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
- NCT06617897RecruitingPhase 3
Fibrinogen concentrate to control bleeding in PMP surgery
This trial tests a medicine called fibrinogen concentrate (CSL511) to see if it helps control bleeding during a special surgery (cytoreductive surgery with HIPEC) for pseudomyxoma peritonei (PMP). The goal is to reduce the need for blood transfusions and improve safety.
BasingstokeAges 18 years+ - NCT06618638Recruiting
Shared Decision Tool for ADPKD Treatment
This study helps people with ADPKD and moderate kidney damage decide together with their doctor whether to start a treatment called tolvaptan. It tests a shared decision-making tool to see if it helps patients understand their options better.
Anyang-si, Gyeonggi-doAges 18–80 - NCT06619197Recruiting
Reflexology in Sickle Cell Disease Study
This study tests if foot reflexology, a gentle pressure technique on your feet, can help relieve pain from a sickle cell crisis while in the hospital. It is only for children and teens under 18.
BronAges Up to 18 years - NCT06622603RecruitingPhase 4
Testing lower doses of febuxostat in well-controlled gout
This trial tests if gradually lowering the dose of the gout medication febuxostat is safe for people whose gout has been well-controlled for at least 5 years. The goal is to see if a lower dose can still keep uric acid levels in a healthy range.
Seongnam-si, Gyeonggi-doAges 19–79 - NCT06623890Recruiting
Long-term safety of omaveloxolone for Friedreich's ataxia
This study looks at the long-term safety of omaveloxolone (BIIB141) for people with Friedreich's ataxia. You can join if you are just starting this medicine or have been taking it for less than a year.
Los Angeles, CaliforniaAges 16 years+ - NCT06626308Recruiting
Early signs in Huntington’s gene carriers study
This study looks for early changes in the body and brain of people who carry the Huntington’s disease gene but don’t yet have symptoms. It may help find better ways to track the disease early.
GrenobleAges 18–70 - NCT06626672Recruiting
Digital Health Program for Kids with Heart Defects
This study uses digital tools to promote healthy habits in children (ages 8–14) with congenital heart disease. It aims to support both the child and their family in staying active and well.
MünchenAges 8–14 - NCT06627335Recruiting
Eczema creams and pregnancy outcomes study
This study looks at women who used a topical eczema cream called ruxolitinib (Opzelura) or steroid creams during pregnancy. The goal is to compare pregnancy outcomes and baby health up to one year after birth.
Morrisville, North CarolinaAges 14 years+ - NCT06627374Recruiting
Ultrasound to check duct closure in premature babies
This study uses ultrasound to see if a heart blood vessel called the ductus arteriosus has closed after treatment in premature infants. It aims to find a better way to monitor the response to treatment without X-rays.
Roma, romaAges Up to 4 weeks - NCT06628128RecruitingPhase 3
Long-term study of JNT-517 for people with PKU
This study tests a new medicine called JNT-517 for people with phenylketonuria (PKU) over a long period. It aims to see if the drug can help manage PKU safely and effectively.
Gainesville, FloridaAges 4 years+ - NCT06628271Recruiting
Breath-holding Spells Study for Young Children
This study looks at a common condition in young children called breath-holding spells. It aims to better understand how to manage these episodes, which can be scary but are usually harmless.
EslövAges birth–5 years - NCT06628375Recruiting
Studying chemicals in the environment and hypospadias
This study tests whether certain chemicals in the environment (called endocrine disruptors) might be linked to hypospadias, a condition where a baby boy's penis opening is on the underside instead of the tip. By testing hair samples from mothers, researchers hope to find connections that could help prevent this condition in the future.
Montpellier, HéraultAges 1 month+ - NCT06628687Recruiting
Study checking omaveloxolone health effects during pregnancy and breastfeeding
This study follows people with Friedreich's ataxia who took omaveloxolone during pregnancy or while breastfeeding, to check how the drug affected their health and their baby's health. It helps doctors understand the safety of this treatment for mothers and babies.
Morrisville, North CarolinaAges 16 years+ - NCT06628947RecruitingPhase 2
Testing KIO-301 eye injections for advanced retinitis pigmentosa
This trial tests an eye injection called KIO-301 for people with late-stage retinitis pigmentosa (a genetic eye disease that causes vision loss). The goal is to see if it can improve vision or slow further vision loss.
Sydney, New South WalesAges 18 years+ - NCT06629376RecruitingPhase 1
Testing a new gout drug for safety and effect on uric acid
This trial tests an experimental drug, SSS11, to see if it is safe and how it affects uric acid levels in people with gout and high uric acid. You may not take other gout medicines during the study.
ShanghaiAges 18–65 - NCT06630650Recruiting
Study of Congenital Myasthenic Syndrome (CMS) Over Time
This study follows people with congenital myasthenic syndrome (CMS) over time to learn more about how the condition changes and to find better ways to measure symptoms. You will not receive any treatment, but your participation helps researchers understand CMS better.
Bethesda, MarylandAges 6 months–99 years - NCT06631534RecruitingPhase 4
Testing dexmedetomidine for kids having heart defect closure
This trial tests adding a sedative medicine called dexmedetomidine to standard anesthesia for children aged 3 to 12 who are having a procedure to close a hole in their heart (atrial septal defect). The goal is to see if it helps them recover more smoothly.
Alexandria, Alexandria GovernorateAges 3–12 - NCT06632834RecruitingPhase 2
Targeted treatment study for dilated cardiomyopathy
This study tests whether a personalized treatment approach can improve heart function in people with dilated cardiomyopathy (a condition where the heart becomes enlarged and weak). It focuses on patients who still have low heart function despite standard medications.
Taipei County, Zhongzheng DistrictAges birth–99 years - NCT06633003Recruiting
Testing an oral extract for spinocerebellar ataxia
This trial tests whether an oral extract from a mushroom called Antrodia cinnamomea can help improve symptoms in people with spinocerebellar ataxia (a condition that affects coordination and balance). Participants must be able to communicate in Chinese.
TaipeiAges 30–70 - NCT06634108RecruitingPhase 1/Phase 2
Genistein for heart failure from ATTR amyloid cardiomyopathy
This trial tests whether genistein, a plant-based supplement, can help people with a specific type of heart disease called ATTR amyloid cardiomyopathy. You might join if you have heart failure with reduced pumping function and this rare protein condition.
London, OntarioAges 40–80 - NCT06634628RecruitingEarly Phase 1
Testing a new brain scan tracer for Huntington's disease
This trial tests a new radioactive tracer called [11C]CHDI-00491009 that may show mutant huntingtin protein in the brain using a PET scan. It aims to find out if the tracer is safe and helps see Huntington's disease changes. If you have early-stage Huntington's or are a healthy volunteer, you may be able to join.
LeuvenAges 18–64 - NCT06635902RecruitingPhase 2
Citrulline for sickle cell pain crisis
This trial tests if an intravenous medication called L-citrulline can help treat pain crises in children and young adults with sickle cell disease. Participants receive either the study drug or a placebo while in the hospital for a severe pain episode.
Washington D.C., District of ColumbiaAges 4–21 - NCT06636240Recruiting
Mulberry for eczema in children
This study tests whether mulberry extract could help improve eczema symptoms in children. It is a small, early trial to see if this natural prebiotic is safe and helpful.
New Taipei City, TaiwanAges 6–17 - NCT06636383Recruiting
A follow-up study for people with GSDIa who received DTX401
This study monitors the long-term health of people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401, either in a previous clinical trial or after it was approved. The goal is to gather more information about their condition over time.
Orange, CaliforniaAges 2 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.