Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,858 recruiting studies in this area right now.
By phase
- Not applicable2,771
- Phase 2283
- Phase 1235
- Phase 3216
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome54
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease38
- Hemophilia A38
- 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 Cancer Institute (NCI)23
Where studies are running
- California340
- Maryland161
- Alabama132
- Massachusetts116
- New York84
- Arizona78
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT06659588Recruiting
Study of chronic hepatitis risk in people with immune weaknesses
This study looks at people with certain immune system weaknesses to see if they are at risk for chronic hepatitis caused by a virus in the gut. Researchers want to understand how the immune system affects this infection.
ParisAges Any age - NCT06663358Recruiting
Safety and effectiveness of Elfabrio in Fabry disease patients
This study is for people with Fabry disease who are taking or planning to take the medication Elfabrio (pegunigalsidase alfa). It will look at how safe and effective the treatment is in real-world use.
Birmingham, AlabamaAges 18 years+ - NCT06665997RecruitingPhase 4
Depot shot for pain in women with sickle cell disease
This study tests if a hormone shot (depot medroxyprogesterone acetate) can help reduce pain episodes in women with sickle cell disease. It aims to see if the shot improves pain and markers of inflammation.
Atlanta, GeorgiaAges 18–50 - NCT06666413RecruitingPhase 4
Study of Nexviazyme in children with infantile Pompe disease
This study tests the safety and effects of the enzyme replacement therapy avalglucosidase alfa (Nexviazyme) in children with infantile-onset Pompe disease. It is for children who have had Pompe symptoms since infancy and heart problems at diagnosis.
ShanghaiAges Up to 17 years - NCT06666777Recruiting
Speech therapy and parenting for early communication skills
This study tests a program that combines speech therapy with parenting support to help young children with developmental delays improve their communication and social skills. It is designed for children aged 6 months to 5 years who have developmental challenges, and their parents.
Como, ComoAges 6 months–4.9 years - NCT06666816Recruiting
Watching changes in walking for muscle and nerve diseases
This study watches how people with certain muscle or nerve diseases walk over time. It may help doctors understand changes in walking patterns and is open to adults who can walk and have a genetic diagnosis of a neuromuscular condition.
Bosisio Parini, LeccoAges 18–75 - NCT06667141RecruitingPhase 1
Study of ACR-2316 for advanced solid tumors
This study tests a new targeted drug called ACR-2316 for people with specific advanced solid tumors that have already been treated but are no longer responding. It aims to see how well the drug works and how safe it is, while looking for a tumor that has certain markers (like a protein or genetic change) that may make the drug more effective.
Phoenix, ArizonaAges 18 years+ - NCT06668389RecruitingPhase 4
Testing a diabetes medicine for repaired tetralogy of Fallot
This study tests a type of diabetes medicine (SGLT2 inhibitor) to see if it can help people who had surgery for tetralogy of Fallot feel better and have stronger heart and lung function. It may be a good fit if you are an adult with repaired tetralogy of Fallot and do not have certain other health problems.
Hong KongAges 18 years+ - NCT06669000Recruiting
How long to dissolve gout crystals
This study looks at how long it takes for urate crystals (the cause of gout) to dissolve in the body. It may help if you have gout, are not on uric acid-lowering medicine, and have higher uric acid levels.
ParisAges 18 years+ - NCT06669949Recruiting
Studying the natural history of SPLIS disease
This study collects health information from people with SPLIS to better understand the condition over time. It may include your siblings if they also have SPLIS, and data from patients who have passed away.
San Francisco, CaliforniaAges Any age - NCT06670781Recruiting
Comparing two ways to do the Valsalva maneuver during a heart ultrasound
This trial compares two ways of doing a breathing maneuver (Valsalva) during a heart ultrasound to see if it better detects a common hole in the heart (PFO). If you are already scheduled for this test, the study will help find the best method for you.
Ottawa, OntarioAges 18 years+ - NCT06672237RecruitingPhase 3
Study of NTLA-2001 for hereditary ATTR amyloidosis with nerve damage
This study is testing a new gene-silencing treatment called NTLA-2001 for people with hereditary ATTR amyloidosis that causes nerve damage. It works by stopping the body from making a faulty protein that builds up and harms nerves.
Buenos AiresAges 18–85 - NCT06672913Recruiting
Skin-to-skin care study for premature babies
This study looks at how holding your baby skin-to-skin affects their health and development, for babies born at 32 weeks or earlier.
AnkaraAges 1 day+ - NCT06673290RecruitingPhase 2/Phase 3
Testing different doses of sirolimus for cystic lymphatic malformations
This trial tests different doses of sirolimus (a medication that slows cell growth) to find the best dose for treating cystic lymphatic malformations in children. It may help shrink the abnormal fluid-filled growths and improve symptoms.
Chengdu, SichuanAges 1–18 - NCT06674707Recruiting
Agility training with foot exercises for kneecap pain
This trial tests whether adding foot exercises to agility training can better relieve long-lasting knee pain (patellofemoral pain syndrome) in adults ages 20 to 40. Eligible participants will have had pain behind the kneecap for over 6 months.
Lahore, Punjab ProvinceAges 20–40 - NCT06674811Recruiting
Childhood cancer surgery recovery study
This study looks at how children and young adults recover after surgery for solid tumors. It also includes their caregivers to understand how resilience affects recovery.
Rochester, MinnesotaAges 3–25 - NCT06675266Recruiting
AI for detecting fetal brain issues on ultrasound
This trial uses artificial intelligence to help find brain abnormalities in babies during a routine second-trimester ultrasound. It may improve how doctors spot problems early in pregnancy.
RomeAges 18–60 - NCT06676345Recruiting
Heart Attack Complications Study
This study looks at people who have had a heart attack and developed a related structural problem in the heart (like a tear or valve issue). It aims to learn more about these complications to improve future care.
VareseAges 18 years+ - NCT06676904Recruiting
Platelet transfusion study for very preterm babies
This study looks at the best way to decide when to give platelet transfusions to very premature babies. It aims to help prevent bleeding while avoiding unnecessary transfusions.
Birmingham, AlabamaAges 1 hour–2 days - NCT06677866Recruiting
Group therapy for teens with Fragile X or autism
This trial tests if group cognitive behavioral therapy (CBT) can help teens with Fragile X syndrome or autism spectrum disorder improve social and emotional skills. It looks at how teens with these conditions respond to a structured group program.
RomeAges 13–19 - NCT06678737RecruitingPhase 2
TMS plus behavioral therapy for teens with tics
This study tests whether combining a brain stimulation technique called TMS with behavioral therapy can reduce tics in adolescents and young adults. If you have had tics for at least a year and they are moderate or severe, this trial may be a good fit.
Minneapolis, MinnesotaAges 12–21 - NCT06682208Recruiting
Pain relief catheter vs freeze ablation for pectus surgery recovery
This trial compares two ways to manage pain after the Nuss procedure for pectus excavatum. One uses a special catheter that numbs nerves, the other freezes them. The goal is to see which works better for teens and young adults.
Cincinnati, OhioAges 12–21 - NCT06682819Recruiting
Looking at eye nerve layers in NOHL gene carriers
This study uses a special eye scan (OCT) to look at the layer of nerve fibers in the retina of people who carry (or don't carry) a specific gene change linked to Leber Hereditary Optic Neuropathy (NOHL). The goal is to find early warning signs in the blood that might show who is at risk of losing vision.
Paris, ParisAges 18–60 - NCT06683092Recruiting
Restarting triple therapy with close monitoring for side effects
This trial tests whether carefully restarting a triple-combination CFTR modulator (VTD) is safe and helpful for people with cystic fibrosis who had to stop or change Trikafta because of mental health side effects or liver injury. You will be closely monitored with surveys and daily diaries to ensure your safety.
Birmingham, AlabamaAges 6 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,858 recruiting medical genetics studies from the U.S. registry right now, out of 5,779 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.