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
- NCT07373821Recruiting
Lying face up after cornea transplant surgery
This study tests if lying face up after DMEK surgery (a cornea transplant for Fuchs dystrophy) helps the surgery work better. It may be an option for people with Fuchs dystrophy or bullous keratopathy who can lie flat on their back.
HeidelbergAges Any age - NCT07378553Recruiting
Ultrasound markers for Duchenne and Becker muscular dystrophies
This study uses a new type of ultrasound scan to look at muscle health in people with Duchenne or Becker muscular dystrophy. It aims to find better ways to track the disease over time.
NantesAges 5–60 - NCT07372027Recruiting
Single-needle return for long-lasting dialysis access
This study compares a new single-needle return technique for your dialysis access (AVF) to the usual two-needle method. It aims to see if using just one needle on your fistula and getting the blood back through a leg vein can help your fistula last longer.
Suzhou, AnhuiAges 18–100 - NCT07374913Recruiting
Study of COL4A1 and COL4A2 gene mutations
This study looks at people with a mutation in the COL4A1 or COL4A2 genes, which can cause problems in small blood vessels throughout the body. It aims to learn more about the condition and may also include family members who carry the mutation or who can serve as healthy controls.
Florence, FIAges Any age - NCT07375940Recruiting
Understanding Behçet Disease in children and teens
This study looks at the health records and blood tests of children and young adults with Behçet disease to learn more about the condition. It also compares their results with healthy children to find clues that may help doctors better understand and treat Behçet disease.
Florence, FlorenceAges 6 months–70 years - NCT07376785Enrolling by invitation
Can smartphones check heart sounds for birth defects?
This study tests whether a smartphone app can accurately detect inborn heart diseases by listening to heart sounds. Participation involves having your heart sounds recorded with a smartphone, which may help find problems earlier.
Hong KongAges 6 years+ - NCT07371663RecruitingPhase 1/Phase 2
TCC1727 combination therapy for advanced solid tumors
This trial tests a new drug, TCC1727, combined with other treatments for people with advanced solid tumors that have stopped responding to standard therapy. It aims to see if these combinations can help shrink or control the cancer.
Beijing, Beijing MunicipalityAges 18 years+ - NCT07371793RecruitingPhase 1/Phase 2
Study of BBI-001 for healthy people and those with iron overload
This trial tests an experimental drug called BBI-001. It first looks at safety in healthy volunteers, then tests whether it can help people with hereditary hemochromatosis, a condition where the body stores too much iron.
Sydney, New South WalesAges 18–70 - NCT07373639Recruiting
Long-term follow-up after BEAM-101 treatment
This study is for people who have already received BEAM-101 in a previous study. It simply follows them over time to track their health and any long-term effects.
Birmingham, AlabamaAges 14–37 - NCT04478734RecruitingPhase 2
Thiamine plus biotin for Huntington’s disease symptoms
This Phase 2 study tests whether taking thiamine and biotin together can improve symptoms in people with Huntington’s disease. It looks especially at people with confirmed genetic Huntington’s disease and certain types of movement and/or mood symptoms.
San Sebastián, San SebastianAges 18 years+ - NCT05126147RecruitingPhase 4
Hydroxychloroquine for mild Graves’ eye swelling
This trial studies whether hydroxychloroquine can improve mild Graves’ orbitopathy (swelling and discomfort around the eyes). It’s meant for people with mild symptoms and who haven’t recently used certain eye or immune treatments.
HsinchuAges 18–75 - NCT05550324Recruiting
Pediatric heart disease patient data repository
This trial is a data repository that collects information from children with heart (cardiovascular) conditions and related syndromes. It focuses on organizing cases and comparing groups to help researchers better understand pediatric heart disease.
Columbus, OhioAges Any age - NCT06048523Recruiting
Study of genetic brain diseases
This study follows people with a confirmed genetic nerve or brain disease over time, collecting blood and sometimes spinal fluid samples to learn more about the condition. It may help researchers understand how the disease progresses.
BordeauxAges 6 years+ - NCT06112340RecruitingPhase 2/Phase 3
Extension study of linsitinib for thyroid eye disease
This trial tests two doses of an oral medication (linsitinib) for people with active, moderate-to-severe thyroid eye disease who were in a previous study and didn't improve enough or had their bulging come back. It aims to see if continuing treatment can help with eye bulging and other symptoms.
Miami, FloridaAges 18 years+ - NCT07370792Recruiting
Genetic mapping for multiple birth defects or intellectual disability
This study uses a new genetic technique to find hidden causes in people with multiple birth defects or intellectual disability who had standard genetic tests come back normal. It might help identify a genetic reason when other tests did not.
Clermont-FerrandAges Any age - NCT07362875Recruiting
Muscle imaging study for myotonic dystrophy
This study uses MRI scans to measure muscle changes in people with myotonic dystrophy (DM1 or DM2) and healthy volunteers. The goal is to find better ways to track the disease over time.
Winston-Salem, North CarolinaAges 18–65 - NCT07365254Recruiting
Newborn whole genome sequencing for genetic disease risk
This study looks at using whole genome sequencing in newborns and families with ongoing pregnancies, including those conceived naturally or with fertility treatments. It aims to better understand and manage genetic disease risks early.
Hangzhou, ZhejiangAges Any age - NCT07364526Recruiting
Genetic screening for embryos in egg donation
This trial tests a non-invasive genetic screening method for embryos to check for chromosomal abnormalities. It aims to improve pregnancy outcomes for women who receive donated eggs.
AlicanteAges 18–50 - NCT07362407Enrolling by invitation
Cannabinoid treatment study for patients in Brazil
This study tracks how cannabis-based medicines work in real life. It looks at how people feel and function while using these treatments prescribed by their doctor.
Florianópolis, Santa CatarinaAges 18 years+ - NCT07363304Recruiting
How ETI therapy affects body and gut in cystic fibrosis
This study looks at how the triple combination therapy (elexacaftor/tezacaftor/ivacaftor, or ETI) affects your body's metabolism, epigenetics, and gut bacteria in people with cystic fibrosis. It aims to understand the broader health benefits of this treatment.
Bari, BariAges 2 years+ - NCT07366593Recruiting
Immune & respiratory health in Down syndrome kids with pneumonia
This study looks at how the immune system and breathing are affected in children with Down syndrome who have pneumonia. It aims to find patterns that could lead to better treatments.
CairoAges 1–6 - NCT07369414Recruiting
NTM Germs in CF Homes: A Household Study
This study looks at whether people with cystic fibrosis (CF) can pick up nontuberculous mycobacteria (NTM) from their home environment. It compares homes of people with CF who have NTM to those who don't, to see where germs might be hiding.
Borstel, Schleswig-HolsteinAges Any age - NCT07369024RecruitingPhase 2
Study of low-dose ketamine for sickle cell pain crisis
This study tests whether a low-dose of ketamine (a pain medicine) can help children and young adults with sickle cell disease who are in a pain crisis. It aims to provide better pain relief while avoiding some side effects of standard painkillers.
Torrance, CaliforniaAges 5–20 - NCT07363538Recruiting
Comparing heart surgery methods for newborns with complex heart defects
This trial tests new surgical strategies for newborns with certain complex heart conditions. It aims to see which techniques lead to better recovery and quality of life in the first year after surgery.
Beijing, Beijing MunicipalityAges Up to 1 week
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.