Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,859 recruiting studies in this area right now.
By phase
- Not applicable2,773
- Phase 2283
- Phase 1236
- Phase 3215
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis128
- Congenital Heart Disease85
- Cystic Fibrosis78
- Down Syndrome55
- 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
- California341
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona78
- Texas72
- Minnesota67
- Pennsylvania66
- Ohio57
Recruiting studies3,859 total
- NCT05285345Recruiting
A new discharge plan for very premature babies with lung disease
This trial tests a standardized, team-agreed discharge plan to help preterm babies with lung disease (often called BPD) leave the hospital safely. It may help by making the discharge process more consistent, especially for babies going home on breathing support like oxygen.
Boston, MassachusettsAges birth–1 year - NCT05283759Recruiting
Brugada syndrome registry study at UZ Brussel
This study keeps a registry (a secure list) of people who have been diagnosed with Brugada syndrome. It helps doctors learn about the condition over time and may support future research aimed at better care.
BrusselsAges 18–100 - NCT05284006Recruiting
Non-invasive tests for Morquio A
This trial studies Morquio A (MPS IVA) using non-invasive ways to measure how the body is working, and to better understand why the condition happens. If you have Morquio A, this may help researchers learn more and improve care.
Wilmington, DelawareAges Any age - NCT05285839RecruitingPhase 4
Dupixent plus UVB light for moderate to severe eczema
This study tests whether taking Dupixent and receiving narrowband UVB light can improve moderate to severe atopic dermatitis (eczema). It may help people whose symptoms are significant despite usual care.
East Windsor, New JerseyAges 18 years+ - NCT05285917RecruitingPhase 3
Hydroxyurea safety study for young children with sickle cell
This trial tests a way to use hydroxyurea more safely and effectively in children with sickle cell anemia. It may help doctors fine-tune dosing to reduce complications and side effects.
LuandaAges 6 months–12 years - NCT05287022Recruiting
Nasal nitric oxide test to improve primary ciliary dyskinesia care
This study tests whether a breathing test called nasal nitric oxide can help improve care for people with primary ciliary dyskinesia (PCD) or related lung/sinus problems. The goal is to see if the test can better guide diagnosis and treatment planning.
Little Rock, ArkansasAges 2–99 - NCT05287841Recruiting
Battten graft during septum and turbinate surgery
This study tests whether adding a “batten graft” during septoplasty and turbinate reduction improves nasal breathing and nasal airflow. It aims to see if the graft helps when breathing blockage is mainly from the septum, enlarged turbinates, and the outside nasal valve collapsing.
Poplar Bluff, MissouriAges 18 years+ - NCT05288894Recruiting
Italian registry for people with repaired Tetralogy of Fallot
This trial is a registry that collects information about people who have had Tetralogy of Fallot (TOF) repaired. It may help doctors better understand long-term outcomes and improve future care.
Ancona, The MarchesAges 10 years+ - NCT05293184Recruiting
Angelman syndrome registry to track symptoms worldwide
This trial is building a global registry for people diagnosed with Angelman syndrome. It helps researchers learn more about how symptoms change over time, which can support future studies and treatments.
Brisbane, QueenslandAges Any age - NCT05294978Recruiting
Testing a new light-based treatment for certain eye vision loss
This trial studies a light-guided eye treatment in people with generalized inherited retinal disease (a genetic cause of vision loss). It aims to see whether the approach works safely and can improve function for people whose vision is already significantly reduced.
San Francisco, CaliforniaAges Any age - NCT05295433RecruitingPhase 1/Phase 2
Long-term follow-up for an mRNA vaccine dose you already received
This is a follow-up study that checks the long-term safety and health benefits of mRNA-3705 for people who took it in an earlier trial (or had to pause dosing due to COVID-19 vaccination). You may be invited if you completed the earlier study schedule or can transition early because you missed several doses.
Los Angeles, CaliforniaAges 1 year+ - NCT05299710Recruiting
Store ovarian tissue for future fertility in children
This study looks at freezing (cryopreserving) ovarian tissue before certain cancer treatments or surgeries that may affect fertility. It may help preserve the ability to have children later, especially when future fertility is at risk.
Chicago, IllinoisAges Up to 11 years - NCT05302271RecruitingPhase 1
Testing gene therapy for Friedreich’s ataxia heart disease
This Phase 1 trial tests an AAV gene therapy that aims to improve heart problems caused by Friedreich’s ataxia. You may be eligible if you have genetically confirmed Friedreich’s ataxia with measurable heart weakening but still enough heart function to safely receive treatment.
New York, New YorkAges 12–50 - NCT05315167RecruitingPhase 1/Phase 2
Study drug PRJ1-3024 for advanced solid tumors
This trial tests how safe PRJ1-3024 is and how the body handles it, and it also looks for early signs that it can shrink advanced solid tumors. It may help people whose cancer can’t be treated with standard options.
Zhengzhou, HenanAges 18 years+ - NCT05318222Recruiting
Virtual genetic evaluation for undiagnosed rare disease
This study helps children who have a rare genetic illness but have not yet received a diagnosis. It uses virtual evaluation to try to find answers and possible next steps for care.
Edinburg, TexasAges 1 day–18 years - NCT05321875RecruitingPhase 3
Candesartan to prevent heart failure in certain gene carriers
This trial tests whether candesartan (a blood-pressure medicine) can delay or prevent heart muscle weakening in people who carry a disease-causing dilated cardiomyopathy gene variant but still have fairly strong heart pumping. Participants are randomly assigned to candesartan or a placebo to see which one keeps the heart healthier over time.
Majadahonda, MadridAges 18–64 - NCT05326451Recruiting
At-home brain stimulation for Huntington’s behavior and thinking symptoms
This trial tests an at-home, noninvasive brain stimulation treatment to help with behavioral and cognitive (thinking) symptoms in people with Huntington’s disease. You and a caregiver work together during sessions and questionnaires to see if symptoms improve and stay stable.
Houston, TexasAges 18–80 - NCT05328050Recruiting
Registry for people with achondroplasia or hypochondroplasia
This is a research registry that enrolls people with achondroplasia or hypochondroplasia. It helps researchers collect information about the condition to improve understanding and future studies.
MilanAges Any age - NCT05331105RecruitingPhase 2
Drug for adults with NF1 and non-surgically treatable nerve tumors
This Phase 2 trial tests HL-085 in adults with neurofibromatosis type 1 (NF1) who have a nerve tumor (plexiform neurofibroma) that cannot be removed by surgery and causes symptoms. It aims to shrink or control the tumor by measuring changes on MRI and monitoring side effects.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT05335603Recruiting
Study of adults with liver conditions linked to fatty liver
This study looks at adults with different long-term liver conditions that are associated with fatty liver. It helps researchers better understand these diseases and factors linked to them.
HvidovreAges 18 years+ - NCT05336201RecruitingPhase 2
Brain training program for teens with sickle cell disease
This Phase 2 study tests a cognitive (thinking) training program to help teens with sickle cell disease prepare for the changes in their care as they grow older. It may help with skills like learning, attention, and planning for transition to adult care.
Birmingham, AlabamaAges 10–18 - NCT05340582RecruitingPhase 2
Ibuprofen plus acetaminophen for a heart condition in very premature babies
This Phase 2 study tests whether giving ibuprofen together with acetaminophen improves outcomes for an important heart connection (PDA) in extremely premature infants. It compares how this medicine approach affects the baby’s heart-related measurements and short-term progress.
Newcastle, New South WalesAges Up to 6 months - NCT05347238Recruiting
Dopamine or norepinephrine for low blood pressure in very preterm babies
This trial compares two common medicines—dopamine versus norepinephrine—to treat low blood pressure in very preterm babies who have suspected or confirmed late-onset bloodstream infection (sepsis). It may help doctors find which medicine works better and more safely for this specific situation.
Phoenix, ArizonaAges 5 months–7 months - NCT05348564Recruiting
Family genetic screening methods for heart rhythm risk
This study compares two ways to find relatives who may carry a specific inherited genetic change. It may help your family learn faster who should get follow-up testing and care.
Lancaster, PennsylvaniaAges 18 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,859 recruiting medical genetics studies from the U.S. registry right now, out of 5,745 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.