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
- NCT07013045Enrolling by invitation
Exercise and video games for teens with FMF
This study compares two types of exercise programs for teenagers with Familial Mediterranean Fever (FMF). One program uses structured muscle-strengthening moves, and the other uses fun video games that get you moving. The goal is to see which helps teens feel better and stay active.
IstanbulAges 12–18 - NCT07013292Recruiting
Omaveloxolone for swallowing issues in Friedreich's ataxia
This study looks at whether omaveloxolone (a medication) can help improve swallowing problems in people with Friedreich's ataxia. If you have been taking omaveloxolone for at least 6 months, your data may help researchers understand if it helps with swallowing.
Nice, Alpes MaritimesAges 16 years+ - NCT07010705Recruiting
Digital measures for Huntington's disease trials
This study uses smartphone and wearable sensors to track movement and thinking in people with Huntington's disease. It aims to find better ways to measure how the disease progresses in clinical trials.
Rochester, New YorkAges 25–65 - NCT07004283Enrolling by invitation
Fetal MRI reference ranges for normal brain development
This study uses fetal MRI scans from a large group of healthy pregnancies to create standard growth charts for brain measurements. This helps doctors better understand normal fetal brain development.
Guangzhou, GuangdongAges 18 years+ - NCT07008989Recruiting
Phone app to improve memory in Down syndrome
This study tests a smartphone app called HippoCamera to see if it can help teenagers and young adults with Down syndrome remember everyday events better. You would use the app at home with some help from a parent or guardian.
Chestnut Hill, MassachusettsAges 15–35 - NCT06879431Recruiting
Sleep help before heart surgery for young children
This study tests if helping babies and toddlers with sleep problems sleep better before heart surgery can lower the risk of confusion or agitation after surgery. It is for children aged 0 to 3 years who have trouble sleeping and are scheduled for heart surgery.
BeijingAges 6–12 - NCT06999954Recruiting
Global survey for Shwachman-Diamond syndrome and related conditions
This trial is a global survey and registry for people with Shwachman-Diamond Syndrome and related inherited blood disorders. It aims to connect patients, families, and researchers to better understand these conditions and improve care.
Woburn, MassachusettsAges Any age - NCT06998134Recruiting
Exoskeleton walking training for children and young adults
This trial tests whether a robotic leg exoskeleton can help improve walking in children and young adults with certain conditions (like cerebral palsy, muscular dystrophy, spina bifida, or spinal cord injury) or those with no walking issues. The goal is to find out if the exoskeleton is safe and effective for everyday use, especially in younger people who might lose the ability to walk as they get older.
Bethesda, MarylandAges 5–25 - NCT07001280Recruiting
Garadacimab for hereditary angioedema (HAE)
This study looks at how well a drug called garadacimab works and how safe it is for people with hereditary angioedema (HAE), a condition that causes sudden swelling. It tests the drug in people who are starting it for the first time as part of their regular care.
Phoenix, ArizonaAges 12 years+ - NCT06997588RecruitingPhase 2
Testing alpelisib for overgrowth syndrome in children and adults
This trial is testing a drug called alpelisib for people with PROS (a rare condition that causes parts of the body to grow too much). The drug targets a specific gene change (PIK3CA mutation) that is often the cause. If you have this condition and it's causing serious symptoms, this study might help shrink or slow the overgrowth, improving your quality of life.
St Louis, MissouriAges 2–100 - NCT07002762Recruiting
Burdock root extract for high uric acid without symptoms
This trial tests whether burdock root extract can lower uric acid levels in people who have high uric acid but no symptoms of gout. It may help prevent future health problems related to high uric acid.
KyivAges 35–65 - NCT06998524RecruitingPhase 3
Testing emicizumab for type 3 von Willebrand disease
This study tests whether emicizumab can help prevent or treat bleeding episodes in people with type 3 von Willebrand disease, a rare and severe bleeding disorder. It may offer a new option for those who have not had good control with standard treatments.
Sacramento, CaliforniaAges 1 month+ - NCT06998043RecruitingPhase 2
Phage therapy for cystic fibrosis with pseudomonas lung infection
This trial tests a new treatment using phages (viruses that kill bacteria) for people with cystic fibrosis who have a chronic pseudomonas lung infection. It aims to see if adding phages to your usual care can help control the infection.
Birmingham, AlabamaAges 18 years+ - NCT06993844RecruitingPhase 1/Phase 2
Study of ETX-636 for advanced solid tumors with PIK3CA mutation
This trial tests an experimental drug called ETX-636 in people whose cancer has a specific gene change (PIK3CA mutation) and has not responded to at least one prior treatment. The goal is to see if the drug can shrink tumors or stop them from growing.
Newport Beach, CaliforniaAges 18 years+ - NCT07002398RecruitingPhase 1/Phase 2
Gene therapy for Stargardt disease (VG801)
This trial tests a new gene therapy called VG801 for people with Stargardt disease, a genetic condition that causes vision loss. The treatment aims to improve or slow vision loss by delivering a working copy of the ABCA4 gene to the retina.
Shanghai, Shanghai MunicipalityAges 6 years+ - NCT06994520Enrolling by invitationPhase 2
Topical cannabis extract for eczema itch
This study tests a cannabis-based cream on eczema-related itch over 4 weeks. It may help reduce itching if other treatments haven't worked, but requires pausing other skin products during the trial.
Khon Kaen, Changwat Khon KaenAges 25 years+ - NCT06996756RecruitingPhase 1
Gene therapy for alpha-1 antitrypsin deficiency
This trial tests a gene therapy for people with alpha-1 antitrypsin deficiency who have mild to moderate lung disease. The aim is to see if the treatment can improve lung health and is safe.
New York, New YorkAges 18–70 - NCT06994754Recruiting
Expanding the upper jaw with clear aligners in cleft lip and palate patients
This trial tests whether clear aligners can safely widen the upper jaw in children with cleft lip and palate who still have some baby teeth. It may be an option for those needing jaw expansion without braces.
Cairo, SelectAges 6–12 - NCT07000838Recruiting
Corrective insoles for foot overpronation in healthcare workers
This study tests whether special rubber insoles can help healthcare workers with flat feet (overpronation) reduce foot strain while standing and walking for long shifts. If you work in a hospital or clinic and spend most of your shift on your feet, this may help you understand if the insoles improve comfort and foot alignment.
Milan, ItalyAges 18–65 - NCT07000747Recruiting
Dengue and sickle cell disease study
This study looks at how severe dengue fever affects people with sickle cell disease. It aims to understand why some people with sickle cell get very sick from dengue and how their cells react.
Pointe-à-PitreAges 6–25 - NCT06997042Recruiting
Comparing Two Fistula Creation Techniques for Dialysis
This study compares two surgical techniques for creating an arteriovenous fistula (AV fistula), a special connection between an artery and a vein in the arm that makes dialysis easier. If you need dialysis for advanced kidney disease, this trial aims to see which method works better and lasts longer.
Barcelona, BarcelonaAges 18 years+ - NCT07001189Recruiting
Numbing for sickle cell pain crisis: a study
This trial tests whether numbing a specific area (regional anesthesia) can reduce inflammation and pain during a sickle cell pain crisis. It may offer a new way to manage severe pain without strong medications.
Pointe-à-PitreAges 18 years+ - NCT07001631Recruiting
EMDR for pain relief in children with sickle cell disease
This trial tests if EMDR therapy can help children with sickle cell disease reduce pain interference in daily life. If you or your child has sickle cell disease and experiences significant pain, this study may offer a new way to manage it.
AmsterdamAges 6–18 - NCT06996951RecruitingPhase 3
Sinus treatments in cystic fibrosis with modern modulator therapy
This study looks at how different sinus treatments affect people with cystic fibrosis who are taking modern modulator therapy. It aims to understand which rinses work best for chronic sinus problems.
Indianapolis, IndianaAges 18 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.