Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,856 recruiting studies in this area right now.
By phase
- Not applicable2,770
- Phase 2282
- Phase 1235
- Phase 3215
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 353
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis129
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome54
- 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
- California340
- Maryland161
- Alabama132
- Massachusetts116
- New York84
- Arizona79
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,856 total
- NCT07152392RecruitingPhase 4
Testing IBI311 for inactive thyroid eye disease symptoms
This trial tests a medication called IBI311 for people with inactive thyroid eye disease that still causes bothersome symptoms like double vision or trouble closing the eyes. The goal is to see if it helps improve these problems safely.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT07152366RecruitingPhase 4
Hormone and tetuzumab for active thyroid eye disease
This trial tests a new sequence of hormone therapy followed by a targeted drug (tetuzumab) for people with moderate to severe thyroid eye disease that is still active after steroid treatment didn't help enough.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT07152340RecruitingPhase 4
Sequential hormone and IBI311 therapy for active TAO
This trial tests a two-step treatment for active, moderate-to-severe thyroid eye disease (TAO). You will first receive hormone therapy, followed by a drug called IBI311. The goal is to see if this sequence works better for people who have not had steroid treatment before.
Changhua, Shanghai MunicipalityAges 18–80 - NCT07152834Recruiting
Screening for Alpha-1 Antitrypsin Deficiency in Lung Blockage
This study screens for a rare genetic condition called Alpha-1 Antitrypsin Deficiency in people with lung blockage. It aims to find out if this deficiency is a hidden cause of your breathing problems, which could lead to better treatment options.
Muğla, MenteşeAges 18 years+ - NCT07154381Recruiting
Hybrid PET/MRI for heart amyloidosis imaging
This study uses a hybrid PET/MRI scanner to take detailed images of the heart in people with amyloidosis. The goal is to see how well this scan shows the buildup of amyloid protein, which could help doctors better diagnose and manage the condition.
Essen, North Rhine-WestphaliaAges 40 years+ - NCT07154394Recruiting
Heart amyloid and function in Alzheimer's disease
This study examines whether amyloid plaques in the heart affect heart function in people with Alzheimer's disease. It uses brain amyloid PET and MRI scans to explore a potential link.
Essen, North Rhine-WestphaliaAges 18 years+ - NCT07154667Recruiting
Testing a new ear reconstruction system in children
This trial is testing a new device called the Auryzon EAR 2.0 System to help rebuild ears in children and teens who have a deformity from birth or injury. The goal is to see if it works well for patients having surgery at Nemours Children's Hospital in Florida.
Orlando, FloridaAges 5–20 - NCT07156318Recruiting
Drumming lessons for children with Down syndrome
This study tests whether drumming lessons can help children with Down syndrome. It may help with coordination, focus, or mood.
Nashville, TennesseeAges 7–15 - NCT07157254RecruitingPhase 2
Study of GTX-102 for People with Angelman Syndrome
This study tests an experimental drug called GTX-102 for people with Angelman syndrome. It aims to see if the drug is safe and can help with symptoms. The trial includes different age groups and genetic types of Angelman syndrome.
Los Angeles, CaliforniaAges 1–64 - NCT07161908Recruiting
Remote oxygen monitoring for preterm infants with BPD
This study tests a way to monitor and adjust oxygen therapy from home for premature babies with a long-term lung condition called bronchopulmonary dysplasia. The goal is to see if remote monitoring can improve oxygen management and reduce complications.
České Budějovice, Czech RepublicAges Up to 8 months - NCT07162077Recruiting
A new treatment for white spots on front teeth
This trial tests a special technique (etch-bleach-seal) using Icon® to treat white or cloudy spots on permanent front teeth caused by a condition called Molar Incisor Hypomineralization (MIH). This approach may improve the appearance of these spots without drilling or filling.
LatakiaAges Any age - NCT07163403RecruitingPhase 1
Preventing cancer in Lynch syndrome carriers with dendritic cell vaccine
This trial tests whether a personalized vaccine made from your own immune cells can help prevent cancer in people with Lynch syndrome. You must have no history of cancer and be willing to have regular colonoscopies.
Barcelona, BarcelonaAges 18 years+ - NCT07163780Recruiting
Home Therapy Program for Babies With Down Syndrome
This study tests a special therapy program called HEP (home early intervention program) for babies with Down syndrome. It looks at whether this program can help your baby's development.
Istanbul, BeykozAges 4 months–1 year - NCT07166172Recruiting
Long-term outcomes after fetal surgery for severe CDH
This study follows pregnant women and their babies after a special fetal surgery (FETO) for severe diaphragmatic hernia (CDH). It aims to understand long-term health outcomes for both mother and child.
Baltimore, MarylandAges 18 years+ - NCT07167329RecruitingPhase 2
Belzutifan for VHL syndrome: a real-world study
This trial tests the drug belzutifan (a pill taken by mouth) in people with VHL syndrome who have kidney or other VHL-related tumors that are growing or can be measured. The study looks at how well belzutifan works in real-world conditions and whether certain genetic factors affect how the drug works.
São Paulo, São PauloAges 14 years+ - NCT07168434RecruitingPhase 3
Yeast supplement for irritable bowel syndrome (IBS)
This trial tests if a yeast supplement called Saccharomyces boulardii can help people with irritable bowel syndrome (IBS), except for those with constipation as their main symptom. Participants take the supplement and see if their symptoms improve.
BragaAges 18–65 - NCT07170176Recruiting
PFO closure and migraine: can it help?
This trial tests if closing a common heart defect (PFO) can improve migraine symptoms and brain function. It compares people with migraine who get the closure procedure to those without migraine.
Luoyang, HenanAges 18–60 - NCT07171905RecruitingPhase 2
Study of a new imaging agent for Von Hippel-Lindau disease
This trial tests a special imaging agent that targets a protein found in tumors related to Von Hippel-Lindau (VHL) disease. It may help doctors better detect and monitor these tumors.
Milan, ItaliaAges 18 years+ - NCT07172243Recruiting
Amycon biobank and data registry for amyloidosis
This study collects blood and tissue samples and health data from people with amyloidosis to build a biobank and registry. Researchers hope this will help improve future diagnosis and treatment.
LeuvenAges 18 years+ - NCT07172464Recruiting
Study of a new device to close a hole in the heart after a stroke
This trial tests a new device to close a PFO (a small hole between the upper chambers of the heart) in people who have had a stroke caused by a blood clot. The goal is to prevent future strokes by blocking the hole.
Los Angeles, CaliforniaAges 18–65 - NCT07175051RecruitingPhase 2
Testing a kidney-protecting drug in sickle cell disease
This study tests whether empagliflozin, a medicine that protects the kidneys in people with diabetes, can also protect the kidneys in people with sickle cell disease. The goal is to see if it can reduce protein in the urine, which is a sign of kidney stress.
Chicago, IllinoisAges 18–60 - NCT07177300RecruitingPhase 4
Hydroxyurea for sickle cell disease in children
This trial tests different ways to start hydroxyurea for children with sickle cell anemia. The goal is to find the safest and most effective approach to help manage symptoms and prevent complications.
Cincinnati, OhioAges 6 months+ - NCT07178873Recruiting
Online support program for adults with spina bifida
This study tests an online support program for adults with spina bifida to help with emotional and social well-being. It is done from home through video calls.
Bilbao, BizkaiaAges 18–75 - NCT07180355RecruitingPhase 1
Gene therapy trial for Friedreich's ataxia
This trial tests a gene therapy called SGT-212 for people with Friedreich's ataxia. It aims to help treat the condition by delivering a working copy of the gene.
Los Angeles, CaliforniaAges 18–40
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,856 recruiting medical genetics studies from the U.S. registry right now, out of 5,791 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.