Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,859 recruiting studies in this area right now.
By phase
- Not applicable2,773
- Phase 2283
- Phase 1236
- Phase 3215
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis128
- Congenital Heart Disease85
- 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
- Pennsylvania66
- Ohio57
Recruiting studies3,859 total
- NCT05454332RecruitingPhase 4
Caffeine for premature babies right after birth
This study tests whether caffeine given to very premature babies around the time they move from fetus to newborn stage can improve outcomes. It may help babies who need support as they breathe and adapt after birth.
Porto Alegre, Rio Grande do SulAges Up to 2 hours - NCT05454774RecruitingEarly Phase 1
Gene therapy for adults with severe hemophilia A
This early-phase study tests an FVIII gene therapy to help your body make more of the missing clotting factor in hemophilia A. It’s aimed at adults with severe low FVIII levels who have already used FVIII replacement products for a long time and have not developed an immune blocker (an inhibitor).
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT05456321Recruiting
Brain stimulation and thinking training for prenatal alcohol effects
This study tests whether brain stimulation plus structured thinking practice can improve thinking and brain function in people affected by heavy alcohol exposure before birth. It may help parents and researchers learn how to support learning and daily functioning in fetal alcohol spectrum disorders.
Minneapolis, MinnesotaAges 8–17 - NCT05461222Recruiting
Balloon treatment before birth for severe diaphragmatic hernia
This study looks at placing a small balloon in a baby’s windpipe before birth to help severe congenital diaphragmatic hernia (CDH). The goal is to improve lung growth and birth outcomes by testing whether this timing and method can help babies with the most severe cases.
Stanford, CaliforniaAges 18–50 - NCT05462106RecruitingPhase 1/Phase 2
ACI-24.060 study for early Alzheimer’s in adults and Down syndrome
This Phase 1/2 study tests an Alzheimer’s treatment called ACI-24.060 to see if it affects the disease in people with early Alzheimer’s changes. It includes two groups: older adults with early symptoms and people with Down syndrome who also show Alzheimer’s-related changes on brain scans.
Phoenix, ArizonaAges 35–85 - NCT05463133RecruitingPhase 1/Phase 2
Stem cell transplant for CGD using targeted medications and steroids-free support
This trial tests an intensive stem cell transplant approach for people with chronic granulomatous disease (CGD). The study aims to improve outcomes by preparing the body for a donor stem cell graft and using immune “brakes” to reduce immune problems.
Bethesda, MarylandAges 4–65 - NCT05463757Recruiting
Oral drugs for advanced or multiple basal cell skin cancer
This study registers people in the Netherlands who have certain types of basal cell skin cancer and take an oral hedgehog-blocking medicine. It may help doctors understand how well these tablets work and how patients do in real life.
Nijmegen, GelderlandAges Any age - NCT05468580Recruiting
Release or sham surgery for clogged celiac artery
This trial studies whether a surgical “release” of the celiac artery (to improve blood flow) helps people with MALS who have specific symptoms and artery narrowing. Some people will have a sham (placebo) procedure to compare outcomes, so you’ll be closely monitored either way.
Enschede, OverijsselAges 18 years+ - NCT05469373Recruiting
Testing ESIS for children with hard-to-treat epilepsy
This trial studies ESIS in children and teens (ages 1 to 30) who have epilepsy that has not been controlled by medications. It may help by improving how doctors evaluate and treat seizures, especially when an additional brain-monitoring test is planned.
Cincinnati, OhioAges 1–30 - NCT05470920Recruiting
Genetic testing help tool for ovarian or pancreatic cancer
This study tests a decision aid that helps people with ovarian or pancreatic cancer understand and decide about genetic (inherited) testing. It may help you make a clearer choice about next steps for yourself and your family.
Boston, MassachusettsAges 18 years+ - NCT05473637Recruiting
Study causes of small vessel brain disease in stroke and memory issues
This trial looks at people who may have “small vessel” problems in the brain, which can cause stroke, memory trouble, walking changes, movement symptoms, or headaches. It may help researchers better understand the genetic and non-genetic causes, which could improve future diagnosis and treatment.
TaipeiAges 18 years+ - NCT05477563RecruitingPhase 3
One-time gene therapy for severe sickle cell and transfusion anemia
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
New York, New YorkAges 12–35 - NCT05477680Recruiting
Measuring brain shift during surgery using navigation
This study tests whether a special brain-navigation method can better measure how the brain moves during surgery for certain brain conditions. If it works, it may help surgeons avoid mistakes caused by brain shifting while you are under anesthesia.
MoscowAges 18–100 - NCT05478083RecruitingPhase 2
Leuprolide treatment for severe polycystic liver growth
This Phase 2 study tests whether a monthly hormone medicine (leuprolide, a GnRH agonist given as an injection) can slow down ongoing, fast-growing polycystic livers. It is aimed at pre-menopausal women with very large livers that are still getting bigger on past and current scans.
Nijmegen, GelderlandAges 18–45 - NCT05481879RecruitingPhase 1/Phase 2
Study medicine for DM1 to improve muscle function
This early-stage study tests DYNE-101 to see how safe it is and whether it can improve measurable muscle function in people with myotonic dystrophy type 1 (DM1). You may be asked to do several walking and strength tests and undergo heart and breathing screening measurements.
Stanford, CaliforniaAges 18–65 - NCT05484245Recruiting
Ultrasound-guided surgery for certain brain mass lesions
This trial looks at using real-time ultrasound to help surgeons remove or treat certain brain growths and bleeding areas. It may help by making the procedure more precise for newly diagnosed cases in the upper part of the brain.
MoscowAges 18–100 - NCT05484570Recruiting
Study of how rare DNA repair disorders affect the body over time
This natural history study follows people with specific DNA repair disorders to better understand symptoms and how they change over time. It may help researchers plan future treatments by mapping what complications to expect and when.
Minneapolis, MinnesotaAges 6 months+ - NCT05489549Recruiting
Study of heart amyloid risk in people with TTR gene variants
This study looks at people who carry certain TTR gene variants, which can cause a specific type of heart amyloid buildup. It also includes people with symptoms, to understand how the condition develops and how it may be monitored.
New York, New YorkAges 30–80 - NCT05491525RecruitingPhase 2/Phase 3
Vibegron for children with nerve bladder using intermittent catheter
This study tests how well and how safely vibegron works long-term in children (ages 2 to under 18) with a nerve-related bladder condition, who regularly use intermittent catheterization. It also tracks how the body processes the medicine and whether it helps bladder control over time.
Little Rock, ArkansasAges 2–17 - NCT05492708Recruiting
Pregnancy study for first-time-to-mid pregnancy participants in Heartland states
This study is enrolling pregnant people who are early in pregnancy and live in certain Heartland states. It aims to learn from participants during pregnancy and may help researchers better understand pregnancy care needs.
Indianapolis, IndianaAges 18 years+ - NCT05493605Recruiting
MRI study of heart involvement in Wilson disease
This study looks at whether Wilson disease affects the heart, using MRI scans (and possibly contrast dye). Your results may help doctors better understand cardiac (heart) involvement and how it progresses in Wilson disease.
ParisAges 18 years+ - NCT05495776Recruiting
Study checks how often Lynch syndrome happens in colon cancer
This registry study looks at how many people with colon cancer also have Lynch syndrome, an inherited condition that can affect cancer risk. If you enroll, your care team collects basic information from your diagnosis to better estimate how common it is.
MoscowAges 18 years+ - NCT05499091Recruiting
Study rare disease genetics using family and health data
This study tries to find genetic causes for rare diseases that are not yet understood. It uses a national rare-disease data bank and collects consent from patients and sometimes close family members.
AngersAges Any age - NCT05499741Recruiting
Heat-based therapy to improve sleep in adults with Tourette’s
This trial tests a forehead temperature-regulating therapy to help improve insomnia symptoms in adults who have Tourette’s (and significant tics). It may help you sleep better by testing whether changing forehead temperature can improve sleep quality and efficiency.
Los Angeles, CaliforniaAges 18–50
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,859 recruiting medical genetics studies from the U.S. registry right now, out of 5,745 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.