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
- NCT04258787Recruiting
Eye drop treatment for Fuchs corneal swelling
This trial studies a treatment for people with Fuchs’ dystrophy that affects the clear layer at the back of the cornea. It may help improve how clear your vision is after the cornea can no longer pump fluid out properly.
Portland, OregonAges 18 years+ - NCT04265040Recruiting
Heart muscle disease registry for non-ischemic conditions
This trial is a registry, meaning it collects health information to help doctors study heart muscle diseases and plan future cardiovascular studies. You may be able to join if you have a non-ischemic cardiomyopathy such as dilated cardiomyopathy, certain forms of thick heart muscle, non-compaction, or arrhythmogenic right-sided disease.
Heidelberg, Baden-WurttembergAges 18–80 - NCT04270045Recruiting
Breathing test in babies with bronchopulmonary dysplasia
This trial studies a breathing measurement test (forced oscillometry) in babies who were born early and have bronchopulmonary dysplasia (BPD), a long-term lung condition. It may help doctors better understand how babies’ lungs are working and guide future care.
Kansas City, MissouriAges Any age - NCT04272697Recruiting
Registry study for familial cholesterol conditions
This trial is a global registry that collects health and genetic information from people with familial hypercholesterolemia (FH) and sometimes from their relatives. The goal is to better understand FH and how it presents, which can help improve care for families affected by high cholesterol.
LondonAges Any age - NCT04278131RecruitingPhase 1/Phase 2
Test eye gene therapy for retinitis pigmentosa
This Phase 1/2 study tests a gene therapy treatment (BS01) in people with retinitis pigmentosa to try to improve or preserve vision. You may be a candidate if your vision in at least one eye is quite limited and you haven’t had certain gene therapies before.
Teaneck, New JerseyAges 18 years+ - NCT04280510Recruiting
Study of immune-related gut illnesses in adults
This study looks at adults with immune-related stomach and intestine problems to better understand what may cause them. If you qualify, you can share information and consent to help researchers learn how these conditions work.
CréteilAges 18 years+ - NCT04281056Recruiting
Tooth loss and dentures: taste and food preferences
This study looks at how losing teeth and wearing dentures affects your mouth sensations and your food choices. It compares people who are fully without teeth (and use dentures) with people who still have their natural teeth.
ParisAges 18–79 - NCT04289571RecruitingPhase 1
Virtual reality vision testing for people with retinal disease
This early-stage study uses virtual reality tasks to check how well a person can move and function using their eyesight. It may help researchers measure vision abilities in a way that could guide future care for retinal disease.
Bethesda, MarylandAges 5–120 - NCT04294043RecruitingPhase 1
Gallium IV study for adults with cystic fibrosis and NTM lung infection
This Phase 1 study tests whether an IV medicine called gallium can treat hard-to-treat lung infections (NTM) in adults with cystic fibrosis. It is mainly for people who have not been treated yet for NTM or who have NTM that has not cleared despite antibiotics.
Birmingham, AlabamaAges 18 years+ - NCT04299828Recruiting
Study of inflammation activity during complex heart surgery
This study measures how active white blood cells and inflammation are during complex heart surgery using a heart-lung machine. It may help doctors better understand what triggers inflammation during surgery and how to improve care.
Boston, MassachusettsAges Any age - NCT04310098Recruiting
CADASIL registry for confirmed patients and mutation carriers
This study builds a group “registry” of people affected by CADASIL (a genetic brain blood-vessel condition) and people who carry the NOTCH3 mutation, even if they have no symptoms. It helps researchers learn more about the condition over time and compare experiences between patients, carriers, and healthy relatives or controls.
Fuzhou, FujianAges 18–85 - NCT04308330RecruitingPhase 1
Vorinostat plus chemotherapy for advanced tumors in brain
This Phase 1 trial studies how safe and tolerable vorinostat is when combined with chemotherapy for cancers that have come back or are hard to treat, including tumors in the brain or spinal cord. It may help doctors find a better way to treat advanced solid tumors and CNS (brain/spinal cord) cancers.
Valhalla, New YorkAges 1–30 - NCT04319848RecruitingPhase 1
Tissue-engineered cornea transplant for specific eye swelling conditions
This Phase 1 trial tests a new, lab-made tissue transplant to improve corneal clarity in people with certain types of corneal endothelium failure (eye swelling) caused by specific conditions. The main goal is to check safety and see if vision and corneal health improve.
SingaporeAges 21–80 - NCT04334031Recruiting
Hospital follow-up study for immune-related disease
This is a study that follows people with an immune-related illness at Lille University Hospital to better understand their care needs over time. By collecting information regularly, the study may help improve how future patients are monitored and treated.
LilleAges 18 years+ - NCT04336644Recruiting
Checks heart rhythm continuously or intermittently during treatment
This study compares two ways of monitoring your heart rhythm while you start certain cancer treatments. It may help doctors detect heart rhythm changes earlier and decide which monitoring approach is better.
St Louis, MissouriAges 18 years+ - NCT04338048Recruiting
Study for autosomal dominant polycystic kidney disease
This study enrolls people who have been diagnosed with autosomal dominant polycystic kidney disease (ADPKD), a genetic kidney condition that can cause kidney cysts. It aims to learn about this specific form of the disease so better care is possible for the right patients.
Washington D.C., District of ColumbiaAges Up to 18 years - NCT04339699Recruiting
PFO closure comparison trial for cryptogenic stroke
This trial studies ways to close a patent foramen ovale (PFO) to help prevent another stroke in people whose prior stroke had an unknown cause. If you qualify, you may receive one of the PFO closure approaches the study is comparing.
Tucson, ArizonaAges 18–60 - NCT04342026Recruiting
Study environment and hormones in undescended testicles
This study looks at whether environmental exposures and hormone disruption are linked to undescended testicles in children. It may help researchers better understand causes and risk factors, which could improve future care for affected families.
MontpellierAges 1 month–18 years - NCT04342234Recruiting
Neural network tool to measure cleft palate from scans
This trial studies a computer “neural network” that uses existing dental impressions, scans, and photos to measure cleft palate features. The goal is to reduce the amount of repeated cleft lip and palate clinic work for patients and families.
BaselAges Any age - NCT04344769Recruiting
Studying how a kidney-protecting pathway works in ADPKD
This study looks at how the body’s “Nrf2 response” works in people with autosomal dominant polycystic kidney disease (ADPKD). It may help researchers better understand disease biology and future treatments by comparing kidney-related signals in ADPKD patients and healthy volunteers.
Rochester, MinnesotaAges 18–30 - NCT04356326RecruitingPhase 3
Aspirin to prevent complications in pregnancy with chronic high blood pressure
This trial studies whether taking acetyl salicylic acid (aspirin) during pregnancy can reduce problems caused by chronic high blood pressure. It focuses on pregnant people already known to have high blood pressure before or early in pregnancy.
BordeauxAges 18 years+ - NCT04365998Recruiting
Testing a new light device for newborn jaundice
This study tests a new phototherapy (special light treatment) device to treat newborn jaundice. It may help by seeing whether this device can treat jaundice safely and effectively.
LilleAges Up to 4 weeks - NCT04367246Recruiting
TP53 (Li-Fraumeni) family biobank and sample study
This study collects and stores health information and biological samples from people who have a known TP53 change (Li-Fraumeni syndrome) and from close family members. The goal is to help researchers learn more about risks and future treatments for this genetic condition.
Philadelphia, PennsylvaniaAges Any age - NCT04370899Recruiting
Early checkup for inherited high cholesterol in children
This study looks at children who may have inherited high cholesterol, especially when one parent is affected. It may help doctors detect the condition earlier and understand how it runs in families.
Reus, TarragonaAges 2–18
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.