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
- NCT03919981Recruiting
Testing Cysteamine for cystinosis bone health
This study looks at how cysteamine affects the bone health of people with nephropathic cystinosis, a rare genetic condition. It includes children and adults who are already taking cysteamine by mouth and will follow the study plan closely.
BesançonAges 2 years+ - NCT06203106Recruiting
NYSCF Scientific Discovery Biobank
This study collects blood, saliva, or skin samples from people with certain neurological conditions and from healthy volunteers to help researchers better understand and find treatments for these diseases.
New York, New YorkAges 4 weeks+ - NCT00874783Recruiting
Testing nerve-cell growth from donated body cells
This trial uses donated body cells or small hair samples from people with neurological diseases (and healthy donors) to try to grow “nerve-like” cells for research. It may help scientists learn how to better study and treat different neurological conditions.
Jerusalem, IsraelAges 18 years+ - NCT05017766Recruiting
Study for infections from antibiotic-resistant bacteria
This study looks at new ways to fight antibiotic-resistant bacteria in people with certain common infections. It may help by testing approaches tailored to the specific germs found in your urine, lungs, or deeper infections.
BaselAges 18 years+ - NCT07349004Recruiting
Migraine with aura and PFO study
This study looks at the link between migraine with aura and a small hole in the heart called PFO. It may help researchers understand what causes migraines in some people.
Bologna, BOAges 18 years+ - NCT07347249RecruitingPhase 2
New drug for congenital Factor VII deficiency
This study tests a new drug called Sutacimig for people with congenital Factor VII deficiency, a rare bleeding disorder. The drug is designed to help prevent or control bleeding episodes.
LondonAges 18–60 - NCT07346859RecruitingPhase 3
Study of BP-SCIG 20% for primary immune deficiency
This trial tests a new medicine (BP-SCIG 20%) given under the skin to help children and adults with primary immunodeficiency who have trouble making antibodies. It aims to see if the medicine is safe and works well to prevent infections.
CherkasyAges birth+ - NCT07348601RecruitingPhase 2
Study of CSTI-500 for people with Prader-Willi syndrome
This trial tests an investigational drug called CSTI-500 for people with Prader-Willi syndrome who are in the hyperphagia (constant hunger) phase. It aims to see if the drug can improve behavior and reduce overeating. You and your caregiver will need to visit the clinic regularly and answer questions about symptoms.
Nashville, TennesseeAges 13–50 - NCT06857838Recruiting
Bypass surgery for symptomatic myocardial bridge
This trial tests whether bypass surgery is effective for people with a myocardial bridge (a heart muscle that arches over an artery) that still causes symptoms despite medication. It aims to see if surgery can improve symptoms and outcomes.
Xi'an, ShaanxiAges 18–80 - NCT07346079RecruitingPhase 4
Corticotropin gel for acute gout flares
This study tests two doses of a medicine called Purified Cortrophin Gel for people having a gout flare that started in the last 5 days. It might be an option if other treatments haven't worked or aren't safe for you.
Boston, MassachusettsAges 18–90 - NCT07347743Recruiting
Probiotic supplement for crying and colic in healthy newborns
This trial tests whether a daily probiotic supplement can reduce excessive crying and colic in healthy, full-term newborns. If your baby is healthy and born at UZ Brussel, this study may help find a gentle way to soothe colic.
Jette, Brussels CapitalAges 1 day–2 weeks - NCT07344714Recruiting
Predicting breathing help needs for newborns in NICUs
This study looks at newborns in a lower-level NICU to see which ones will need extra breathing support. The goal is to better predict which babies can be kept at their current hospital instead of being moved.
Rochester, MinnesotaAges 7 months–9 months - NCT07344012Recruiting
Parenting hopes in women with Turner syndrome
This study looks at women with Turner syndrome who wanted to become parents. It aims to understand what support they received and what happened with their parenting plans.
StrasbourgAges 18–40 - NCT07346976Recruiting
Jaundice management study using carbon monoxide breath test in newborns
This study uses a simple breath test to measure carbon monoxide levels in newborns, which may help doctors better manage jaundice. It aims to understand how these levels change and how they relate to jaundice treatment.
Yiwu, ZhejiangAges birth–4 weeks - NCT07343752Recruiting
Blood flow restriction therapy for teen flat feet
This study tests whether adding a special blood-flow restriction technique to physical therapy helps teenagers with stiff, spasming flat feet more than physical therapy alone. It is for teens whose flat feet didn't get better with initial treatment.
AsyutAges 10–18 - NCT07345338Enrolling by invitation
Genetic testing for non-ischemic heart muscle disease
This study offers genetic testing to adults with a type of heart muscle disease (non-ischemic cardiomyopathy or dilated cardiomyopathy) that is not caused by blocked arteries. The goal is to find genetic causes, which may guide treatment and family screening.
Calgary, AlbertaAges 18 years+ - NCT07348926Recruiting
Family well-being & independence in rare genetic disorders
This study looks at how a child's ability to function independently affects the well-being of their family. It is for caregivers of young children (0–4 years) with rare genetic disorders who have been in physiotherapy for at least 6 months.
IstanbulAges 18–65 - NCT07345143Enrolling by invitation
AI study for gestational diabetes
This trial uses artificial intelligence to help manage gestational diabetes in pregnant women. It may improve care for women diagnosed early in pregnancy.
São José do Rio Preto, São PauloAges 18–40 - NCT03434548Recruiting
Imaging study for early and late Huntington’s disease changes
This study uses repeat brain scans (including PET and MRI) to track changes in the brain caused by Huntington’s disease over time. It may help researchers understand disease progress and test how well the scans reflect early vs later disease.
London, EnglandAges 21–75 - NCT02378805Recruiting
Alport syndrome registry to help delay kidney failure
This is a registry that collects information from people with Alport syndrome to better understand the disease and how to delay kidney failure. It may help researchers learn which factors and treatments are most important over time.
Göttingen, Lower SaxonyAges Any age - NCT07334912RecruitingPhase 2
Study of AEF0217 for people with Down syndrome
This study tests if an investigational drug called AEF0217 can help improve thinking and daily living skills in people with Down syndrome. It's for ages 16-32 who have a caregiver to support them throughout the trial.
BordeauxAges 16–32 - NCT07335523Recruiting
Testing for poor digestion in Alagille syndrome after liver transplant
This study looks for signs of poor digestion after a liver transplant in people with Alagille syndrome. You collect stool samples for 3 days while on a high-fat diet to see if your pancreas is making enough enzymes.
Aurora, ColoradoAges 7–50 - NCT07338370Recruiting
PGE2 levels and heart duct closure in preterm babies
This trial measures a substance called prostaglandin E2 (PGE2) in preterm babies to see if it can predict whether a heart blood vessel (ductus arteriosus) will close on its own or need treatment. This could help doctors decide when to use medication to close the vessel.
Cairo, AbbasiaAges 1 day–1 week - NCT07334444Recruiting
Improving sleep apnea care for children with Down syndrome
This study looks at how sleep apnea is managed in children with Down syndrome. It aims to find better treatment approaches by reviewing data from kids who visited ear, nose, and throat (ENT) or sleep specialists in Strasbourg.
StrasbourgAges birth–17 years
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.