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
- NCT06969586Enrolling by invitation
Eye drops study for Fuchs corneal disease and glaucoma
This study tests whether special eye drops (Rho-kinase inhibitors) can improve the health of the cornea in people who have both Fuchs endothelial corneal dystrophy and glaucoma. The drops may help reduce corneal swelling and preserve your vision.
Zagreb, CroatiaAges 40–90 - NCT03674372Recruiting
Fetal airway blockage procedure for severe CDH during pregnancy
This trial studies a procedure done before birth that partially blocks the baby’s windpipe to help lung development in babies with diaphragmatic hernia. It may help babies with severe lung underdevelopment, but only if specific pregnancy and baby factors are met.
Aurora, ColoradoAges 18 years+ - NCT06081036Recruiting
Normal brain volumes from fetal MRI scans
This study looks at past fetal MRI scans to create a map of normal brain growth in babies before birth. It may help doctors better understand what healthy brain development looks like.
MarseilleAges 5 months–9 months - NCT06959251Recruiting
Glycine and magnesium with thiamine for primary ciliary dyskinesia
This trial tests whether a combination of glycine, magnesium, and thiamine can help people with primary ciliary dyskinesia (a condition that affects the tiny hairs in your lungs). It aims to see if these supplements improve breathing and reduce lung problems.
Mexico CityAges 5 years+ - NCT06959771RecruitingPhase 1/Phase 2
Gene therapy for CD40L deficiency (Hyper-IgM syndrome) - single patient study
This trial tests a new gene therapy that uses a person's own blood stem cells and T cells, which are corrected with a gene-editing tool called base editing. It is designed for people with a specific CD40L gene mutation who also have liver problems and portal hypertension. The goal is to see if this treatment can improve their immune system and liver health.
Bethesda, MarylandAges 37–120 - NCT06952842RecruitingPhase 1/Phase 2
Gene therapy trial for retinitis pigmentosa with RHO mutation
This trial tests a new gene therapy called ZVS203e for people with retinitis pigmentosa caused by a specific RHO gene mutation. It aims to see if the treatment is safe and can help improve vision.
Beijing, Beijing MunicipalityAges 18 years+ - NCT06953466RecruitingPhase 2/Phase 3
A lotion study for people with Netherton syndrome
This trial tests a lotion called QRX003 for Netherton syndrome, a rare skin condition. It aims to see if the lotion is safe and helpful for managing symptoms.
Chicago, IllinoisAges 14 years+ - NCT06953505Recruiting
At-home ammonia monitoring for inherited metabolic disorders
This trial tests a device for measuring ammonia levels at home in people with rare metabolic conditions that cause high ammonia. It aims to make monitoring easier and more convenient for patients.
Portland, OregonAges 12 years+ - NCT06961565RecruitingPhase 1
Study of PAS-004 for adults with NF1 and plexiform neurofibromas
This trial tests an oral medication called PAS-004 for adults with Neurofibromatosis Type 1 who have at least one symptomatic plexiform neurofibroma that cannot be surgically removed. The goal is to see if the drug can shrink these tumors or improve symptoms, and to understand side effects.
Birmingham, AlabamaAges 18 years+ - NCT06960213RecruitingPhase 3
Study of ADX-324 for preventing HAE attacks
This study tests a new medicine called ADX-324 to see if it can prevent HAE attacks in adults with type I or II HAE. If you've had attacks recently and are willing to pause your current prevention medication, this trial may be an option.
Litchfield Park, ArizonaAges 18 years+ - NCT06958120Recruiting
Comparing two pain blocks for children's heart surgery
This study compares two types of numbing injections given with ultrasound guidance to reduce pain after heart surgery in children. It may be an option for children having certain types of heart surgery through a chest incision.
CairoAges 6 months–4 years - NCT06957691RecruitingPhase 2
Fezolinetant for hot flashes in men on hormone therapy
This study tests a medication called fezolinetant to see if it reduces hot flashes in men undergoing hormone therapy for prostate cancer. If you experience frequent hot flashes, this treatment might help alleviate them.
Boston, MassachusettsAges 40 years+ - NCT06954142Recruiting
Fluid levels to prevent lung disease in preterm babies
This trial compares giving less fluid versus the usual amount to very early preterm babies to see if it helps prevent bronchopulmonary dysplasia, a chronic lung condition. The goal is to find the safest way to manage fluids and protect their developing lungs.
AarauAges 1 week+ - NCT06960902Recruiting
Study of Heart Artery Plaque in Familial Hypercholesterolemia
This study uses a heart CT scan to look for plaque buildup in the arteries of people with familial hypercholesterolemia (a genetic high-cholesterol condition). It aims to understand why some people with FH develop heart disease while others don't.
DijonAges 40 years+ - NCT06955260RecruitingPhase 3
Empagliflozin for Fontan circulatory failure
This trial tests if a diabetes medicine called empagliflozin can help people with Fontan circulatory failure feel better and do more of their daily activities.
Vancouver, British ColumbiaAges 18 years+ - NCT06952933Recruiting
Trauma and resilience in adults with congenital heart disease
This study surveys adults with congenital heart disease to understand how psychological trauma and resilience affect their health. You would complete online questionnaires about your experiences and coping.
Washington D.C., District of ColumbiaAges 18 years+ - NCT06962306RecruitingPhase 4
Pain relief study for children having cleft palate surgery
This trial tests different ways to manage pain after cleft palate repair in young children. The goal is to find which approach works best to lower pain and improve recovery.
Durham, North CarolinaAges 6 months–4 years - NCT06953284Enrolling by invitation
Tape or electrical stimulation for ankle stiffness in CP
This trial compares kinesiotaping (a special stretchy tape) to functional electrical stimulation (mild electrical pulses to muscles) for treating equinus deformity (walking on tiptoes) in children with cerebral palsy. It aims to see which method helps improve ankle movement and walking better.
TantaAges 4–10 - NCT06954909Enrolling by invitation
Hydration self-assessment for firefighters
This study looks at how well firefighters can check their own hydration using urine color. You may help by providing urine and blood samples over a few days.
Phoenix, ArizonaAges 18–65 - NCT06961591Recruiting
Cooking skills for long-term weight loss in young adults with ID
This program teaches cooking skills to help young adults with intellectual disabilities lose weight and keep it off for 2 years. You’ll learn to prepare healthier meals with support from a family member or caregiver.
Kansas City, KansasAges 18–35 - NCT06313333Recruiting
National registry for moderate to severe adult atopic dermatitis
This registry tracks adults with moderate to severe atopic dermatitis (eczema) over time. It helps doctors learn more about the disease and how treatments work in real life.
Brescia, BresciaAges 18 years+ - NCT06944067Recruiting
Genetic risk of immune response after blood transfusions in sickle cell disease
This study looks at whether certain genes make people with sickle cell disease more likely to develop an immune reaction to blood transfusions. It aims to help doctors predict transfusion risks and improve care.
Bethesda, MarylandAges 2–99 - NCT06120582RecruitingPhase 1/Phase 2
Gene therapy safety study for severe hemophilia B
This study tests a new gene therapy called ANB-002 for men with severe hemophilia B (factor IX levels at or below 2%). The goal is to see if it's safe and how well it might help your blood clot normally.
HomyelAges 18 years+ - NCT06945081Recruiting
Measuring skin elasticity in Wilson's disease patients on D-Penicillamine
This study checks if D-Penicillamine, a treatment for Wilson's disease, causes skin damage by measuring skin elasticity on the forearm and cheek. It compares results between patients taking the drug and healthy volunteers matched by age and sex.
BronAges 12 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.