Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,859 recruiting studies in this area right now.
By phase
- Not applicable2,773
- Phase 2283
- Phase 1236
- Phase 3215
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis128
- Congenital Heart Disease85
- 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
- Pennsylvania66
- Ohio57
Recruiting studies3,859 total
- NCT05213676RecruitingPhase 4
Testing stopping inhaled nitric oxide in newborns with CDH
This trial studies whether stopping inhaled nitric oxide (a breathing medicine) can be safely used in newborns with congenital diaphragmatic hernia (CDH). It may help the team decide the best way to use this medicine during early newborn care.
Birmingham, AlabamaAges birth–1 month - NCT05217589Recruiting
Mood and emotions study on eating before exercise
This study looks at how different moods and emotions affect your body’s responses to eating a sports drink before you exercise. You may need to run for 30 minutes and watch/listen to video clips during the study.
Norfolk, VirginiaAges 18–55 - NCT05219617RecruitingPhase 3
Carisbamate for frequent drop seizures in Lennox-Gastaut
This Phase 3 study tests whether carisbamate can reduce drop seizures in people with Lennox-Gastaut syndrome (LGS). It also checks whether the treatment is safe when added to existing seizure medicines.
Palo Alto, CaliforniaAges 4–55 - NCT05224778Recruiting
Study for young children with neonatal myotonic dystrophy type 1
This study looks at measurements and child-focused outcomes in babies and toddlers with myotonic dystrophy type 1 (DM1) that started in the newborn period. It may help researchers better track how the condition affects children and evaluate care plans more accurately.
Los Angeles, CaliforniaAges Up to 4.9 years - NCT05225311Recruiting
Registry for pregnancies with Ebstein and tricuspid valve issues
This registry collects information about pregnancies where the baby is diagnosed with Ebstein anomaly and/or tricuspid valve dysplasia. It helps doctors learn how these heart conditions affect pregnancies and outcomes, and can support better future care.
Toronto, OntarioAges Any age - NCT05226624Recruiting
Improving care for babies born with opioid withdrawal
This program helps participating hospitals improve care for newborns who may go through opioid withdrawal (also called neonatal abstinence syndrome). It mainly aims to make mother-baby support and treatment plans work better after delivery.
Edmonton, AlbertaAges Any age - NCT05231798RecruitingPhase 2
PET and brain scans in adults with Down syndrome
This study looks at how well a brain chemical system works in adults with Down syndrome as they age, including how it relates to Alzheimer’s-type brain changes and thinking skills. It uses brain scans and memory/thinking tests to see whether these measures can help track risk or changes over time.
Nashville, TennesseeAges 18–55 - NCT05231876Recruiting
French Wilson disease registry
This study keeps a registry (a confidential list) of people with Wilson disease. It helps researchers learn more about the disease and improve future care, without requiring specific treatment changes.
Paris, Île-de-France RegionAges birth–99 years - NCT05234723Recruiting
CMV Infection Treatment Outcomes in Transplant Patients
This study tracks how well ganciclovir (an antiviral medicine) works in transplant recipients who develop a resistant CMV infection. Researchers will review medical records to understand treatment success and patient outcomes.
Houston, TexasAges 18 years+ - NCT05234567Recruiting
Study of children with HPP on (or starting) asfotase alfa
This study follows children with hypophosphatasia (HPP) to better understand how they do while receiving asfotase alfa. It may help improve care for families living with pediatric-onset HPP.
Hartford, ConnecticutAges Any age - NCT05238610Recruiting
Registry for older adults with stroke of unclear cause and PFO
This study follows adults over 60 who had a stroke where the cause is unclear, and where a heart opening (PFO) may have contributed. It mainly collects information to better understand this stroke pattern and how these patients do over time.
SeoulAges 60 years+ - NCT05236153Recruiting
Study of heart valve procedure in repaired Tetralogy of Fallot
This study looks at how a transcatheter pulmonary valve replacement works in people with repaired Tetralogy of Fallot or double outlet right ventricle. The goal is to better understand the heart’s anatomy and how it may affect the valve procedure.
Boston, MassachusettsAges Any age - NCT05239858Recruiting
International registry for people with Wilson’s disease
This trial is a patient registry that collects health information from people with Wilson’s disease, including those without symptoms yet and those taking any type of treatment. It helps researchers understand the disease and how it progresses, which can support future studies and care improvements.
LeuvenAges 12 years+ - NCT05241418Recruiting
MRI study of heart scar using gadolinium in repaired heart defects
This trial looks at heart MRI images to see whether a contrast dye (gadolinium) can highlight scar-like areas after congenital heart problems have been repaired. It may help doctors better understand healing and plan care, especially for future heart procedures.
Boston, MassachusettsAges 13 years+ - NCT05241262RecruitingPhase 1
N-acetylcysteine for people with m.3243A>G and low brain glutathione
This trial tests whether the supplement N-acetylcysteine (NAC) can help people who have the m.3243A>G mitochondrial genetic change and low levels of a brain chemical called glutathione. The study aims to see if raising glutathione in the brain is safe and potentially beneficial for these patients.
New York, New YorkAges 18–80 - NCT05247645Recruiting
Collecting data from people with rare bone diseases
This study gathers information about people with rare illnesses that mainly affect the bones. It may help researchers better understand these conditions and improve future care.
Bologna, Emilia-RomagnaAges Any age - NCT05248230RecruitingPhase 2
Study drug for adults with cystic fibrosis lung disease
This Phase 2 trial tests 4D-710 to improve lung health in adults with cystic fibrosis who either cannot take CFTR modulator medicines or are not doing well enough on them. You may be eligible if you have moderate lung function and stable oxygen levels, and you meet other safety and health requirements.
Birmingham, AlabamaAges 18 years+ - NCT05253560Recruiting
Study prodromal Parkinson signs in GBA1 mutation carriers
This study looks at early (before full disease) Parkinson-like symptoms in people who carry a specific genetic change called GBA1. It may help researchers understand who could develop Parkinson’s and how early signs can be tracked.
Jerusalem, Please Select...Ages 40–75 - NCT05258981Recruiting
MRI and testing study linking genes and thinking in adults
This study looks at how patterns on brain MRI scans and genes relate to thinking and test performance in adults. It may help researchers better understand long-term brain and cognitive outcomes and how they differ between people with specific early-life heart conditions and healthy controls.
Boston, MassachusettsAges 24–35 - NCT05262907Recruiting
Study of heart imaging in people with one pumping ventricle
This study uses routine heart scans (either echocardiogram or MRI) to better understand single-ventricle heart conditions. It may help improve how doctors evaluate and monitor your heart over time.
Durham, North CarolinaAges Any age - NCT05264753Recruiting
Follow-up study for a device that closes an abnormal heart blood vessel
This study checks how well and how safely the Occlutech PDA Occluder works over time to close a patent ductus arteriosus (PDA). You may be considered if your PDA can be treated with this non-surgical device and you can return for follow-up visits.
MontrealAges Any age - NCT05268107RecruitingPhase 4
Study dupilumab skin effects in different ethnic groups
This Phase 4 study looks at how dupilumab works in people with moderate-to-severe atopic dermatitis (eczema), and whether the mechanism differs across ethnic backgrounds. If you’re eligible, it may help doctors understand who benefits most from dupilumab.
Ann Arbor, MichiganAges 18 years+ - NCT05279040Recruiting
Trikafta plus exercise program for adults with cystic fibrosis
This study tests how starting Trikafta affects exercise ability and breathing in adults with cystic fibrosis. You may be asked to do supervised breathing and exercise tests to see if Trikafta helps your lung function and stamina.
Vancouver, British ColumbiaAges 19 years+ - NCT05283993Recruiting
Hospital study following people with plasma cell disorders
This is a hospital-based study that follows people with plasma cell disorders to better understand these conditions. It mainly gathers information from patients who are confirmed to have one of several related diseases.
BeijingAges Any age
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,859 recruiting medical genetics studies from the U.S. registry right now, out of 5,745 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.