Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,756 recruiting studies in this area right now.
By phase
- Not applicable2,677
- Phase 2277
- Phase 1238
- Phase 3210
- Phase 1/Phase 2208
- Phase 497
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease134
- Atopic Dermatitis125
- Congenital Heart Disease84
- Cystic Fibrosis76
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy37
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)28
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
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
- Massachusetts General Hospital22
Where studies are running
- California325
- Maryland161
- Alabama133
- Massachusetts107
- Arizona86
- New York85
- Texas70
- Minnesota68
- Pennsylvania62
- Florida59
Recruiting studies3,756 total
- NCT07797673RecruitingPhase 1
Stem cell therapy for babies with biliary atresia
This trial tests a new treatment using stem cells from umbilical cord blood or tissue, given to infants with biliary atresia during their Kasai surgery. The goal is to improve liver function and reduce the need for a liver transplant.
Jakarta Pusat, DKI JakartaAges 4 weeks–3 months - NCT07800247Recruiting
Sickle cell disease registry for young patients
This study collects information about children, teens, and young adults with sickle cell disease to better understand the condition in Egypt. It may help improve care and treatments for people with sickle cell disease.
CairoAges Up to 39 years - NCT06527963Recruiting
Balloon types for fistula narrowing in dialysis patients
This trial compares two types of balloons used to open up narrowed areas in the blood vessels of dialysis patients. It aims to find which balloon works better for keeping the fistula working well.
Zhengzhou, HenanAges 18–75 - NCT06091371Recruiting
Using a small implant for flatfoot in growing kids
This trial tests a new implant to treat flexible flatfoot in children. It may help reduce pain and improve foot function during growth.
BolognaAges 8–14 - NCT06534307Recruiting
Long-term follow-up for children from PETN studies
This study checks on the health and development of children whose mothers took part in the PETN studies. It involves questionnaires and a physical exam for kids aged 5 and older.
Tübingen, Baden-WurttembergAges 6–18 - NCT07787975Enrolling by invitation
Understanding genetic test results with RNA sequencing
This study uses a special test called RNA sequencing to find genetic causes of diseases when standard genetic tests didn't give answers. It might help you get a clearer diagnosis or find out if a genetic change actually causes your condition.
San Francisco, CaliforniaAges 2 years+ - NCT07786701Recruiting
Surgery for camptodactyly: skin flap versus graft
This trial tests a surgical method to fix camptodactyly (a bent finger) using a skin flap technique versus a skin graft. It's for people of any age who meet the stiffness criteria and haven't had prior finger surgery.
São Paulo, São PauloAges Any age - NCT07785011Recruiting
Sensory integration for gross motor and balance in children with Down syndrome
This study tests whether sensory integration therapy helps improve gross motor skills and balance in children with Down syndrome. It focuses on kids aged 3-5 who can stand alone and have specific IQ and sensory issues, while excluding those with heart problems, severe cognitive issues, recent leg surgery, obesity, or hip problems.
CairoAges 3–5 - NCT07791069Enrolling by invitation
Study of a device to manage blood flow in the lungs for heart disease in young children
This study looks at how well a new device works to control blood flow to the lungs in children with heart problems. It reviews medical records of children who had this device placed in their lung arteries.
Le Plessis-RobinsonAges Up to 3 years - NCT03240653Recruiting
Study to sort people with Gaucher disease into groups
This study looks at people with Gaucher disease (types I, II, or III) to better “group” patients for understanding and future research. If you (or your child) have confirmed Gaucher disease, you may be asked to share information and follow a study schedule.
BirminghamAges Any age - NCT07788014RecruitingPhase 1
Testing a new medicine for healthy adults
This study tests a new investigational drug called GV-100 to see how safe it is and how the body handles it. It is for healthy adults and will be given in different doses to find the best one.
Adelaide, South AustraliaAges 18–65 - NCT07787091RecruitingPhase 1/Phase 2
First study of ARD001 in healthy people and those with hemophilia A
This study tests a new medicine, ARD001, for the first time in humans. It aims to see if the drug is safe and how the body handles it, and it is also being studied in people with hemophilia A to find a safe dose for future trials.
SingaporeAges 21–45 - NCT07792408RecruitingPhase 1
Study of VX-407 and oral contraceptive interaction
This study tests how a new drug, VX-407, affects the levels of a combined oral contraceptive in your body. Participants must be healthy adults aged 18 to 65 with a certain body weight and BMI.
MontrealAges 18–50 - NCT07785141Recruiting
Monitoring heart oxygen during pediatric open-heart surgery
This study uses a non-invasive light-based technique to monitor oxygen levels in the heart muscle during open-heart surgery in children. It aims to see if this method can help doctors better manage the surgery and improve outcomes.
Helsinki, UusimaaAges 1–17 - NCT05654025Recruiting
Study of eyesight outcomes after congenital lens dislocation
This follow-up study looks at how vision changes over time in children and adults with congenital lens dislocation, and what complications may occur. It may help doctors better predict outcomes and prevent problems after diagnosis.
Guangzhou, GuangdongAges 3 years+ - NCT07782827RecruitingPhase 3
Angel syndrome treatment study for children and adults
This study tests an experimental medicine for Angelman syndrome (AS), a genetic condition affecting the nervous system. The medicine is given by lumbar puncture and aims to improve symptoms.
Carrboro, North CarolinaAges 2–50 - NCT07783425RecruitingPhase 3
Treatment for moderate-to-severe eczema
This study tests a new medicine called Tilrekimig for people with moderate-to-severe eczema (also called atopic dermatitis) whose symptoms have not improved with creams or ointments. The goal is to see if this medicine helps reduce symptoms and improve skin condition.
Phoenix, ArizonaAges 12 years+ - NCT07778836RecruitingPhase 3
Study of daily injections for Friedreich's ataxia
This study tests an investigational drug given as a daily injection to see if it can improve walking and balance in people with Friedreich's ataxia.
Tampa, FloridaAges 12–40 - NCT07776626RecruitingPhase 1
Study of YOLT-203 for Primary Hyperoxaluria Type 1
This study tests a new treatment for primary hyperoxaluria type 1, a rare condition that causes too much oxalate in the body, which can lead to kidney stones and kidney damage. The treatment is given as an injection, and the study will see if it is safe and how the body responds.
Shanghai, Shanghai MunicipalityAges 6 years+ - NCT06385054RecruitingPhase 2
Probiotics for infant colic in breastfed babies
This study tests a probiotic (friendly bacteria) to see if it can reduce crying and fussing in babies with colic. It is for exclusively breastfed infants 8 weeks and younger who have colic symptoms.
Bécon-les-GranitsAges Up to 2 months - NCT06553443RecruitingPhase 3
Comparing coated and plain balloons for dialysis access narrowing
This trial tests whether a drug-coated balloon works better than a plain balloon to open up narrowed spots in your dialysis access. If you have a failing AVF with a narrowing that can be treated, this study may help improve your dialysis.
SingaporeAges 21 years+ - NCT07781566Recruiting
Speech therapy intensity for cleft palate speech errors in children
This study looks at how well two different intensities of speech therapy work for children with a cleft palate who have speech sound errors. It aims to find the best way to help your child speak more clearly.
Ghent, East-FlandersAges 4 years+ - NCT07780981Recruiting
Body position study for endoscopy pressure measurements
This study looks at whether lying on your back or side affects a measurement taken during a routine endoscopy procedure to check how well your esophagus and stomach opening are working. It may help doctors get more accurate readings to guide your care.
Baltimore, MarylandAges 5–21 - NCT07776444Recruiting
Measuring brain blood flow with light and MRI
This study uses a special light-based device and MRI to measure how blood vessels in the brain respond to changes. It may help researchers better understand blood flow in people with and without sickle cell disease.
St Louis, MissouriAges 6–70
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,756 recruiting medical genetics studies from the U.S. registry right now, out of 5,439 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.