Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,863 recruiting studies in this area right now.
By phase
- Not applicable2,776
- Phase 2282
- Phase 1237
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis129
- Congenital Heart Disease85
- Cystic Fibrosis78
- Down Syndrome55
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease39
- Hemophilia A39
- 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 Human Genome Research Institute (NHGRI)23
Where studies are running
- California339
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona77
- Texas72
- Minnesota67
- Pennsylvania66
- Ohio57
Recruiting studies3,863 total
- NCT06512454Recruiting
Study on inherited alpha-1 antitrypsin deficiency and related liver problems
This study looks at how alpha-1 antitrypsin deficiency affects the liver in adults. It aims to learn more about the liver problems caused by this condition and may help find better ways to manage them.
Gainesville, FloridaAges 18 years+ - NCT06449651RecruitingPhase 3
Nipocalimab to prevent low platelets in newborns
This study tests a medicine called nipocalimab for pregnant women whose babies are at risk of a bleeding condition called FNAIT. The medicine may help prevent low platelet counts and serious bleeding in the newborn.
LeuvenAges 18–45 - NCT07044804Recruiting
Brain stimulation to improve language in Down syndrome
This trial tests a non-invasive brain stimulation technique to help adolescents and young adults with Down syndrome improve their language skills. It aims to see if this safe method can boost word-finding and speaking ability.
RomeAges 12–21 - NCT06597006RecruitingPhase 3
Study of inclisiran for children with homozygous familial high cholesterol
This trial tests a new medicine called inclisiran to lower very high cholesterol in children aged 2 to 12 who have a specific genetic condition (homozygous familial hypercholesterolemia, or HoFH). You may not need to change your current cholesterol treatments, and the study will check if inclisiran can help reduce your LDL cholesterol further.
San Francisco, CaliforniaAges 2–11 - NCT07629999RecruitingPhase 1
Safety study of experimental drug BMS-986533 in healthy people
This early-stage study tests how a new experimental drug (BMS-986533) is processed and tolerated by healthy volunteers. Researchers will also check how food and stomach acid affect the drug, and whether it interacts with other medications.
Lincoln, NebraskaAges 18–55 - NCT07573631Enrolling by invitationPhase 2
Long-term Safety Study of BMN 351 for Duchenne Muscular Dystrophy
This study continues treatment with an investigational medicine called BMN 351 for people with Duchenne muscular dystrophy (a genetic muscle-weakening condition). It checks whether the medicine remains safe and effective when used over a longer period, building on results from an earlier trial.
Milan, ItalyAges 4 years+ - NCT06219863Recruiting
Harness walking help for babies with Down syndrome
This study tests if a special harness can help babies with Down syndrome learn to walk earlier. It is designed for infants under 2 years old who can sit but are not yet walking.
Boston, MassachusettsAges Up to 2 years - NCT07723560Recruiting
Surgery study for hemophilia A patients using Altuvoct
This study looks at how people with hemophilia A do when they have surgery while using the medicine Altuvoct (efanesoctocog alfa). It helps doctors learn more about surgical practices for this condition.
BronAges Any age - NCT05919264RecruitingPhase 1/Phase 2
FOG-001 for advanced solid tumors with certain gene changes
This trial tests a new drug, FOG-001, alone or with other treatments, for people with advanced solid tumors that have specific changes in the WNT pathway genes. It aims to see if the drug can shrink tumors or slow their growth.
Phoenix, ArizonaAges 18 years+ - NCT07723248RecruitingPhase 3
Oral Rinzimetostat for Advanced Prostate Cancer After Abiraterone
This study tests an experimental pill called rinzimetostat (ORIC-944) for men with advanced prostate cancer that has spread and stopped responding to abiraterone. It may offer a new option if you have not had many other treatments yet.
Charlotte, North CarolinaAges 18 years+ - NCT07724171Recruiting
Long-term results after PFO closure surgery
This study looks at how well patients do over time after having a hole in their heart (patent foramen ovale, or PFO) closed with a small device inserted through a vein. It may help doctors understand the benefits and risks of the procedure.
SeoulAges 19 years+ - NCT06921720Recruiting
Phosphate diabetes muscle study using phosphorus scan
This study uses a special MRI scan to look at phosphorus levels in the muscles of people with phosphate diabetes (a condition where the body loses too much phosphate through the kidneys). It aims to better understand how this condition affects muscles and may help guide future treatments.
BronAges 10 years+ - NCT02744677Recruiting
Pulmonary valve procedure to improve blood flow issues
This trial studies a transcatheter pulmonary valve (a valve placed through a catheter) in people whose right-sided outflow tract valve/conduit is not working well. It aims to improve blood flow and reduce pressure problems, while checking safety and results after the procedure.
Birmingham, AlabamaAges Any age - NCT07483255Recruiting
Fetal embolization trial for vein of Galen malformation
This trial tests a fetal procedure to treat a rare brain blood vessel condition called vein of Galen malformation. The goal is to improve the baby's outcome by closing off abnormal connections inside the brain before birth.
Boston, MassachusettsAges 18 years+ - NCT07496463Enrolling by invitationPhase 2
Setmelanotide for weight loss in pseudohypoparathyroidism type 1a
This study tests an experimental medication called setmelanotide to see if it can help with weight loss in people who have pseudohypoparathyroidism type 1a (PHP1a), a rare genetic condition that often leads to obesity. The goal is to find a new treatment option for weight problems related to PHP1a.
Boston, MassachusettsAges Any age - NCT07698197Recruiting
Exercise study for immune system problems
This study tests if a moderate exercise program can help people with certain antibody deficiencies. It aims to see if exercise improves immune function in people who are not taking immunoglobulin therapy.
Milford, ConnecticutAges 18–65 - NCT07491172RecruitingPhase 1
Trial of CTX310 for stubborn high cholesterol and fats
This trial tests a new medicine, CTX310, for people with persistently high levels of fats (triglycerides) and cholesterol that have not improved with standard treatments. It aims to see if the drug is safe and tolerable.
Hialeah, FloridaAges 18–75 - NCT07616154RecruitingPhase 2
Gene-Matched Family Stem Cell Transplant for Severe Sickle Cell
This trial tests whether a stem cell transplant from a partially gene-matched family member can cure severe sickle cell disease in children and young adults. The transplant uses a new approach that may work even when a perfectly matched sibling isn't available, and it might help patients whose sickle cell is causing serious complications like strokes, organ damage, or frequent pain crises.
Memphis, TennesseeAges Up to 22 years - NCT07137637RecruitingPhase 1
HPB-092 for relapsed or refractory AML
This trial tests a new drug, HPB-092, in adults with acute myeloid leukemia that has come back or not responded to treatment. The goal is to see if it can help when other treatments have not worked.
TianjinAges 18 years+ - NCT07537075Enrolling by invitationPhase 2/Phase 3
Continuing SKY-0515 treatment for Huntington's disease
This is a follow-up study for people with Huntington's disease who already completed an earlier SKY-0515 trial and tolerated it well. Researchers want to see how safe and effective the drug continues to be with longer-term use.
New Lambton Heights, New South WalesAges 25 years+ - NCT07438405RecruitingPhase 2
Long-term safety study of AMG 732 for thyroid eye disease
This trial tests the long-term safety and effectiveness of AMG 732 for people with moderate-to-severe thyroid eye disease who have already completed an Amgen study of this drug. It allows continued treatment without needing immediate eye surgery.
Little Rock, ArkansasAges 18–99 - NCT06899204Recruiting
Abrocitinib for atopic dermatitis after biologic therapy
This study looks at how well abrocitinib (a daily pill) works for people with moderate to severe eczema who have already tried biologic medicines and didn't get enough relief. It aims to see if this treatment can help you better manage your eczema.
San Diego, CaliforniaAges 12 years+ - NCT01238250Recruiting
Online autism genetics study for families with certain genetic changes
This study asks people with specific genetic changes linked to autism and their family members to complete online tasks and questionnaires. It may help researchers better understand how these genetic differences relate to autism traits.
Boston, MassachusettsAges Any age - NCT05142189RecruitingPhase 1
Testing BNT116 for advanced non-small cell lung cancer
This trial tests an experimental immunotherapy called BNT116, given alone or with other drugs, for people with advanced non-small cell lung cancer (NSCLC). The goal is to see if it is safe and if it can help shrink tumors.
Lexington, KentuckyAges 18 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,863 recruiting medical genetics studies from the U.S. registry right now, out of 5,730 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.