Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,767 recruiting studies in this area right now.
By phase
- Not applicable2,684
- Phase 2280
- Phase 1241
- Phase 3210
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease134
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)38
- 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
- Massachusetts108
- Arizona87
- New York85
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,767 total
- NCT05951478Recruiting
Study for people with primary ciliary dyskinesia follow-up
This study (RaDiCo-DCP) follows people who have a confirmed diagnosis of primary ciliary dyskinesia, a condition that can affect how your lungs clear mucus. The trial looks at long-term symptoms over time, which may help improve care planning and understanding of the condition.
BesançonAges Any age - NCT05945576Recruiting
RaDiCo IDMet study for people with inherited ID
This study enrolls people of all ages who have a confirmed inherited condition called ID. It collects information to better understand the condition and support future care.
AngersAges Any age - NCT05954403Recruiting
Eye birth defect study from newborns to adults
This is a national study that follows people with certain rare eye birth defects and their families, including pregnant people. It aims to better understand how often related health issues happen and to study inheritance (DNA) in families.
Paris, Île-de-France RegionAges Any age - NCT05955014Recruiting
Study collects data from people with von Hippel-Lindau disease
This study is for people with von Hippel-Lindau (vHL) disease to collect information for research. It may help researchers better understand the condition and how to care for people with it.
Houston, TexasAges 18 years+ - NCT05943002Recruiting
Track symptoms and life effects using a phone survey
This study uses patient-reported questionnaires and health/economics/psychosocial questions for people with Friedreich ataxia. It aims to better understand how the condition affects daily life and what outcomes matter most, which can help future care and research.
InnsbruckAges 12 years+ - NCT05973656Recruiting
Study how alcohol affects the risk of mouth cancer
This study looks at how a breakdown product from alcohol (acetaldehyde) may play a role in oral (mouth) cancer. It may help researchers understand who is more affected by alcohol and why.
Minneapolis, MinnesotaAges 18–45 - NCT05962346Recruiting
Pregnancy balloon procedure for severe left diaphragmatic hernia
This trial studies whether placing a small balloon in the fetus’s airway during pregnancy (a procedure called FETO) can improve lung development and outcomes for babies with severe left congenital diaphragmatic hernia. You may be considered if the condition is isolated, very severe, and you can meet specific timing and follow-up requirements.
Rochester, MinnesotaAges 18–50 - NCT05964387Recruiting
Silver fluoride treatment and local numbing for tooth cavities
This study tests whether applying silver diamine fluoride (a cavity-stopping liquid) changes how well local numbing works during dental treatment for certain tooth defects. It may help determine the safest and most comfortable way to treat weak, cavity-prone molars in children.
Ann Arbor, MichiganAges 6–11 - NCT05960461Recruiting
Online program for mental health providers working with young children
This study tests an online training tool for mental health providers who serve children ages 3 to 12 (and their families). It’s meant to help providers improve the mental health support they offer through their agency or practice.
Rochester, New YorkAges 18 years+ - NCT05966038Recruiting
Study of ALS and related movement nerve conditions
This study collects and stores medical information from people diagnosed with ALS or certain related conditions affecting movement. It may help researchers better understand how these illnesses change over time and improve future treatments.
Loma Linda, CaliforniaAges 18–90 - NCT05948943RecruitingPhase 2/Phase 3
Alpelisib for lymphatic malformations with a PIK3CA change
This trial tests alpelisib (a targeted medicine) for people who have lymphatic malformations (LyM) that cause symptoms and have a PIK3CA mutation. It may help shrink or control these lesions when other procedures are delayed until after the first 24 weeks.
Oakland, CaliforniaAges birth–100 years - NCT05962398Enrolling by invitation
Follow-up study for men previously treated with CSL222
This study checks long-term safety and outcomes in men with hemophilia B who previously received CSL222. It may help your care team better understand how well the treatment lasts over many years.
Phoenix, ArizonaAges 18 years+ - NCT05953584RecruitingPhase 2
Etavopivat for preventing stroke risk in sickle cell children
This trial studies etavopivat to see if it can improve blood-flow measurements in the brain that predict future stroke risk in children with sickle cell disease. If you qualify, you would take the study medicine and be monitored with brain blood-flow testing.
Raipur, ChhattisgarhAges 12–16 - NCT05967351Enrolling by invitationPhase 3
Long-term follow-up for Duchenne patients treated before
This study checks how people with Duchenne muscular dystrophy are doing long-term after receiving delandistrogene moxeparvovec (SRP-9001) in an earlier trial. It helps researchers understand the long-term safety and effects of that treatment.
Little Rock, ArkansasAges Any age - NCT05927467Recruiting
Study of Alport syndrome patients in a kidney–skin research group
This study follows people diagnosed with Alport syndrome to better understand the condition and its effects. It may help researchers learn what to watch for over time and how the disease progresses.
Paris, Île-de-France RegionAges Any age - NCT05921162Enrolling by invitation
Long-term follow-up after a vision gene eye injection
This study checks how people who previously received a gene therapy eye injection (vMCO-I) are doing over the long term. It may help researchers understand long-lasting effects and safety.
Cuttack, OdishaAges 18 years+ - NCT05933057RecruitingPhase 3
Givinostat for wheelchair-bound boys with Duchenne muscular dystrophy
This Phase 3 trial tests givinostat to see if it can improve safety and functional outcomes in boys with Duchenne muscular dystrophy who cannot walk. You might be a candidate if you meet age, genetic diagnosis, wheelchair-level mobility, and specific arm-strength/respiratory/heart criteria.
LeuvenAges 9–17 - NCT05929807Enrolling by invitationPhase 2/Phase 3
Weekly injection study for children with achondroplasia
This trial looks at how safe and effective weekly under-the-skin (subcutaneous) TransCon CNP is for children and teens with achondroplasia over the long term. You may be considered if your child has already finished a previous TransCon CNP study and can follow weekly injection and follow-up visits.
Little Rock, ArkansasAges 3–15 - NCT05935358RecruitingPhase 4
Nuwiq added around surgery for people with hemophilia A
This Phase 4 study looks at how well Nuwiq works to manage bleeding risk around the time of major elective surgery in people with severe hemophilia A who already take emicizumab to prevent bleeds. It may help your surgical team plan safer blood-clotting support during and after surgery.
Dallas, TexasAges 12 years+ - NCT05936580RecruitingPhase 4
Nuwiq dosing for girls and women with hemophilia A surgery
This trial studies how to dose Nuwiq (a factor medicine) and track outcomes in girls and women with hemophilia A who need major surgery. It may help confirm the best way to manage bleeding risk around surgery.
San Antonio, TexasAges 12 years+ - NCT05919264RecruitingPhase 1/Phase 2
FOG-001 for advanced solid tumors with certain gene changes
This trial tests a new drug, FOG-001, alone or with other treatments, for people with advanced solid tumors that have specific changes in the WNT pathway genes. It aims to see if the drug can shrink tumors or slow their growth.
Phoenix, ArizonaAges 18 years+ - NCT00500123Recruiting
Alpha-1 genetic and blood testing for people at risk
This study checks whether doing alpha-1 screening (genetic test and a blood level test) can help identify alpha-1 antitrypsin deficiency in people at risk. It may be useful if you have symptoms or a family history, but you haven’t had qualifying testing yet.
Gainesville, FloridaAges Any age - NCT05937594Recruiting
Testing microRNA markers to spot newborn opioid withdrawal
This study looks at tiny genetic signals (microRNA) in newborn samples to identify neonatal opioid withdrawal syndrome. It may help doctors recognize withdrawal sooner, so babies can get the right care faster.
Hershey, PennsylvaniaAges 1 day–5 days - NCT05911139Enrolling by invitation
Study how anesthesia affects brain injury markers in craniosynostosis surgery
This study looks at how general anesthesia may affect “brain injury marker” levels during and after surgery for single-suture craniosynostosis in infants. It may help doctors better understand anesthesia timing and safety for babies having corrective surgery.
BratislavaAges Up to 18 years
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,767 recruiting medical genetics studies from the U.S. registry right now, out of 5,497 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.