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
- NCT06878950Recruiting
Early feeding for babies with gastroschisis
This study tests whether starting feedings earlier than usual helps babies born with gastroschisis recover better. It aims to find a safer and more effective feeding plan for these newborns.
Los Angeles, CaliforniaAges birth–1 year - NCT06868979Recruiting
Eye imaging for X-linked disorders
This study uses special eye imaging to look at changes in the brain and eyes of people with creatine transporter deficiency (a condition that affects brain energy) or fragile X syndrome. It aims to find a way to see these brain changes without needing a biopsy or spinal tap.
BronAges 5–60 - NCT06873035Enrolling by invitationPhase 2/Phase 3
Infigratinib for children with hypochondroplasia
This study tests an oral medication called infigratinib in children with hypochondroplasia, a form of short stature. It aims to see if the drug can help improve growth and is for children who are still growing.
Oakland, CaliforniaAges 3–18 - NCT06224413Recruiting
Study on eli-cel for cerebral adrenoleukodystrophy
This study follows people with cerebral adrenoleukodystrophy (CALD) who have been treated with eli-cel, a gene therapy. It tracks their long-term health to see how well the treatment works in everyday care.
Boston, MassachusettsAges Any age - NCT06563414Recruiting
A study tracking eye scrapes in people with DEB
This study follows people with dystrophic epidermolysis bullosa (DEB) who get scrapes on the surface of their eye (corneal abrasions). It collects information to better understand how often and how severely these eye injuries happen.
Pittsburgh, PennsylvaniaAges 6 months+ - NCT06865482Recruiting
Understanding heart disease in Friedreich's ataxia
This study looks at how heart disease progresses in people with Friedreich's ataxia, a genetic condition that affects coordination and heart health. By taking part, you help researchers better understand the condition, which may guide future treatments.
La Jolla, CaliforniaAges 6 years+ - NCT06858397RecruitingPhase 1/Phase 2
Study of a new drug for Fabry disease
This study tests a new investigational drug called HM15421/GC1134A for people with Fabry disease. It aims to see if the drug is safe and tolerable, and to learn how well it works. You may be eligible if you have Fabry disease with symptoms, have not had treatment for at least 6 months, and your kidneys are functioning well.
Los Angeles, CaliforniaAges 18 years+ - NCT06867107Enrolling by invitationPhase 2
Long-term follow-up study of SAT-3247 for Duchenne muscular dystrophy
This study follows people with Duchenne muscular dystrophy who were in an earlier SAT-3247 trial, or who meet certain other criteria, to see how the medicine works over a longer time. It aims to gather more safety and effectiveness information.
Los Angeles, CaliforniaAges 16–40 - NCT04704089Recruiting
Dental enamel defect study using tooth photos and measurements
This study compares different types of enamel problems by taking detailed pictures of your front teeth. It may help researchers better tell these enamel conditions apart and improve future diagnosis and care.
ParisAges 8–55 - NCT06864351Recruiting
Study of a new thyroid dosing method for children
This study tests a new way to adjust thyroid medication doses for children with either congenital hypothyroidism or Graves' disease. It aims to help find the right dose more easily and improve treatment outcomes.
YerevanAges Up to 18 years - NCT06862583Recruiting
Study of acute type A aortic dissection treatments
This trial is studying treatments for people with a sudden tear in the main artery from the heart (type A aortic dissection) or a similar condition. It aims to improve outcomes after this emergency.
StockholmAges 18 years+ - NCT06866925Recruiting
Brain stimulation for depression with catatonia in Down syndrome
This study tests whether a gentle brain stimulation treatment called tDCS can help people with Down syndrome who are also experiencing severe depression with symptoms like being very still or not speaking. The goal is to see if this safe, non-invasive treatment can improve mood and movement.
Lyon, Auvergne-Rhône-AlpesAges 18 years+ - NCT06861309Recruiting
Placental imaging study for pregnant women
This study uses special imaging techniques to look at the placenta during pregnancy. It aims to learn more about normal pregnancies and those where the baby is growing more slowly than expected.
Roanoke, VirginiaAges 18–45 - NCT06859411Recruiting
Posterior corneal transplant study
This study looks at how well people do after a specific type of corneal transplant called posterior lamellar keratoplasty. It aims to understand long-term outcomes for patients who had this surgery.
MetzAges 18 years+ - NCT06864039Enrolling by invitation
Quality of life in treated congenital hypothyroidism
This study looks at the long-term quality of life for people who were diagnosed with congenital hypothyroidism at birth and started treatment within the first month of life. It aims to understand how early treatment affects their health and well-being as they grow up.
NaplesAges 16–21 - NCT06859177Recruiting
Remote fetal monitoring trial for complicated pregnancies
This study tests if women with high-risk pregnancies can safely use a remote monitor at home instead of staying in the hospital. You would use a belly patch to send your baby's heart rate and your contractions to your care team.
Veldhoven, North BrabantAges 18 years+ - NCT05860569RecruitingPhase 1
Gene therapy safety study for inherited high triglycerides and pancreatitis
This Phase 1 trial tests the safety of a gene therapy for people with inherited, hard-to-control high triglycerides (fat in the blood) who have had pancreatitis episodes. It may help by targeting the genes involved in triglyceride control, but the main goal here is to check safety and tolerability.
Beijing, Beijing MunicipalityAges 18–60 - NCT06391736RecruitingPhase 1/Phase 2
Gene therapy for late-onset Pompe disease
This trial tests a new gene therapy for late-onset Pompe disease. The therapy aims to help your body produce the enzyme needed to break down glycogen, which may improve muscle and breathing function.
BeijingAges 6 years+ - NCT06421831RecruitingPhase 1/Phase 2
Gene therapy trial for spinal muscular atrophy type 3
This trial is testing a one-time gene therapy drug for people with SMA type 3. The goal is to see if it can safely improve muscle function and strength.
Beijing, Beijing MunicipalityAges 2 years+ - NCT06800651RecruitingPhase 2
A new drug for fast-growing polycystic kidney disease
This trial tests a drug (JMKX003142) for people with autosomal dominant polycystic kidney disease that is getting worse quickly. It may help slow down kidney damage.
Beijing, Beijing MunicipalityAges 18–55 - NCT06619197Recruiting
Reflexology in Sickle Cell Disease Study
This study tests if foot reflexology, a gentle pressure technique on your feet, can help relieve pain from a sickle cell crisis while in the hospital. It is only for children and teens under 18.
BronAges Up to 18 years - NCT06851767Enrolling by invitationPhase 1/Phase 2
Gene therapy for X-linked severe combined immunodeficiency
This study tests a gene therapy to treat X-linked severe combined immunodeficiency (X-SCID) without a stem cell transplant from a matched sibling. It aims to improve immune function and reduce infections using your own stem cells modified to carry the correct gene.
Bethesda, MarylandAges 3–99 - NCT06849037Recruiting
Preventive antibiotics for meconium-stained fluid at birth
This trial tests whether giving a single dose of the antibiotic Zinacef (cefuroxime) to women with meconium-stained amniotic fluid during labor can reduce infections. You may be eligible if you have a full-term, single baby and your water is stained with meconium.
NahariyaAges 18 years+ - NCT06850038Recruiting
Long-term study of odevixibat in Alagille syndrome
This study follows people with Alagille syndrome who are already taking or starting odevixibat (Bylvay). It looks at how well the medicine works and how safe it is over a long period of time.
Los Angeles, CaliforniaAges 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.