Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,756 recruiting studies in this area right now.
By phase
- Not applicable2,677
- Phase 2277
- Phase 1238
- Phase 3210
- Phase 1/Phase 2208
- Phase 497
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease134
- Atopic Dermatitis125
- Congenital Heart Disease84
- Cystic Fibrosis76
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy37
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)28
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
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
- Massachusetts General Hospital22
Where studies are running
- California325
- Maryland161
- Alabama133
- Massachusetts107
- Arizona86
- New York85
- Texas70
- Minnesota68
- Pennsylvania62
- Florida59
Recruiting studies3,756 total
- NCT06557369Recruiting
New light therapy for intermediate age-related macular degeneration
This trial tests a new light-based treatment for intermediate age-related macular degeneration (AMD), a common eye condition that can lead to vision loss. The goal is to see if it is safe and helps slow down the disease.
BergamoAges 50 years+ - NCT06551961Recruiting
How two fibers affect your gut health
This study tests how two types of fiber (inulin and methylcellulose) affect your gut when eaten regularly. You'll eat provided meals and have your breath tested to see how your gut breaks down the fiber.
Nottingham, NottinghamshireAges 18–65 - NCT07644715Recruiting
AI Analysis of Heart Rhythms and Immune-Related Heart Damage
This study uses artificial intelligence to analyze heart electrical recordings (ECGs) from existing patient databases to better detect dangerous heart rhythm problems and heart inflammation caused by cancer immunotherapy. Researchers are not giving any new treatments—they're studying existing data to improve diagnosis.
ParisAges Any age - NCT07640503Enrolling by invitationPhase 2
Lamivudine Treatment Study for Rett Syndrome
This study tests whether lamivudine, a medication originally used for HIV, might help improve symptoms in people with Rett syndrome. Researchers will monitor how safe and effective the treatment is over time.
Fortaleza, CearáAges 2 years+ - NCT07642466Recruiting
Bladder Health and Life Quality in Spina Bifida
This study asks adults and teens with spina bifida about how their bladder care affects their daily life and well-being. Your answers will help doctors better understand what matters most to patients.
London, OntarioAges 13 years+ - NCT07644078Recruiting
Understanding Cavovarus Foot Deformity in Nerve Disorders
This study examines how nerve disorders like Charcot-Marie-Tooth disease cause a specific type of foot deformity (cavovarus feet, where the foot has a high arch and turned-in appearance). Researchers will either examine you directly or review your existing medical records to better understand this condition and how it affects people.
StanmoreAges 18 years+ - NCT07645898Recruiting
Glucose monitoring study of cornstarch in glycogen storage disease
This study uses a glucose monitor (a small sensor you wear) to measure how your blood sugar responds after eating raw cornstarch, a common treatment for glycogen storage disease type I. The goal is to understand how well cornstarch helps keep your blood sugar steady.
Naples, NapoliAges 18 years+ - NCT07643844RecruitingPhase 1
Gene therapy for propionic acidemia in infants
This trial tests a new gene therapy treatment designed to help the body process certain amino acids better in infants with propionic acidemia. The therapy uses a modified virus to deliver a working copy of a faulty gene, potentially reducing dangerous acid buildups and hospital visits.
Rochester, MinnesotaAges 6 months–2 years - NCT04117685Recruiting
Global hip dysplasia registry for young children
This trial is a worldwide registry that collects information from children with hip dysplasia or who are at higher risk. It helps researchers understand who is affected and how hip evaluations are done, which can improve future care.
Vancouver, British ColumbiaAges 1 minute–10 years - NCT06267508Recruiting
HIV testing and treatment for newborns
This study looks at ways to test and treat newborns for HIV early to improve their long-term health. It is for mothers with HIV whose babies are less than 3 days old and who are willing to have their babies tested and followed at home.
BeiraAges 18 years+ - NCT04880356Recruiting
Study of very rare inherited brain diseases over time
This study follows adults over time who have a very rare inherited brain disease, or whose illness seems like it may run in families. The goal is to better understand these conditions and how they progress.
Milan, MilanoAges 18 years+ - NCT07633184Recruiting
Lung Ultrasound to Predict Breathing Problems in Premature Babies
This study uses ultrasound images of the lungs to see if doctors can predict which very premature babies will develop bronchopulmonary dysplasia (BPD)—a chronic lung condition common in extremely premature infants. The goal is to identify at-risk babies early so they can receive targeted treatment.
ModenaAges Up to 7 months - NCT07629778RecruitingPhase 1/Phase 2
HL-300 Ointment for Mild-to-Moderate Atopic Dermatitis
This study tests a new ointment (HL-300) designed to treat mild-to-moderate atopic dermatitis (a chronic skin condition causing itching and inflammation). You would apply it to affected skin areas to see if it safely reduces symptoms.
Hangzhou, ZhejiangAges 18–75 - NCT07629999RecruitingPhase 1
Safety study of experimental drug BMS-986533 in healthy people
This early-stage study tests how a new experimental drug (BMS-986533) is processed and tolerated by healthy volunteers. Researchers will also check how food and stomach acid affect the drug, and whether it interacts with other medications.
Lincoln, NebraskaAges 18–55 - NCT07637188Recruiting
PLLA Injections for Loose Skin After Weight Loss Surgery
This trial tests injections to tighten and lift loose, sagging skin that develops after bariatric (weight loss) surgery. The injections use a substance called PLLA to stimulate your body's natural skin-rebuilding process.
SienaAges 18–60 - NCT07636811RecruitingPhase 2
Mivelsiran for Early Alzheimer's in Down Syndrome
This trial tests a new drug called mivelsiran designed to slow early-stage Alzheimer's disease in adults with Down syndrome. The drug works by targeting amyloid, a protein that builds up in the brain and causes cognitive decline.
Maitland, FloridaAges 40–55 - NCT07630207Recruiting
Blood vessel health in adults with Down syndrome
This study looks at how blood vessels work in adults with Down syndrome compared to those without Down syndrome. It aims to understand heart health better and may help improve future care.
Las Vegas, NevadaAges 18–35 - NCT07635030Enrolling by invitation
Brain Chemistry Study in Young People with Friedreich's Ataxia
This study looks at two brain chemicals (GABA and glutathione) in children and teens aged 8–15 with Friedreich's ataxia, a rare genetic condition affecting movement and coordination. The research aims to understand how these chemicals change in the brain and may help guide future treatments.
Philadelphia, PennsylvaniaAges 8–16 - NCT07637084Recruiting
Light therapy for chronic pain and fatigue in Ehlers-Danlos Syndrome
This trial tests whether a non-invasive light therapy called photobiomodulation can reduce chronic pain and fatigue in people with hypermobile Ehlers-Danlos Syndrome (hEDS), a genetic condition affecting connective tissue. The light is applied to the skin and may help cells produce energy and reduce inflammation.
Boulogne-Billancourt, Île-de-France RegionAges 18 years+ - NCT07629986Recruiting
How antibiotics work in cystic fibrosis patients on ETI therapy
This study measures how antibiotics are processed by the body in people with cystic fibrosis who are taking a combination drug called elexacaftor/tezacaftor/ivacaftor (ETI). Understanding this helps doctors prescribe the right antibiotic doses for patients on this newer treatment.
RoscoffAges 12 years+ - NCT07630389Recruiting
Remote monitoring for childhood myotonic dystrophy
This study uses video calls and online assessments to track how myotonic dystrophy (a muscle-weakening condition) affects children over time. Researchers will also look at genetic factors to better understand the disease and improve care.
Rochester, New YorkAges birth–17 years - NCT07629635RecruitingPhase 3
Closing a heart hole to treat migraines
This trial tests whether closing a small hole in the heart (called a PFO) can help eliminate migraines. If you have frequent migraines and no other major health issues, this procedure might reduce or stop your headaches.
New York, New YorkAges 18–55 - NCT06250257RecruitingPhase 3
Bromocriptine for Heart Weakness in Women 18-50
This trial tests a medicine called bromocriptine to see if it helps women with a weak heart muscle (dilated cardiomyopathy). It is for women between 18 and 50 whose heart weakness was found recently or is caused by blocked arteries, and who are not pregnant or planning pregnancy.
Jimma, OromiyaAges 18–50 - NCT06694103Recruiting
Two ways to reduce pain during a baby's eye exam
This study tests two drug-free methods to help reduce pain when premature babies get a special eye exam for retinopathy of prematurity. It may help your baby feel more comfortable during the procedure.
Izmir, MenemenAges 6 months–8 months
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,756 recruiting medical genetics studies from the U.S. registry right now, out of 5,439 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.