) can move in water. The goal is to see if gentle water exercises can help with muscle weakness and overall development.
GarchesAges Up to 1 year - NCT06153407Recruiting
Genetic test for early valve disease in bicuspid aortic valve
This study looks at how genes may affect the risk of calcium buildup in the aortic valve in people with a bicuspid aortic valve. By joining, you help researchers find better ways to predict and manage this heart condition.
SeoulAges 19–80 - NCT06156670Recruiting
A study of closing a PFO to prevent strokes
This trial looks at the safety and results of closing a PFO (a small hole in the heart) in people who have had a stroke or embolism with no other clear cause. If you qualify, doctors will close your PFO using a small tube inserted through a blood vessel and then follow your health over time.
MoscowAges 18–70 - 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+ - NCT06160310Recruiting
TSC & LAM Pregnancy Registry Study
This study is a registry that gathers information from pregnant women with Tuberous Sclerosis Complex (TSC) or Lymphangioleiomyomatosis (LAM), as well as infants born to them. The goal is to better understand how these conditions affect pregnancy and the baby's health.
Cincinnati, OhioAges Any age - NCT06162338Recruiting
Study of LY-M001 for Gaucher Disease Type I
This study tests an injection called LY-M001 for adults with Gaucher disease type I. The goal is to see if it can safely help replace the missing enzyme and improve symptoms.
Hanzhou, ZhejiangAges 18–60 - NCT06165341RecruitingPhase 3
Fazirsiran for mild liver scarring in alpha-1 antitrypsin deficiency
This study tests an investigational drug called fazirsiran, which is designed to treat alpha-1 antitrypsin deficiency-related liver disease with mild scarring. The goal is to see if fazirsiran is safe and can improve or slow liver damage in people with the PiZZ genetic type.
Phoenix, ArizonaAges 18–75 - NCT06169150Recruiting
Studying nervous system issues in immune disorders
This study looks for and tracks nervous system problems in people with immune system disorders. It aims to better understand how infections or inflammation affect the brain and nerves in these patients.
Bethesda, MarylandAges 2–120
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.