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
- NCT06928246RecruitingPhase 1
Botox for Eczema: A Skin & Nerve Study
This trial tests whether Botox injections can improve eczema by calming nerve-immune system interactions. It is for adults with mild-to-moderate eczema who have not used certain stronger treatments recently.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06928727Recruiting
Eye health in craniosynostosis study
This study looks at eye features in people with craniosynostosis (a condition where skull bones fuse too early). It may help doctors better understand eye problems that can occur with this condition.
AmiensAges Any age - NCT06929572Recruiting
Umbilical cord patch for closing spine birth defects after birth
This trial tests using a patch made from a newborn's own umbilical cord to help close an open spine defect (myelomeningocele or myeloschisis) shortly after birth. The goal is to protect the spinal cord and improve healing.
Houston, TexasAges Up to 1 day - NCT06930274Recruiting
Ultrasound Study to Predict Stillbirth Risk
This study uses a special ultrasound to look for signs that might help predict stillbirth risk. If you join, you'll have an extra ultrasound and your pregnancy will be followed closely to see if this test can help keep babies safer.
Vancouver, British ColumbiaAges 19 years+ - NCT06930365Recruiting
Moisturizer study for eczema and healthy skin
This trial compares two moisturizers for people with eczema (moderate rash) and healthy volunteers. It looks at how well they work and how the skin tolerates them after using a steroid cream.
Batumi, AdjaraAges 18 years+ - NCT06930417Recruiting
Williams syndrome and related 7q11.23 changes study
This study follows people with Williams syndrome, 7q11.23 duplication, or related genetic changes over time to understand how these conditions affect health and development. It also includes close family members to learn about the genetics.
Philadelphia, PennsylvaniaAges Any age - NCT06930703RecruitingPhase 1/Phase 2
CBD for sickle cell pain
This trial tests if cannabidiol (CBD) can help reduce pain interference in adults with sickle cell disease. You will take CBD daily for 4 weeks and report your pain levels.
Manhattan, New YorkAges 18 years+ - NCT06932081Recruiting
Study of SGLT2i in adults with congenital heart disease
This trial is a registry that follows adults with congenital heart disease who have started taking an SGLT2 inhibitor medication. The goal is to learn how well this treatment works for this group of patients.
Baltimore, MarylandAges 18 years+ - NCT06933212Recruiting
Mediterranean diet for CADASIL and CAA
This trial is testing whether a Mediterranean diet (rich in fruits, vegetables, whole grains, and healthy fats) can help people with CADASIL or cerebral amyloid angiopathy (CAA) — two conditions that affect blood vessels in the brain. If you have one of these diagnoses, the study may help you understand how diet can support your brain health.
MilanAges 18 years+ - NCT06935253Recruiting
AI to Improve Cardiology Care
This trial tests whether large language models (a type of AI that understands human language) can help cardiologists give better care. If you are a qualified cardiologist who currently treats patients, you may be eligible.
Palo Alto, CaliforniaAges 18 years+ - NCT06935799Recruiting
Freezing for 1 vs 2 minutes after Nuss surgery
This study compares how well one minute of freezing (cryoanalgesia) works versus two minutes to control pain after Nuss surgery for pectus excavatum. It will help find the best way to freeze nerves to reduce pain after surgery.
Warsaw, Wybierz WojewództwoAges 12–18 - NCT06936163Recruiting
Surgery for foot deformities in hereditary spastic paraplegia
This study looks at how well surgery works for foot problems like tight heel cords or clubfoot in people with hereditary spastic paraplegia (HSP), a condition that causes leg stiffness. It aims to see if surgery can improve walking, reduce pain, and prevent falls.
Shanghai, Shanghai MunicipalityAges 10–45 - NCT06936787RecruitingPhase 1
Study of IGT001 for retinitis pigmentosa
This trial tests an experimental drug called IGT001 for people with retinitis pigmentosa, a genetic eye disease that causes vision loss. The goal is to see if it is safe and can help slow or improve vision.
ShanghaiAges 18 years+ - NCT06938100Recruiting
CADASIL Study: Genes, Brain Scans and Symptoms
This study looks at how genetic and brain imaging changes relate to symptoms in people with CADASIL, a genetic condition that affects small blood vessels in the brain and can cause strokes. It aims to better understand the disease to improve future care.
MilanAges 18 years+ - NCT06940336RecruitingPhase 4
Testing tafamidis for transthyretin amyloid polyneuropathy
This trial tests whether the drug tafamidis can help people with a specific type of nerve damage caused by abnormal protein buildup. It aims to slow or improve symptoms in adults with this condition.
JinanAges 18–80 - NCT06940531Recruiting
Tracking causes of lung flare-ups in cystic fibrosis
This study aims to understand what causes and increases the risk of lung infections (exacerbations) in people with cystic fibrosis. It involves regular monitoring and sample collection at home and in the clinic to identify early warning signs.
Manchester, ManchesterAges 5 years+ - NCT06940713Recruiting
Brain imaging study for newborn infants
This study uses a safe, non-invasive ultrasound technique to measure brain activity in newborn babies. It aims to understand brain development and connectivity in preterm infants, infants with birth-related oxygen deprivation, growth-restricted babies, siblings of children with autism, and healthy full-term babies.
ParisAges Up to 5 months - NCT06941025Recruiting
Safety of Elfabrio in pregnant and breastfeeding women with Fabry disease
This study follows women with Fabry disease who took the medicine Elfabrio during pregnancy or breastfeeding to see if it is safe for them and their babies. It helps doctors learn more about how this treatment affects mothers and infants.
Washington D.C., District of ColumbiaAges Any age - NCT06941389Recruiting
Comparing stem cell transplant vs standard therapy for sickle cell disease
This study compares two treatment approaches for children with sickle cell anemia: a stem cell transplant from a matched family donor versus continuing regular disease-modifying medicines. It aims to see which treatment works better over time.
Birmingham, AlabamaAges 3–20 - NCT06941532Recruiting
Study of GMP powder for PKU and tyrosinemia
This trial tests a new powdered protein substitute (GMP) for people with PKU or tyrosinemia. It aims to see if it is a safe and effective option to help manage their condition.
BirminghamAges 3 years+ - NCT06941870RecruitingPhase 4
Efanesoctocog Alfa Preventative Treatment for Hemophilia A with Joint Thickening
This study tests a new preventive treatment (efanesoctocog alfa) for people with hemophilia A who have joint lining thickening. The goal is to see if it helps protect joints.
Los Angeles, CaliforniaAges 12 years+ - NCT06941662Recruiting
Huntington's disease biobank: remote monitoring study
This study is building a biobank to better understand Huntington's disease by using remote monitoring and deep phenotyping (collecting detailed health information). It may help develop better ways to track the disease and improve care for people with the Huntington's gene expansion.
Palo Alto, CaliforniaAges Any age - NCT06942572RecruitingPhase 1/Phase 2
Gene therapy trial for Stargardt disease
This trial tests a new gene therapy called SB-007 for people with Stargardt disease (STGD1). It is given as an injection under the retina to see if it is safe and can help slow vision loss.
Aurora, ColoradoAges 12–65 - NCT06943170Recruiting
Aerobic exercise with or without a low calorie diet for teens with Down syndrome
This trial studies how aerobic exercise, alone or combined with a low calorie diet, affects body fat and weight in children with Down syndrome who are overweight or obese. The goal is to find a safe, effective way to help manage weight.
Lahore, Punjab ProvinceAges 8–16
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.