Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,762 recruiting studies in this area right now.
By phase
- Not applicable2,682
- Phase 2280
- Phase 1239
- Phase 3209
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease133
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy36
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)29
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
- Children's Hospital of Philadelphia23
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
Where studies are running
- California324
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York84
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT07229079Recruiting
Body awareness therapy for flat feet
This study tests whether basic body awareness therapy can help people with flexible flat feet (when the foot arch flattens under weight but returns when not standing). If you have flat feet without other foot problems, you may qualify.
Mardin, Mardi̇nAges 18–45 - NCT07223658RecruitingPhase 1/Phase 2
Study of ARO-DIMERPA for high triglycerides and cholesterol
This trial tests an experimental drug, ARO-DIMERPA, for people with very high triglycerides and cholesterol. It aims to lower those levels and reduce heart risks.
Charlestown, New South WalesAges 18 years+ - NCT07223944RecruitingPhase 3
Gene therapy trial for Gaucher disease type 1
This trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
Los Angeles, CaliforniaAges 18 years+ - NCT07228364RecruitingPhase 1
Testing safety of AZD1613 for polycystic kidney disease
This trial tests a new study drug called AZD1613 for adults with a certain type of kidney disease (ADPKD). Researchers want to see if it is safe, how the body handles it, and what the right dose might be.
Birmingham, AlabamaAges 18–65 - NCT07227207RecruitingPhase 1/Phase 2
Study of TSRA-196 for adults with Alpha-1 Antitrypsin Deficiency
This trial tests a new medicine called TSRA-196 for people with Alpha-1 Antitrypsin Deficiency who have the PiZZ genetic type. The study looks at whether TSRA-196 can help with lung or liver problems related to this condition.
Jacksonville, FloridaAges 18–70 - NCT07225231Enrolling by invitation
Home EEG monitoring for fenfluramine treatment in dravet and lgs
This trial tests whether a reduced, home-based EEG monitoring device can help doctors safely adjust the dose of fenfluramine, a treatment for seizures in Dravet syndrome and Lennox-Gastaut syndrome. Participation may help find the right dose with fewer hospital visits.
Louisville, KentuckyAges 3 years+ - NCT07224581RecruitingPhase 3
Study of a new drug for GRIN-related developmental disorder
This trial tests a new drug for children with GRIN-related neurodevelopmental disorder caused by certain gene changes. The drug aims to improve seizures and other symptoms.
Los Angeles, CaliforniaAges 1 month–18 years - NCT07224360RecruitingPhase 2
Safety of a new drug for sickle cell pain
This study tests a new medicine called anumigilimab to see if it is safe for people with sickle cell disease. The study focuses on those who have had pain crises recently, to help manage and prevent them.
Little Rock, ArkansasAges 18 years+ - NCT07221851RecruitingPhase 3
Weekly vs daily growth hormone for short stature in children
This trial tests a weekly growth hormone injection (lonapegsomatropin) compared to the standard daily growth hormone shot in children with short stature due to certain conditions. It aims to see if the weekly shot works as well or better and is safe.
Palo Alto, CaliforniaAges 2–17 - NCT07226206RecruitingPhase 1/Phase 2
Gene therapy for adults with severe hemophilia A
This trial tests a new gene therapy called SPK-8011QQ for adults with severe or moderately severe hemophilia A. The goal is to see if a single infusion can help your body produce its own factor VIII, reducing or stopping the need for regular factor VIII injections.
Orange, CaliforniaAges 18 years+ - NCT07228793Recruiting
A study tracking EYS-related retinitis pigmentosa over time
This study follows people with retinitis pigmentosa caused by changes in the EYS gene over 4 years. It collects data on how the disease progresses and does not test any new treatments.
MoscowAges 14–100 - NCT07229430Recruiting
Ultrasound treatment for loose neck and chin skin
This study tests a high-intensity focused ultrasound (HIFU) device to lift and tighten loose skin under the chin and neck. If you are in good health and want a non-surgical option for mild to moderate skin laxity, this trial may be for you.
Los Angeles, CaliforniaAges 30–65 - NCT07223697RecruitingPhase 2
Long-term safety of Afimkibart for eczema patients
This study tests the long-term safety and effectiveness of a medicine called Afimkibart for people with atopic dermatitis (eczema). It is for people who already completed a previous study of this medicine and had at least a 50% improvement in their eczema.
Los Angeles, CaliforniaAges 18 years+ - NCT06337864Recruiting
Large neutral amino acids for adults with classical PKU
This study tests whether a specific mix of large neutral amino acids (LNAAs) can help adults with classical PKU by lowering phenylalanine levels in the brain. It includes an MRI and PET scan to see how the treatment affects brain activity.
CopenhagenAges 18–50 - NCT06325488Recruiting
Kidney Health Study in Fabry Disease
This study uses MRI scans to look at how Fabry disease affects the kidneys, including scarring, swelling, and oxygen levels. It may help researchers understand kidney problems better and find new ways to monitor the disease.
CopenhagenAges 18 years+ - NCT06907186Recruiting
Psychological support program for amyloidosis patients and families
This study tests a structured psychological support program for people with hereditary transthyretin amyloidosis (ATTRv) with heart involvement, their caregivers, and those who carry the gene but have no symptoms yet. The goal is to see if the program is practical and well-accepted.
PaviaAges 18 years+ - NCT04936074Recruiting
Fusion vs no fusion after neck cord decompression
This trial compares two surgery approaches for people with a gradually worsening spinal cord problem in the neck: doing fusion (joining bones together) versus not doing fusion after decompression (making more space for the spinal cord). It aims to see which approach works better and is safer for symptoms and recovery.
UppsalaAges 18 years+ - NCT05904301Recruiting
Registry for autoimmune and autoinflammatory disease patients
This study builds a nationwide list (registry) of people with certain autoimmune or inflammation-related illnesses. It helps researchers understand these diseases better over time, which can guide future care.
YerevanAges Any age - 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 - NCT07220785RecruitingPhase 3
Testing mibavademab for generalized lipodystrophy in adults and children
This trial tests a new drug called mibavademab for people with generalized lipodystrophy, a condition where the body can't store fat properly, leading to problems like diabetes and high triglycerides. It aims to see if the drug safely improves these metabolic issues.
Ann Arbor, MichiganAges 2 years+ - NCT07215715Recruiting
Heart ultrasound study for older adults with heart failure
This study uses a special ultrasound to measure heart stiffness in people 60 and older who have heart failure with preserved or mildly reduced pumping function, with or without a condition called ATTR amyloidosis (a protein buildup in the heart). It also includes healthy controls.
Boston, MassachusettsAges 60 years+ - NCT07220564RecruitingPhase 1
Study of a new medicine for haemophilia A
This study tests a new medicine called Inno8 in people with haemophilia A. It looks at how different doses work in the body and may help improve treatment options.
Aurora, ColoradoAges 18–64 - NCT07216027RecruitingPhase 2
A study of a new eczema medicine for adults
This trial tests a study drug (PF-08049820) for people with moderate to severe eczema who haven't found a suitable prescription treatment. It aims to see if the drug can improve skin symptoms and itching.
Phoenix, ArizonaAges 18–64 - NCT07221331Recruiting
Neurofibromatosis type 1 study in Egypt
This study looks at how neurofibromatosis type 1 (NF1) affects people in Egypt, including how it progresses and how it is managed. Researchers will review medical records of patients diagnosed with NF1 between 2010 and 2023 to learn more about the condition.
Al MansurahAges Any age
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,762 recruiting medical genetics studies from the U.S. registry right now, out of 5,482 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.