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
- NCT03612310Recruiting
Study uses stem cells to model genetic diseases
This study looks at how to create laboratory models of genetic diseases using stem cells made from people’s cells. It may help researchers understand disease causes and how treatments could work, using genetic patients (and some non-patient controls).
Edinburgh, MidlothianAges 1–120 - NCT03621137Recruiting
Registry for people with moderate-to-severe eczema
This registry collects information about people with atopic eczema who begin light therapy (phototherapy) or medicine that changes the immune system. It may help doctors better understand how these treatments work in everyday care in the Netherlands.
Amsterdam, North HollandAges Any age - NCT03624374Recruiting
Study of LBSL in people with DARS2 mutations
This trial is a natural history study, meaning it tracks how LBSL (a genetic condition) affects the brain, brainstem, and spinal cord over time. It may help researchers better understand disease patterns and provide information that could support future treatments.
Baltimore, MarylandAges Any age - NCT03626480Recruiting
Nutrition support before and after heart surgery for babies
This trial studies a nutrition plan given around the time of heart surgery for babies with congenital heart disease. It may help improve how babies grow and recover by supporting feeding before and after the operation.
Hangzhou, ZhejiangAges 1 day–1 year - NCT03631420RecruitingPhase 1
Stem cell treatment to prevent lung disease in very premature babies
This Phase 1 trial tests whether giving stem cells into a breathing tube is safe and may help prevent bronchopulmonary dysplasia (BPD), a serious lung condition in very premature infants. It focuses on babies soon after birth who already need breathing support and are at risk.
TainanAges 3 days–2 months - NCT03635645Recruiting
Testing stimulation in people with an implanted retinal prosthesis
This study looks at how retinal stimulation works for people who already have an Argus II retinal prosthesis implanted. It may help researchers understand how well the stimulation creates a visual perception and how reliably people can do the required testing.
Ann Arbor, MichiganAges 25–89 - NCT03657693Recruiting
MRI test in newborns with breathing problems (BPD)
This study uses MRI (a special scan) to look at babies with bronchopulmonary dysplasia, a long-term breathing condition that can happen after being in the NICU. It aims to learn more about what’s happening in the lungs and how MRI can be used safely in these babies.
Cincinnati, OhioAges Up to 4 months - NCT03662178Recruiting
Ultrasound check plan for healthy baby growth during pregnancy
This trial looks at how a structured ultrasound schedule may help track fetal (baby) growth during routine prenatal care. It may benefit patients by improving how pregnancy growth checks are planned and recorded.
Oxford, OxfordshireAges 16–60 - NCT03670472Recruiting
Gene therapy for cystic fibrosis with specific CFTR mutations
This study looks at correcting “stop” mistakes (nonsense mutations) in the CFTR gene, which can cause cystic fibrosis. If your CFTR test shows the exact type of mutation the study needs, you may be able to take part.
AmiensAges 8 years+ - NCT03676868Recruiting
Study of brain blood vessel malformations and outcomes
This study looks at how brain arteriovenous malformations (abnormal connections between arteries and veins in the brain) behave over time, and how different treatment choices affect outcomes. It may help researchers better predict prognosis and guide future care.
ParisAges birth+ - NCT03674372Recruiting
Fetal airway blockage procedure for severe CDH during pregnancy
This trial studies a procedure done before birth that partially blocks the baby’s windpipe to help lung development in babies with diaphragmatic hernia. It may help babies with severe lung underdevelopment, but only if specific pregnancy and baby factors are met.
Aurora, ColoradoAges 18 years+ - NCT03695978Recruiting
Testing new clotting factor for people with untreated or minimally treated Hemophilia A
This study tests how well and how safely Octapharma’s clotting factor products (Nuwiq, Octanate, or Wilate) work in people with Hemophilia A who are newly treated or have had very few clotting factor doses. It also looks at how often side effects occur and how the treatments are used in real care.
Miami, FloridaAges Any age - NCT03698149Recruiting
Brain-signal training for hand and speech control after disability
This study tests whether recording brain signals can help people regain better control of movements in the arms and help with speech. It may be an option for people with significant disability from certain nerve and brain conditions who can safely take part in brain-monitoring procedures.
San Francisco, CaliforniaAges 21 years+ - NCT03702309Recruiting
Banking and testing blood samples to study cancer
This study collects blood samples to “test and store” (bank) information that may help doctors understand cancer in the future. You might be able to join if you’re an adult with confirmed cancer or a higher-than-usual risk for cancer.
Toronto, OntarioAges 18 years+ - NCT03704207Recruiting
Testing a diagnostic workup to guide care for PCD
This study checks whether using “PCD diagnostics” helps clinicians make better care decisions for people being evaluated for PCD. You might be able to join if you’re referred for PCD diagnosis and can complete the study steps.
Nashville, TennesseeAges 1 year+ - NCT03708627RecruitingEarly Phase 1
Bimatoprost for eye swelling in Graves’ orbitopathy
This early study tests whether bimatoprost (eye drops) can reduce signs of Graves’ orbitopathy, a condition that can cause bulging eyes. It may help improve eye symptoms for people whose Graves-related eye problems are present.
Seattle, WashingtonAges 18 years+ - NCT03717844Recruiting
Registry for adults with plasma cell disorders
This study is a registry that collects information about adults with plasma cell disorders (conditions involving abnormal antibody-producing cells). It may help researchers understand these diseases better and improve future care by tracking patients over time.
Chapel Hill, North CarolinaAges 18 years+ - NCT03718403RecruitingPhase 4
Theophylline trial for pseudohypoparathyroidism
This Phase 4 study tests whether the medicine theophylline can help people with pseudohypoparathyroidism type 1A or 1B. It may be considered if you’re trying to improve how your body uses parathyroid hormone and manage related calcium levels.
Boston, MassachusettsAges 5–21 - NCT03721068RecruitingPhase 1
CAR T-cell treatment for hard-to-treat childhood tumors
This Phase 1 study tests an experimental CAR T-cell treatment that targets GD2, with added immune-boosting and safety features, for children with relapsed or refractory neuroblastoma or osteosarcoma. It’s designed to see if the treatment is safe and can shrink these cancers when standard therapy hasn’t worked.
Atlanta, GeorgiaAges 1.5 years+ - NCT03723564RecruitingPhase 1
Serial amnioinfusions for severe fetal kidney underdevelopment
This Phase 1 study tests whether repeatedly adding fluid into the amniotic sac (amnioinfusion) can help babies with very severe kidney problems before birth. The goal is to see if this “regenerative therapy” is safe and potentially beneficial, while closely tracking pregnancy and baby outcomes.
Rochester, MinnesotaAges Any age - NCT03731000Recruiting
Testing a glue-like treatment for abnormal brain vessel leaks in children
This trial studies a device that delivers a special “embolic” material (often described as glue) to treat a type of abnormal connection in the brain’s blood vessels (dAVF). It may help by blocking the leaking or risky blood flow, and it will be compared against how doctors handle eligible cases with other embolic materials.
New York, New YorkAges Up to 21 years - NCT03742011Recruiting
Study of babies born to mothers with PCOS in Guangzhou
This study looks at children born to mothers who have polycystic ovary syndrome (PCOS). It aims to learn about their health by following families in Guangzhou over time.
Guangzhou, GuangdongAges Up to 18 years - NCT03749980Recruiting
Study following people with von Hippel-Lindau (VHL)
This study follows people who have von Hippel-Lindau (VHL) over time to better understand how the condition changes. It may help doctors plan future treatments by collecting long-term health information.
Boston, MassachusettsAges Any age - NCT03760835RecruitingPhase 4
Once-daily hydrocortisone for adults with congenital adrenal hyperplasia
This trial studies whether a once-daily hydrocortisone tablet works as well as standard steroid treatment for adults with congenital adrenal hyperplasia caused by 21-hydroxylase deficiency. It aims to improve how safely and conveniently hormone replacement is taken each day.
NaplesAges 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.