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
- NCT04694248Recruiting
Blood thinner plus antiplatelet after iliac vein stent
This trial looks at whether taking a blood thinner and an additional clot-prevention medicine (antiplatelet) after iliac vein stenting can improve treatment of certain deep vein clots. It may help prevent the clot from coming back or spreading, but it also increases bleeding risk, so careful eligibility screening is important.
Hangzhou, ZhejiangAges 18 years+ - NCT04875702RecruitingPhase 4
Compare urate targets to reduce gout attacks or symptoms
This study compares two different treatment goals for a blood level called urate in people with gout. The goal is to see which approach lowers gout flares better while helping people feel better.
South Birmingham, AlabamaAges 18–90 - NCT06287762Recruiting
Natural history study of RYR1-related disorders
This study follows people with RYR1-related disorders over time to learn how the condition progresses. It may help researchers plan future treatments.
Bethesda, MarylandAges 7–100 - NCT06292650RecruitingEarly Phase 1
Gene therapy ZM-02 for advanced retinitis pigmentosa
This experimental gene therapy (ZM-02) is designed for people with advanced retinitis pigmentosa who have very poor vision (counting fingers or worse). It aims to see if the treatment is safe and can improve vision by replacing a faulty gene in the retina.
Beijing, Beijing MunicipalityAges 18–65 - NCT06285071Enrolling by invitation
Study of Alhemo for hemophilia A or B with inhibitors
This study is tracking how well Alhemo works in people with hemophilia A or B who have developed inhibitors (antibodies that block treatment). It is for patients of any age who have already started taking Alhemo as part of their regular care.
Toshima City, TokyoAges Any age - NCT02705846Recruiting
Study tracks outcomes in men with inherited prostate cancer risks
This study looks at how prostate cancer diagnoses and treatments turn out over time. It compares men who carry rare inherited gene changes that raise prostate cancer risk with men who do not, to better understand which treatments and outcomes may differ.
Sutton, SurreyAges 18 years+ - NCT06289348Recruiting
How is PKU announced to families after newborn screening?
This study talks to parents or doctors about how they first learned their child had PKU through newborn screening. It aims to improve how these rare metabolic diseases are communicated to families.
ParisAges 18 years+ - NCT06290713RecruitingPhase 2
Blood flow and exercise study for Duchenne muscular dystrophy
This trial tests if a medicine that improves blood flow, combined with exercise, can help muscles in boys with Duchenne muscular dystrophy. It is for boys who can still walk and are on a stable steroid medication.
Gainesville, FloridaAges 6 years+ - NCT06285539RecruitingPhase 2
Testing a drug for rare inflammatory diseases
This trial is testing a new drug (a JAK inhibitor) for people with rare inflammatory diseases like Behçet's, myositis, or IgG4-related disease that hasn't improved with standard treatments. The goal is to see if it can help control symptoms and reduce inflammation.
AmsterdamAges 18–65 - NCT06293560Recruiting
Genetic study of rare eye conditions in children
This study looks at the genetic causes of rare eye conditions (microphthalmia, anophthalmia, and coloboma). It helps researchers understand why these conditions happen and how they run in families.
Houston, TexasAges Any age - NCT06290401Recruiting
Boosting self-management in teens with sickle cell disease
This study is testing a program to help adolescents with sickle cell disease better manage their health. It looks at how family, friends, and the clinic can support teens to take charge of their care.
Wilmington, DelawareAges 13–21 - NCT05709470Recruiting
Mental health support study for children with heart defects
This study looks at mental health in children and teens (age 5–17) who were born with a heart defect. It may help researchers understand what support is needed and how best to measure mental well-being.
CopenhagenAges 5–17 - NCT05809856Recruiting
Check if a patient’s own valve tissue can be safely used
This study tests whether a new type of heart valve made from your own tissue (an “autologous” valve) can be placed through a catheter safely in adults with leaking and pressure-related problems in the lung heart valve area. If it works well, it could offer a safer option for people who need treatment for pulmonary valve problems.
Berlin, State of BerlinAges 18–80 - NCT06264531RecruitingPhase 2/Phase 3
Bevacizumab for adults with symptomatic, untreatable brain AVMs
This trial tests a medication called bevacizumab (given as an IV infusion) to see if it can improve symptoms in people with brain arteriovenous malformations (AVMs) that cannot be treated with standard therapies. It is for those whose AVMs cause significant disability.
Paris, Île-de-France RegionAges 18 years+ - NCT06253507Enrolling by invitationPhase 1/Phase 2
Gene Therapy for Chronic Granulomatous Disease
This study tests a new gene therapy for people with a rare immune disorder called chronic granulomatous disease (CGD), which causes repeated infections. The treatment uses a modified virus to deliver a healthy gene to your blood stem cells, potentially helping your immune system fight off bacteria and fungi. The goal is to reduce the risk of severe infections and improve overall health.
Bethesda, MarylandAges 3–65 - NCT06259292Recruiting
HHT patient registry tracking long-term outcomes
This registry collects health information from people with HHT over time to better understand the disease and improve care. Joining does not involve any experimental treatment, but your data will help researchers learn more about HHT.
Birmingham, AlabamaAges Any age - NCT06261346Recruiting
Growth factors in corneal transplant surgery
This study tests whether adding a special growth factor solution to the fluid used during corneal transplant surgery helps the transplant heal better. It is for patients undergoing a specific type of corneal transplant called DSEK or DMEK.
Miami, FloridaAges 18–80 - NCT06263075Recruiting
Monitoring during craniosynostosis surgery: comparing newer vs. traditional monitors
This study compares two types of monitors that track your baby's heart and blood flow during surgery to fix craniosynostosis. The goal is to see if a newer monitor works as well as the traditional one.
Bologna, BOAges 3 months–8 months - NCT06275022Recruiting
Using ICG dye to guide treatment of facial birthmarks
This study tests whether a special dye called ICG can help doctors treat a type of facial birthmark called capillary lymphatic malformation (cLM) more effectively. It may help by showing the birthmark more clearly during treatment.
Nanjing, JiangsuAges 4 weeks–16 years - NCT06255886RecruitingPhase 4
Treatment for acid reflux in babies under 1 year
This trial tests a treatment for babies under 1 year old who have frequent and troublesome reflux (GERD). It aims to find a safe and effective way to reduce symptoms like crying, feeding problems, and breathing pauses.
Aabenraa, Region SyddanmarkAges 1 month–1 year - NCT06260891RecruitingPhase 2
Zinc study for sickle cell disease and bone health
This study tests if taking zinc supplements can improve bone health in people with sickle cell disease. It's for those with certain types of sickle cell disease (SS or S-Beta zero thalassemia) who are not on chronic transfusions or zinc supplements.
Oakland, CaliforniaAges 15–40 - NCT06273566Recruiting
Validating a questionnaire for adults with cystic fibrosis
This study is checking if a French questionnaire about physical therapy and daily life works well for people with cystic fibrosis. You would answer questions to help doctors better understand your experience.
CaenAges 18–100 - NCT06261502RecruitingPhase 2
CBD for Anxiety in Fragile X Syndrome
This trial tests if cannabidiol (CBD) can help with anxiety and improve brain function in people with Fragile X syndrome. Participants will receive CBD or a placebo, and may have brain scans (MRI) or magnetic stimulation (TMS) to see how it works.
Sherbrooke, QuebecAges 7–40 - NCT06262282Enrolling by invitation
Phage therapy for stubborn NTM infection in cystic fibrosis
This trial tests whether a special virus therapy (phage) can help clear stubborn NTM lung infections in people with cystic fibrosis who have been on antibiotics for over a year without success. Participants will continue their antibiotics while receiving the phages.
Birmingham, AlabamaAges 6 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.