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
- NCT05354622Recruiting
Genetic testing study for inherited leg stiffness
This study looks at genes (DNA) to understand an inherited condition that causes gradually worsening leg stiffness. It may help researchers learn what causes the condition and potentially guide future care.
Boston, MassachusettsAges 1 month–30 years - NCT05355415Recruiting
Eye imaging study for outer retina diseases using adaptive optics
This study uses a special, high-detail eye imaging machine to take clear pictures of the outer retina. It may help researchers understand these eye conditions better, and how the eye images look when people have an outer retinal disease.
Bethesda, MarylandAges 21 years+ - NCT05359991Recruiting
Exercise study in children and teens with lung or blood conditions
This study tests how safe and effective a structured exercise program is for children and teens. It also compares results between children with sickle cell disease, cystic fibrosis, and healthy peers (and some teens after COVID-19 infection).
Irvine, CaliforniaAges 10–17 - NCT05360082Recruiting
Brain scan study comparing two PET tracers in Huntington disease
This study compares two different brain imaging tracers to see which one shows Huntington disease brain changes better. It may help improve how doctors measure and track Huntington disease using PET-MRI scans.
LeuvenAges 20–75 - NCT05361811Recruiting
Therapy coaching for parents of children with rare genetic syndromes
This trial tests an “Acceptance and Commitment Therapy” program for parents or legal guardians who feel moderate parenting stress. It aims to help caregivers cope in healthier ways and may also improve how they manage stress while supporting their child.
Bethesda, MarylandAges 18 years+ - NCT05361980Recruiting
Study of safety and results of a pediatric bone implant
This study looks at whether a specific pediatric bone implant is safe and works well for children who need it. By joining, you help researchers track outcomes over time and understand possible implant-related side effects.
Vancouver, British ColumbiaAges birth–25 years - NCT05364294Recruiting
Molecular testing for rare inflammatory (autoinflammatory) diseases
This study uses lab testing to better identify the cause of certain rare inflammatory conditions that happen without infection. Finding the cause may help doctors choose more targeted care for you or your child.
ParisAges 1 week–120 years - NCT05371613RecruitingPhase 2/Phase 3
Testing tividenofusp alfa vs idursulfase for MPS II
This trial compares a new treatment (tividenofusp alfa) to the current standard treatment (idursulfase) in children and young adults with MPS II. It’s looking to see which option works better and is safe, especially based on age and whether the disease affects the brain.
Oakland, CaliforniaAges 2–25 - NCT05373498Recruiting
Registry for children’s heart emergencies after resuscitation
This trial is a registry that collects information about children (under 18) who have congenital heart problems and need resuscitation with chest compressions. It helps doctors understand these emergencies and may improve future care for similar patients.
Leipzig, SaxonyAges Up to 18 years - NCT05373771Recruiting
Care plan for sickle cell pain crises in the ER
This trial studies an approach to improve care during emergency room visits for uncomplicated sickle cell pain crises. It may help by guiding how pain is treated in the ER and tracking outcomes for people with sickle cell disease.
Milwaukee, WisconsinAges Up to 18 years - NCT05376046Recruiting
Study blood features and clot risk in sickle cell disease
This study looks at differences in red blood cells and how likely they are to form clots in people with sickle cell disease. The goal is to better understand clot risk and what blood markers may predict it.
RouenAges 18 years+ - NCT05378555RecruitingPhase 3
Oral ketamine for sickle cell pain crises
This trial tests whether taking ketamine by mouth can help treat “vaso-occlusive” pain episodes that may require hospital admission. If it works, it could offer an easier way to manage severe pain during sickle cell crises.
Boston, MassachusettsAges 12–24 - NCT05383911Recruiting
Sickle cell care plan choices for teens and young adults
This trial tests a way to help teens and young adults with sickle cell disease (SCD) and their caregivers actively take part in treatment decisions. It focuses on improving shared decision-making through interviews and a partner-centered approach.
Wilmington, DelawareAges 15 years+ - NCT05384821RecruitingPhase 1/Phase 2
Oral low-dose chemotherapy for relapsed Wilms tumor
This Phase 1/2 trial studies metronomic (very low, steady) chemotherapy taken by mouth or through a feeding tube for children with Wilms tumor that has come back or not responded to earlier treatment. It aims to shrink measurable tumors with better long-term tolerability than standard chemotherapy.
AmiensAges 1.5 years–17 years - NCT05386134Recruiting
Adaptive eye imaging for inherited and acquired retinal conditions
This study tests a special high-detail camera that takes clearer pictures of the retina (the light-sensing layer at the back of the eye). It may help researchers better understand different retinal diseases—and your results could support future diagnosis and care.
Toronto, OntarioAges 5–70 - NCT05386173Recruiting
Tests fetal aortic valve balloon surgery for severe narrowing
This trial studies whether doing a procedure before birth to open a baby’s aortic valve improves outcomes. It compares this approach in a specific group of pregnancies with severe heart flow problems seen on ultrasound.
San Francisco, CaliforniaAges 5 months–7 months - NCT05387954RecruitingPhase 3
PFO closure or blood thinners after stroke in ages 60–80
This trial studies the best way to prevent another stroke after a recent stroke in people ages 60 to 80 who have a “PFO” heart connection. It compares closing the PFO vs taking mouth blood-thinners or antiplatelet medicine, based on what your doctors determine is safest for you.
AmiensAges 60–80 - NCT05390801Recruiting
Congenital aniridia patient survey for adults and families
This trial collects information from people born with congenital aniridia (a rare eye condition) using a questionnaire or survey. It may help researchers better understand patient needs and experiences to improve future care.
ParisAges Any age - NCT05391061Recruiting
Cibinqo tablet study for moderate-to-severe atopic dermatitis
This study looks at Cibinqo (an oral medicine) in people with moderate-to-severe atopic dermatitis (eczema) who are already going to start it under their clinician’s standard care plan. It may help doctors learn about how the medicine works and fits into real-world treatment.
SeoulAges 12 years+ - NCT05392894RecruitingPhase 3
Transplant using a half-matched donor for severe sickle cell
This trial tests whether a half-matched (haploidentical) blood stem cell transplant can treat adults with severe sickle cell disease. It may help reduce serious complications and improve survival, but it requires a fit health status and specific donor and medical conditions.
LondonAges 18 years+ - NCT05394363Recruiting
Victoria children birth-cohort study with parent/guardian consent
This study follows children born in a specific 2-year window and their parents or guardians over time. It helps researchers understand how children and families change as they grow, which can guide future health supports in Victoria.
Parkville, VictoriaAges 1 day+ - NCT05394506Recruiting
Study of factors that affect muscle laminopathy with LMNA changes
This study looks at what influences the course of a muscle disease caused by an LMNA genetic change (a type of laminopathy). It may help researchers better understand the disease and how it affects muscle and sometimes breathing or heart function.
Bron, Auvergne-Rhône-AlpesAges 2 years+ - NCT05395195RecruitingPhase 3
Erythropoietin for newborn brain injury in low-resource settings
This Phase 3 trial studies whether erythropoietin (a medicine related to red-blood-cell growth) can improve outcomes for newborns with moderate to severe brain injury after birth. The study focuses on babies born in low- and middle-income countries and aims to reduce harm from this early brain condition.
DhakaAges 1 hour–6 hours - NCT05399745Recruiting
Study of biliary atresia in children with heart disease
This trial is studying children who have biliary atresia, a serious liver problem, including some who also have tetralogy of Fallot (a congenital heart condition). It also includes healthy children for comparison, and may help researchers understand the illness and treatments better.
CopenhagenAges birth–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.