Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,863 recruiting studies in this area right now.
By phase
- Not applicable2,776
- Phase 2282
- Phase 1237
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis129
- Congenital Heart Disease85
- Cystic Fibrosis78
- Down Syndrome55
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease39
- Hemophilia A39
- 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 Human Genome Research Institute (NHGRI)23
Where studies are running
- California339
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona77
- Texas72
- Minnesota67
- Pennsylvania66
- Ohio57
Recruiting studies3,863 total
- NCT07732244Recruiting
Testing a wireless brain oxygen monitor in the NICU
This study is testing a new wireless device that monitors oxygen levels in the brain. It is for babies already using a similar wired monitor, to see if the wireless version works just as well.
Montreal, QuebecAges Any age - NCT07730788Recruiting
Skin imaging study in eczema patients receiving nemolizumab
This trial tests whether a special skin imaging technique (reflectance confocal microscopy) can see how nemolizumab changes the skin structure in people with moderate-to-severe eczema. It may help researchers understand how this treatment works.
Romans-sur-Isère, DrômeAges 18 years+ - NCT07605429RecruitingPhase 3
Rugonersen treatment for Angelman syndrome
This study tests a new medicine called rugonersen for people with Angelman syndrome. The medicine is given as an injection into the fluid around the spinal cord and may help improve symptoms by targeting the genetic cause of the condition.
San Diego, CaliforniaAges 1–50 - NCT07228364RecruitingPhase 1
Testing safety of AZD1613 for polycystic kidney disease
This trial tests a new study drug called AZD1613 for adults with a certain type of kidney disease (ADPKD). Researchers want to see if it is safe, how the body handles it, and what the right dose might be.
Birmingham, AlabamaAges 18–65 - NCT07482943RecruitingPhase 4
Dapagliflozin for heart function after Fontan procedure
This trial tests whether dapagliflozin, a diabetes drug that also helps the heart, can improve exercise ability and heart function in people who have had the Fontan procedure for a single ventricle heart defect.
Hong KongAges 12 years+ - NCT07467564Recruiting
Does Dupilumab help anxiety and depression in eczema?
This study looks at whether Dupilumab (a treatment for moderate-to-severe eczema) can also help improve symptoms of anxiety and depression. It is for people with eczema who also have these mood symptoms and are about to start Dupilumab.
DohaAges 12 years+ - NCT07725926Recruiting
Combining therapy techniques for tic-like OCD
This study tests a therapy that combines two approaches—one for tics and one for OCD—to help people whose OCD feels like it's linked to tics. The goal is to see if this combined treatment reduces distressing urges and repetitive behaviors.
London, LondonAges 18–65 - NCT07727200Recruiting
Tooth color and enamel loss in children with MIH
This study looks at how the color of white, yellow, or brown spots on children's first permanent molars (the back teeth that come in around age 6) relates to the breakdown of the enamel (the outer layer). It aims to help dentists better identify and treat this condition early.
Basel, Canton of Basel-CityAges 5–8 - NCT06081348RecruitingPhase 2
Sertraline vs. placebo for anxiety in kids with developmental disorders
This trial compares sertraline (a common antidepressant) to a placebo (a pill with no medicine) to see which better reduces anxiety in children and teens who also have a neurodevelopmental disorder like autism, ADHD, or Tourette's. The goal is to help you and your child understand if this treatment might offer relief from anxiety.
Calgary, AlbertaAges 8–17 - NCT02675959RecruitingPhase 2
Transplant with defibrotide for sickle cell disease
This trial tests a stem cell transplant from a half-matched donor using a strong conditioning regimen and a drug called defibrotide to protect organs. It aims to cure sickle cell disease in patients who have had serious complications like stroke, chest syndrome, or frequent pain episodes.
Los Angeles, CaliforniaAges 6 months–21 years - NCT07727044Recruiting
Color and sensitivity of MIH teeth in children
This study looks at whether the color of spotted first permanent molars (caused by MIH) is linked to tooth sensitivity. It compares children with MIH to children with healthy teeth to understand the connection between color and pain.
Basel, Canton of Basel-CityAges 5–8 - NCT07728032Recruiting
How diet and medicine affect gut health in PKU children
This study looks at how a phenylalanine-restricted diet or certain PKU medicines affect gut health in children with PKU. It also includes healthy siblings or household members as a comparison group.
Birmingham, BirminghamAges 3–17 - NCT06714006RecruitingPhase 1
Testing safety of PYC-003 for ADPKD
This study is testing a new investigational medicine called PYC-003 for people with autosomal dominant polycystic kidney disease (ADPKD). It is one of the first studies in humans, so the main goal is to see if the medicine is safe and tolerable. The study is for people with a specific genetic cause (PKD1 mutation) and with certain kidney imaging types.
Southport, Gold CoastAges 18–65 - NCT06735820RecruitingPhase 1/Phase 2
MEK and MDM2 inhibitors for NF1-related tumors
This trial tests two targeted drugs (selumetinib and milademetan) for people with neurofibromatosis type 1 (NF1) who have either a malignant peripheral nerve sheath tumor (MPNST) or an atypical neurofibromatous neoplasm with uncertain biologic potential (ANNUBP). The goal is to see if blocking certain proteins can shrink or control these tumors.
Washington D.C., District of ColumbiaAges 18 years+ - NCT05153967Recruiting
Study late effects after sickle cell curative treatments
This study looks at long-term health effects after curative treatments for sickle cell disease, such as bone marrow transplant. Your participation helps researchers understand what changes over time after treatment, and what might need monitoring or care.
Washington D.C., District of ColumbiaAges 4–65 - NCT07509229Enrolling by invitationPhase 1/Phase 2
Algae Extract for Retinitis Pigmentosa
This study tests an extract from a type of algae that is rich in a special form of vitamin A. It may help improve vision for people with retinitis pigmentosa caused by certain gene mutations that affect how the eye processes vitamin A.
Tel LitwinskyAges 18 years+ - NCT07426796Recruiting
Comparing two pain blocks for pediatric testicle surgery
This trial compares two types of numbing injections for pain after surgery to fix an undescended testicle in young boys. It aims to find which injection works better for comfort during recovery.
IstanbulAges 1–7 - NCT06415344Enrolling by invitationPhase 3
Long-term extension of GTX-102 in Angelman syndrome
This study is a long-term follow-up for people with Angelman syndrome who have already taken part in a GTX-102 trial. It tests whether the treatment continues to be safe and helpful over a longer period.
Los Angeles, CaliforniaAges Any age - NCT07561125RecruitingPhase 2
IVIG for lung disease in very preterm infants
This trial tests whether giving intravenous immunoglobulin (IVIG) early after birth can help prevent or treat a serious lung condition called bronchopulmonary dysplasia (BPD) in babies born at 28 weeks or earlier. It may help reduce the need for breathing support and oxygen.
ShanghaiAges 1 day–2 months - NCT03610802Recruiting
Send blood samples to study genetic and immune differences
This study collects blood (and sometimes related samples) to learn how genes and the immune system work in people with primary immunodeficiency (PID) or in family members who may not have it. The goal is to improve understanding of rare immune conditions and help guide future care.
Bethesda, MarylandAges 1 day–99 years - NCT07725263Recruiting
Low-dose antifungal for kids with low white blood cells
This trial tests a low dose of an antifungal medicine (liposomal amphotericin B) to prevent serious fungal infections in children whose immune system is weak after cancer treatment or a stem cell transplant. It's for kids aged 3 to 17 who are at high risk for these infections.
Haikou, HainanAges 3–17 - NCT07726771Recruiting
Pouch endoscopy study in FAP patients
This study uses a tiny camera to look at the pouch created after FAP surgery. It aims to see how well the pouch is doing and if any polyps are growing there.
London, LondonAges 18 years+ - NCT07726043Recruiting
Rehab with vibration therapy for balance in CMT1A
This trial tests whether adding a special vibration device to an intensive rehab program helps improve balance in people with Charcot-Marie-Tooth disease type 1A. If you are between 18 and 65 and have mild to moderate leg weakness, you may be able to join.
MarseilleAges 18–65 - NCT07181837RecruitingPhase 1/Phase 2
Testing MVX-220 for Angelman Syndrome
This study tests an experimental medicine called MVX-220 to see if it is safe and effective for people with Angelman Syndrome. It includes both children (ages 4-8) and adults (ages 18-50) who can walk independently and have stable seizure medications.
Los Angeles, CaliforniaAges 4–50
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,863 recruiting medical genetics studies from the U.S. registry right now, out of 5,730 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.