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
- 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 - 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+ - 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 - NCT07154550Recruiting
Comparing Mastisol bandage for clubfoot casting
This study compares two types of sticky bandage (Mastisol vs. standard) used during Ponseti casting for babies with clubfoot in both feet. It aims to see which one works better for keeping the casts on and preventing skin problems.
Madison, WisconsinAges birth–3 months - NCT07151274Recruiting
Ehlers-Danlos syndrome and women's gynecological health
This study uses an online questionnaire to explore how Ehlers-Danlos syndrome (EDS) may affect gynecological health. It compares women with EDS to those without, to better understand any differences.
RouenAges 18–50 - 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 - NCT07152197Recruiting
Resistance Exercise for Charcot-Marie-Tooth Disease
This trial tests whether resistance exercises (like lifting weights with your legs) can help people with Charcot-Marie-Tooth disease. It is for young adults aged 15 to 29 who can move their ankle enough to do strength training.
Temuco, La AraucaníaAges 15–29 - NCT07148050RecruitingPhase 1
CAR T-cell therapy for children with solid tumors
This trial tests a new kind of immunotherapy for children and teens with solid tumors that have not responded to standard treatments. The therapy uses specially engineered T cells (CAR T cells) that target a protein called GPC3 found on many tumor cells, and it includes extra signals (IL-15 and IL-21) to help the T cells work better.
Seattle, WashingtonAges 1–26 - NCT07148739RecruitingPhase 4
Ensuring Best Access to CF Treatment
This study tests whether people with cystic fibrosis who are already on a triple combination CFTR modulator can stay on their treatment without interruptions or problems. It aims to make sure everyone gets the best, most consistent treatment for their CF.
Birmingham, AlabamaAges 3 years+ - NCT07145138Recruiting
Multi-ethnic dilated cardiomyopathy registry
This study is a registry for people with dilated cardiomyopathy (DCM)—a condition where the heart's main pumping chamber becomes enlarged and weak. It aims to learn more about DCM, especially in people from different ethnic backgrounds, by collecting health information over time.
Chicago, IllinoisAges 18 years+ - NCT07148583Recruiting
BioAmicus Complete for tummy troubles in babies
This study tests if a probiotic supplement called BioAmicus Complete can help babies with common digestive issues like colic, reflux, constipation, or loose stools. It might be a good option if your baby has these symptoms and you haven't used other probiotics or antibiotics recently.
Haiphong, Hai PhongAges birth–2 years - NCT07154719RecruitingPhase 4
Testing a weight loss drug's effect on muscle and bone
This study tests a medication called tirzepatide (a type of GLP-1 drug) to see how it affects muscle and bone health during weight loss. It is for people with obesity who are not menopausal and do not have diabetes or bone disease.
Baton Rouge, LouisianaAges 18–50 - NCT07152106Recruiting
Amniotic fluid and the preterm baby's gut
This study looks at the fluid around your baby (amniotic fluid) and how it might affect your baby's gut health if they are born early. It may help doctors understand how to better care for preterm infants.
AmsterdamAges 16 years+ - NCT07150000Recruiting
Rheumatic disease research study for patients and healthy volunteers
This study is for people with certain rheumatic conditions (like arthritis, lupus, or vasculitis) and for healthy volunteers. It aims to learn more about these diseases and improve how they are treated.
Bonn, North Rhine-WestphaliaAges 18 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+ - NCT04776824Recruiting
Swiss registry for people with amyloidosis and possible heart involvement
This trial is a registry that collects health information from people with amyloidosis, including cases where the heart may be involved. Sharing your medical details can help researchers learn how this condition affects patients and may guide future care.
BaselAges 18 years+ - NCT05970965Recruiting
Teeth and gum study in children with Down syndrome
This study looks at how gums and inflammation work in children with Down syndrome by collecting and analyzing biological samples. It may help researchers understand gum health differences and improve future care.
Ivry-sur-SeineAges 3–12 - NCT06582875RecruitingPhase 2
GLP-1 drug for polycystic kidney disease and weight
This study tests a GLP-1 receptor agonist (a type of medicine that helps with weight and metabolism) in people with polycystic kidney disease who are overweight. It aims to see if this drug can help slow kidney disease progression.
Aurora, ColoradoAges 18–65 - NCT06952400Recruiting
Melanoma genetics study in Taiwan
This study looks at genetic changes in melanoma tumors to better understand the disease. It may help find the best treatments for different stages of melanoma.
Taipei, Taiwan/TaipeiAges 18 years+ - NCT06958978Recruiting
Quantum probe for skin tightening in liposculpture patients
This trial tests a new device called the QUANTUM Probe, which is used during liposculpture to tighten skin and improve results. It may help people who are already planning body contouring procedures.
Bogotá, DCAges 18–60 - NCT06081075Recruiting
Newborn Genomics Program for Sick Infants
This study looks at using rapid DNA testing (genomics) in newborns and children who are very sick in the hospital (NICU or PICU). The goal is to see if finding a genetic cause early can help guide treatment and improve outcomes.
AucklandAges birth–2 years - NCT07137338RecruitingPhase 1
Gene therapy for BAG3-related dilated cardiomyopathy
This trial tests a one-time gene therapy called RP-A701 for people with dilated cardiomyopathy caused by a BAG3 gene mutation. The goal is to see if it is safe and can improve heart function.
San Diego, CaliforniaAges 18–65 - 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+ - NCT07140614RecruitingPhase 1
Study of EDK060 for adults with CMT1A
This trial tests a new medicine called EDK060 for people with Charcot-Marie-Tooth disease type 1A (CMT1A). The study will check if the drug is safe and how the body processes it, with the goal of finding better treatments for this condition.
Ottawa, OntarioAges 18–60
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.