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
- NCT05849077Recruiting
Testing oxygen saturation targets during newborn resuscitation
This study looks at the best oxygen saturation targets and resuscitation approach for very premature babies right after birth. It may help clinicians guide breathing support more safely in the first minutes of life.
Dallas, TexasAges birth–10 minutes - NCT05848895Recruiting
Osteopathy treatment for infants with positional head shape
This study tests an osteopathic manual therapy plan (hands-on treatment) for babies with positional plagiocephaly, aiming to improve head shape and comfort. It also asks families to attend multiple treatment and measurement visits so researchers can track change over time.
San Diego, CaliforniaAges 1 day–4 months - NCT05849285Recruiting
Testing a program to support adults with lifelong care needs
This study tests a “Transitional and Lifelong Care” program for adults who had a disability since childhood, and includes their main caregiver or support person. It aims to see how this type of care support may improve experiences and support over time.
London, OntarioAges 21 years+ - NCT05849389RecruitingPhase 2
Vosoritide for taller growth in girls with Turner syndrome
This Phase 2 study tests vosoritide to help children with Turner syndrome grow taller before puberty. You may be able to join if your child is pre-pubertal, has short height for age, and either has not tried growth hormone or has not responded well to it.
Washington D.C., District of ColumbiaAges 3–11 - NCT05849662RecruitingPhase 1/Phase 2
Trametinib plus azacitidine for newly diagnosed JMML
This trial studies whether trametinib and azacitidine can control juvenile myelomonocytic leukemia (JMML) soon after diagnosis. It’s designed to test safety and how well the treatment works in children and young adults who meet specific leukemia and genetic criteria.
Phoenix, ArizonaAges 1 month–21 years - NCT05887700Recruiting
Follow-up check for an ASD device closure
This study follows people who had an atrial septal defect (a hole between heart chambers) closed using a specific device. It aims to collect outcomes and safety information after the hospital procedure.
CuritibaAges Any age - NCT05853107Recruiting
AuTNA I implant study for retinitis pigmentosa with minimal vision
This pilot study tests an implant called AuTNA I in adults with retinitis pigmentosa who currently have no or only very limited light perception. The goal is to see whether the implant can work safely and potentially improve vision in a carefully selected group of people.
Shanghai, Shanghai MunicipalityAges 18–70 - NCT05853783Recruiting
Testing a mouth test on biopsy samples from abnormal lesions
This trial looks at archived (saved) mouth biopsy samples to see how well the STRATICYTE™ test can identify potentially dangerous “pre-cancer-like” changes. It may help doctors screen or better understand which abnormal areas in the mouth later turn into oral cancer.
Birmingham, AlabamaAges 18 years+ - NCT05858619RecruitingPhase 4
Study of skin response to dupilumab in eczema
This trial studies how the body’s “molecular signals” change in the skin after using dupilumab for eczema. It may help researchers understand who benefits most from treatment.
San Francisco, CaliforniaAges 18 years+ - NCT05858606Recruiting
Study genes and causes of very short height in children
This study looks at your child’s growth history and performs genetic testing to better understand why some children are very short. It may help researchers identify possible underlying causes and improve care, using blood samples from the child and both parents.
MontpellierAges 4–18 - NCT05860569RecruitingPhase 1
Gene therapy safety study for inherited high triglycerides and pancreatitis
This Phase 1 trial tests the safety of a gene therapy for people with inherited, hard-to-control high triglycerides (fat in the blood) who have had pancreatitis episodes. It may help by targeting the genes involved in triglyceride control, but the main goal here is to check safety and tolerability.
Beijing, Beijing MunicipalityAges 18–60 - NCT05865119Recruiting
Best treatment approach for a certain heart wall defect
This study looks at the best treatment plan for people with a specific type of hole in the heart wall that affects the right side of the heart. It may help doctors choose an approach based on how well it works for patients who need treatment.
BordeauxAges 12 years+ - NCT05867979Recruiting
Find hidden DNA changes in some boys with unclear DSD
This trial looks for “structural variants,” meaning larger DNA rearrangements that standard gene tests may miss, in boys with certain differences in sex development (DSD) when results are unclear. It may help provide a clearer diagnosis that can guide care for your family.
MontpellierAges 6 months+ - NCT05868629Recruiting
Test of two targeted drugs for tumors with BRAF V600E
This trial tests dabrafenib plus trametinib in people whose solid tumors have a BRAF V600E mutation that cannot be fully removed by surgery or has spread. It looks at whether these targeted medicines can shrink tumors and control the disease.
Torrance, CaliforniaAges 1–100 - NCT05869851Recruiting
Infant hip ultrasound study: observe vs bracing
This study looks at babies under 3 months old with certain ultrasound findings of an underdeveloped hip (“hip dysplasia”). It compares outcomes between simply watching the hip and using a brace (when recommended).
Vancouver, British ColumbiaAges Up to 3 months - NCT05871216Recruiting
Study of joint instability in people with loose joints
This trial is testing how to assess and manage joint instability in people who have unusually loose (hypermobile) joints. If you have moderate to severe loose joints, you may be able to join after screening checks like movement tests and health history.
BolognaAges 18–60 - NCT05876416Recruiting
Study genetics of rare bone growth disorders
This study looks at genetic causes of skeletal (bone growth) disorders in people who show signs based on height and bone imaging. Genetic testing may help explain what’s behind the condition, especially when it seems inherited.
StockholmAges Any age - NCT05884892Recruiting
Join a heart condition registry for hypertrophic cardiomyopathy
This program builds a registry of people diagnosed with hypertrophic cardiomyopathy and their family members. By collecting health information over time, researchers hope to better understand the condition and improve care.
AswānAges Any age - NCT05885412RecruitingPhase 1
Study drug for heart rhythm problems in PKP2 heart disease
This Phase 1 trial tests RP-A601 (a gene-delivery type treatment) to see if it can reduce dangerous heart rhythm problems in people with PKP2-related arrhythmogenic cardiomyopathy (ACM). Because it’s an early safety/dose study, the main goal is to check safety and how the body responds.
La Jolla, CaliforniaAges 18 years+ - NCT05888532RecruitingPhase 1/Phase 2
Testing a copper-based scan and treatment in advanced cancers
This Phase 1/2 trial studies a copper-labeled medicine called 64Cu-GRIP B in people with advanced, metastatic cancers. It may help doctors see where cancer is and how well the drug works, especially alongside immune checkpoint therapy for some cancer types.
San Francisco, CaliforniaAges 18 years+ - NCT05889013Recruiting
Nasal swab testing to guide care for kids with breathing issues
This study checks whether special nose tests help doctors choose the best treatments for children with long-term breathing and airway symptoms. It may help your child get more targeted care based on the test results.
Hartford, ConnecticutAges 2 years+ - NCT05889507RecruitingPhase 3
Cooling treatment for newborns with mild brain injury
This Phase 3 study tests whether cooling a newborn’s body can help babies with mild brain injury after a difficult birth. It aims to see if cooling improves brain-related outcomes compared with standard care.
NaplesAges 1 hour–6 hours - NCT05895032Recruiting
Exercise program for children with hemophilia A or B
This trial studies whether a structured exercise program helps children with hemophilia A or B safely. It looks at children’s ability to exercise and function, while aiming to avoid complications like leg bleeds or injuries.
Canterbury, KentAges 6–12 - NCT05896306Recruiting
Monitor blood flow in newborns during resuscitation
This study uses a painless light-based monitor to track how blood is moving in the brains of newborns during resuscitation. It may help doctors better understand what’s happening during care for very sick newborns.
Graz, StyriaAges birth–15 minutes
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.