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
- NCT06843330Recruiting
Lactate meter accuracy for children with GSD type Ia
This trial tests whether a simple finger-stick lactate meter gives accurate results for children and adults with a specific type of glycogen storage disease (GSD Ia). It aims to find a faster, easier way to monitor health during emergency visits or hospital stays.
Hartford, ConnecticutAges birth–60 years - NCT05957549Recruiting
Tracking early sound perception in young children
This study uses special brain-monitoring tools (fNIRS and EEG) to look at how preschool-aged children process sounds. It may help researchers understand early sound perception differences, especially in children with Fragile X Syndrome.
Cincinnati, OhioAges 2–4 - NCT05200715Recruiting
Registry study for inherited and inflammatory eye and joint diseases
This study is a registry, meaning it collects your medical information to better understand several inflammatory (auto-inflammatory) conditions. It may help doctors and researchers improve diagnosis and future treatments for people with these disorders.
AlgiersAges Any age - NCT06769633RecruitingPhase 2
Study of givinostat in young boys with Duchenne muscular dystrophy
This trial tests an experimental drug called givinostat in boys ages 2 to under 6 years with Duchenne muscular dystrophy (DMD). The study looks at how the drug works in the body and if it is safe, with the goal of finding new treatment options for young children.
BrusselsAges 2–6 - NCT06602869Recruiting
Cellular markers in cystic fibrosis with a certain lung infection
This study looks at special blood markers in people with cystic fibrosis who have or haven't been treated for a lung infection caused by nontuberculous mycobacteria. The goal is to better understand the body's response, which could lead to better treatments.
MontpellierAges 18 years+ - NCT06826612RecruitingPhase 1/Phase 2
Gene therapy trial for early Huntington's disease
This trial tests a gene therapy called SPK-10001 for people with early-stage Huntington's disease. It aims to slow the progression of symptoms by targeting the genetic cause of the disease.
Boston, MassachusettsAges 25–65 - NCT06833983RecruitingPhase 3
Study of GS1191-0445 injection for hemophilia A
This trial tests a new injection called GS1191-0445 for people with hemophilia A. It aims to see if it is safe and works well to prevent or treat bleeding episodes.
Hefei, AnhuiAges 18–65 - NCT06831734Enrolling by invitation
Real-world safety study of Alhemo in hemophilia
This trial will track how well Alhemo works and how safe it is for people with hemophilia A or B in everyday use. If you are 12 or older and your doctor has already decided to start you on Alhemo, this study will collect information to help doctors better understand the medicine's real-world benefits and risks.
ChibaAges 12 years+ - NCT06828770Recruiting
Minima stent system long-term safety study
This study checks the safety of the Minima Stent System over time. It is for people who need a stent to keep their blood vessel open. The study wants to see how the device works in real-world use.
Los Angeles, CaliforniaAges Any age - NCT06830499Recruiting
Sapien 3 valve for younger Chinese patients with severe aortic stenosis
This trial tests a newer artificial heart valve (Sapien 3) placed through a small tube in the leg, for younger people (50–70) with severe aortic stenosis. It aims to see if this less invasive option works well in this age group.
Xi'an, ShaanxiAges 50–70 - NCT06834594RecruitingPhase 4
Bleeding patterns in teens with Turner syndrome on progesterone
This study looks at how different ways of taking progesterone (a hormone given along with estrogen) affect bleeding patterns in teens with Turner syndrome. The goal is to find the best hormone schedule to cause regular, predictable periods.
Kansas City, MissouriAges 12–20 - NCT06832644Recruiting
Heart risk prediction study for healthy adults in Italy
This study tests a new way to predict your future risk of heart disease. It is for healthy adults aged 40-80 who have never had heart problems or diabetes. You will use your email and internet to take part.
Milan, MilanAges 40–80 - NCT06829901Recruiting
Uterine entry technique in laser surgery for twin-to-twin transfusion syndrome
This study looks at how doctors enter the uterus during laser surgery to treat twin-to-twin transfusion syndrome (TTTS), a condition where blood flow between twins is unequal. The goal is to see if a certain entry method leads to better outcomes for the babies.
Houston, TexasAges 18 years+ - NCT06832163Recruiting
Smart weaning from inhaled nitric oxide for newborns with PPHN
This trial tests a new, smart way to slowly reduce (wean) the inhaled nitric oxide gas that helps babies with a serious lung condition called PPHN. If your baby is already responding well to iNO and needs it for at least 24 hours, this study might help make the weaning process safer and more effective.
ShanghaiAges Any age - NCT06834035RecruitingPhase 1/Phase 2
IV IgG for collagen VII antibodies in dystrophic epidermolysis bullosa
This trial tests if intravenous immunoglobulin (IV IgG) can help people with a form of epidermolysis bullosa who have antibodies against collagen VII. It may reduce blistering and improve wound healing.
Redwood City, CaliforniaAges 6 years+ - NCT06828653Recruiting
Comparing 3D-printed vs standard ankle braces for walking
This study compares custom-made ankle-foot braces made with a digital 3D printing method versus the traditional plaster cast method. It aims to find out which type is more comfortable and effective for people with conditions like cerebral palsy, stroke, or spinal cord injury who need a brace to help them walk better.
Mississauga, OntarioAges 8 years+ - NCT06832280Recruiting
Helping parents support their child with hypospadias
This study tests a program to help parents feel more supported when their young child is diagnosed with hypospadias. You and your child may be able to join if you are a parent or guardian, your child is under 6, and you have an upcoming urology visit to discuss hypospadias.
Indianapolis, IndianaAges Any age - NCT06834230RecruitingPhase 4
Testing dotinurad for high uric acid in people with high blood pressure
This study tests a medicine called dotinurad to see if it helps lower high uric acid levels in people who also have high blood pressure. It may be an option if you have not taken uric acid lowering drugs recently or have been off them for over 27 days.
Saga, Saga-kenAges 20 years+ - NCT06832800RecruitingEarly Phase 1
Testing a new tool to manage jaundice in newborns
This trial uses a special breath test (ETCOc) to measure jaundice in newborns, aiming to improve diagnosis and treatment. It may help doctors decide when treatment is needed.
HangzhouAges 4 hours–4 weeks - NCT06826209Enrolling by invitation
Comparing arm function in children with and without hemophilia
This study compares how well children with hemophilia use their arms and hands compared to healthy children. If your child has hemophilia A or B, is on regular treatment, and has no inhibitors or recent bleeds in the arm, they may be able to join.
Ankara, AnkaraAges 7–17 - NCT06820515Recruiting
Blood disorder patient registry for all ages
This study is a registry (a large database) that collects information from people who have or may have a blood disorder. It helps researchers learn more about these conditions and improve care.
Hickory, North CarolinaAges Any age - NCT06818838RecruitingPhase 1/Phase 2
Gene therapy for Type 1 Gaucher disease
This study tests a new gene therapy called LY-M001 for adults with Type 1 Gaucher disease. It aims to help the body produce the missing enzyme and reduce symptoms like low blood counts or enlarged organs.
Guangzhou, GuangdongAges 18–60 - NCT06822933Enrolling by invitation
Remote monitoring study for gout patients
This study tests whether using a smartphone or tablet to monitor your gout from home can help manage the condition. You may qualify if you have had at least one gout flare in the last year and have high uric acid levels.
Oslo, Please SelectAges 18 years+ - NCT06816953Enrolling by invitation
Joint health in severe hemophilia A with prophylaxis
This study is looking at how well joint health can be tracked in people with severe hemophilia A who are receiving regular factor VIII treatment. It uses special markers in the blood and ultrasound to check for early joint changes.
Bangkok, PatumwanAges Any age
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.