Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,858 recruiting studies in this area right now.
By phase
- Not applicable2,771
- Phase 2284
- Phase 1235
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- 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
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT05765084RecruitingPhase 1/Phase 2
Atezolizumab plus WT1 vaccine with chemo for pleural mesothelioma
This trial studies a treatment plan that combines an immune drug (atezolizumab) and a personalized cancer vaccine (WT1/DC) with standard first-line chemotherapy for epithelioid malignant pleural mesothelioma that cannot be removed with surgery. It aims to improve control of the cancer by helping the immune system work alongside chemotherapy.
Edegem, AntwerpAges 18 years+ - NCT05765162Recruiting
Research study on safer, more tailored anesthesia
This study looks at safer anesthesia—meaning medicines used during procedures—using a more personalized approach. It may help make anesthesia safer for adults having medical procedures.
Nykøbing FalsterAges 18–100 - NCT05766774Recruiting
Diet study for adults with cystic fibrosis and high sugar intake
This trial studies whether changing eating habits—especially added sugar intake—affects health in adults with cystic fibrosis. It may help by testing a diet approach aimed at better outcomes related to metabolism and overall health.
Atlanta, GeorgiaAges 18 years+ - NCT05767203Recruiting
Study genetic and blood markers in people with inherited intellectual disability
This study looks at genetic-related and blood/biologic markers in people with an inherited (genetic) intellectual disability. The goal is to better understand these differences and how they might relate to the condition.
ParisAges Any age - NCT05767047RecruitingPhase 3
Apremilast study for children with mouth ulcers from Behçet’s
This trial studies whether apremilast can help reduce mouth ulcers in children who have Behçet’s disease or juvenile psoriatic arthritis. You may be considered if your child finished a main study and is under 18, and can safely continue treatment.
ThessalonikiAges 5–18 - NCT05767762Recruiting
Tests whether leg muscle patterns predict treatment outcome in clubfoot
This study looks at how certain leg muscles move to see if that can predict how well an Achilles tenotomy (a small procedure to release tight tendon) works for children with idiopathic clubfoot. It may help doctors choose the best timing and approach for treatment.
NiceAges birth–1 year - NCT05768035RecruitingPhase 1/Phase 2
Testing SMART101 after half-matched stem cell transplant
This trial studies a new immune treatment called SMART101 given after a half-matched (haploidentical) stem cell transplant that uses post-transplant cyclophosphamide to prevent complications. It mainly checks safety, and may also look at how well SMART101 helps people with blood cancers recover after transplant.
MarseilleAges 18 years+ - NCT05767216Recruiting
Study of genetic and epigenetic differences in twin pairs with Down syndrome
This study looks at how genetics and gene-regulation (how genes turn on and off) differ between close family members who have different Down syndrome status. Researchers hope the findings will help explain why Down syndrome outcomes can vary even in very closely related people.
ParisAges 4–11 - NCT05768139RecruitingPhase 1/Phase 2
Testing a new targeted drug for advanced solid tumors with PI3Kα mutation
This trial tests tersolisib, a new targeted medicine, in adults with advanced or hard-to-remove solid cancers. It focuses on tumors that have a specific genetic change (PI3Kα mutation) to see how well the treatment works and what side effects it causes.
Los Angeles, CaliforniaAges 18 years+ - NCT05769621Recruiting
Study of medical records in propionic acidemia patients
This is a “chart review” study that looks at how people with propionic acidemia have symptoms and medical events over time. It uses existing medical records (not an experimental drug) to better understand the disease and care needs.
Stanford, CaliforniaAges 2 years+ - NCT05773638Recruiting
Study blood clots and heart disease in adults with Von Willebrand
This study looks at cardiovascular problems and blood-clot events (like deep vein clots or pulmonary embolism) in adults with Von Willebrand disease in western France. It may help doctors better understand who is at risk and how to manage these complications.
AngersAges 18 years+ - NCT05774288Recruiting
Testing a device to close a heart opening called PFO
This trial studies whether a Cera™ device is safe and effective for closing a patent foramen ovale (PFO), a small hole in the heart. It is for adults whose PFO is linked to certain types of stroke or ongoing clot-related events.
Beijing, Beijing MunicipalityAges 18–60 - NCT05775679Recruiting
CLEFT-Q survey study for people with cleft lip or palate
This study tests how consistently and clearly a questionnaire (CLEFT-Q) measures your symptoms and how well treatment helps. It may help researchers improve care for people who need a planned second surgery for the nose, lips, jaw, or speech.
Malmö, Skåne CountyAges 8–29 - NCT05776069RecruitingPhase 1/Phase 2
Test drug VGA039 for bleeding control in von Willebrand disease
This Phase 1/2 study tests VGA039 in healthy adults and people with von Willebrand disease (VWD). The goal is to see if the drug is safe and how it affects bleeding-related factors, especially in people who have symptoms.
Los Angeles, CaliforniaAges 12–60 - NCT05778188RecruitingPhase 2
RLS-0071 for newborns with moderate or severe HIE
This trial studies whether RLS-0071 is safe and can help some newborns with moderate or severe hypoxic-ischemic encephalopathy (HIE), a brain injury caused by low oxygen around birth. Babies in the study also receive standard whole-body cooling (therapeutic hypothermia), and the researchers will watch for side effects and early signs of benefit.
Little Rock, ArkansasAges Up to 10 hours - NCT05790330Recruiting
Study brain–skin links in teenagers with eczema
This study looks at how eczema may be connected to the brain and sleep, using questionnaires and MRI scans. It may help researchers understand what drives symptoms and how treatments could be improved.
LondonAges 12–18 - NCT05791864RecruitingPhase 1/Phase 2
Gene therapy for vision loss in CLN2, tested for safety
This early-phase trial studies a gene therapy medicine called TTX-381 to see if it is safe and tolerable in people with CLN2 disease that affects the eyes. The study also looks at how well the treatment fits into existing eye enzyme therapy care.
HamburgAges 1–7 - NCT05797272Recruiting
In-utero stem cell treatment for Bart’s hydrops
This trial studies whether giving hematopoietic (blood-forming) stem cells while the baby is still in the womb can treat fetuses with Bart’s hydrops fetalis syndrome. It aims to improve outcomes after prenatal intervention in carefully selected pregnancies.
ShatinAges 18 years+ - NCT05797857Recruiting
Exercise program for transthyretin heart amyloidosis
This trial studies whether a structured exercise training program is safe and helpful for people with transthyretin (ATTR) cardiac amyloidosis. It may help improve fitness and daily functioning, and it may also compare groups for specific eligibility needs.
Boston, MassachusettsAges 60–90 - NCT05799118Recruiting
Genetic modifiers study for inherited blood disorders
This study looks at how your genes can affect inherited blood conditions like sickle cell disease and thalassemia. It may help researchers understand why symptoms differ from person to person, which could guide better future treatments.
Boston, MassachusettsAges 2 years+ - NCT05802264RecruitingPhase 1
Inhaled amphotericin B for healthy volunteers and cystic fibrosis
This early-phase study tests how different doses of an inhaled medicine (amphotericin B) work and are tolerated. It includes healthy adults and people with cystic fibrosis (CF) to see if the dose schedule is safe and potentially beneficial.
Canberra, Australian Capital TerritoryAges 16 years+ - NCT05802836Recruiting
Tracking an antibody response in severe hemophilia A on emicizumab
This trial studies how your body’s anti-factor VIII antibodies change while you are treated with emicizumab for severe hemophilia A. The goal is to better understand the immune response during treatment and whether it relates to how well therapy works.
Frankfurt am Main, HesseAges Any age - NCT05806398Recruiting
Sealant test for molars with enamel defects in children
This study tests whether a glass ionomer sealant can protect molars with molar-incisor hypomineralization (MIH), a type of enamel weakness, by sealing the biting surface. It may help reduce damage in affected teeth when children still have healthy surfaces that can be sealed.
TriesteAges 6–10 - NCT05808153Recruiting
Brain scans and thinking tests to track Huntington’s progression
This study uses special brain imaging and memory/thinking tests to find early “markers” that can predict how Huntington’s disease will progress over time. You may qualify as someone with early-stage Huntington’s or as a healthy control, if you meet specific safety and scoring requirements.
Créteil, Île-de-France RegionAges 18–65
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,858 recruiting medical genetics studies from the U.S. registry right now, out of 5,764 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.