Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,762 recruiting studies in this area right now.
By phase
- Not applicable2,682
- Phase 2280
- Phase 1239
- Phase 3209
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease133
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy36
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)29
Leading sponsors
- Assistance Publique - Hôpitaux de Paris57
- Mayo Clinic39
- Boston Children's Hospital38
- National Institute of Allergy and Infectious Diseases (NIAID)38
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Children's Hospital Medical Center, Cincinnati31
- Hospices Civils de Lyon29
- Children's Hospital of Philadelphia23
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
Where studies are running
- California324
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York84
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT06881771Recruiting
Tracking Fuchs' dystrophy progression and genetics in the UK
This study follows people with Fuchs' endothelial corneal dystrophy over time to see how the condition changes and how it relates to their genes. It aims to help doctors better predict and manage the disease.
LondonAges 18 years+ - NCT05546593Recruiting
Ultrasound check for joint and tendon problems in gout
This study uses ultrasound to look for hidden changes in joints and tendons in people who have gout. It may help doctors better understand what ultrasound can show in gout, but it doesn’t sound like it’s testing a new medicine.
Boulogne-Billancourt, APHPAges 18 years+ - NCT07282847RecruitingPhase 1/Phase 2
Gene therapy for adults with Pompe disease
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
Phoenix, ArizonaAges 18–65 - NCT07285005RecruitingPhase 3
Study of an oral drug for venous, lymphatic, and certain overgrowth syndromes
This trial tests whether the study drug KP-001 can help reduce symptoms like pain, fatigue, or bleeding caused by certain vascular malformations (abnormal blood vessel growths) that can't be surgically removed or haven't responded to other treatments. It's for people age 2 and older with specific conditions such as common venous malformations, lymphatic malformations, or KTS/CLOVES syndrome.
Charleston, South CarolinaAges 2 years+ - NCT07282210RecruitingPhase 2
New medicine for sickle cell hemolysis
This trial tests a new medicine (SIL-8301) to help control red blood cell breakdown (hemolysis) in people with sickle cell disease. It is for those who are already taking hydroxyurea and have had few pain crises but still have complications from hemolysis.
Toronto, OntarioAges 16–35 - NCT07281079RecruitingPhase 3
Testing NNZ-2591 for children with Phelan-McDermid syndrome
This trial tests a new medicine, NNZ-2591, to see if it helps children with Phelan-McDermid syndrome. It is for children aged 3 to 12 who have a specific genetic change (SHANK3) and weigh at least 10 kg.
Birmingham, AlabamaAges 3–12 - NCT07287202RecruitingPhase 1/Phase 2
A study of SVG103 for people with certain brain conditions and seizures
This trial tests a drug called SVG103 (Paxalisib) in adults with brain conditions that cause hard-to-control seizures: Focal Cortical Dysplasia Type II, Tuberous Sclerosis Complex, or Hemimegalencephaly. The goal is to see if SVG103 is safe and helps reduce seizures.
Heidelberg, VictoriaAges 18–65 - NCT07280156RecruitingPhase 2
Study of PRX-115 for gout with or without methotrexate
This trial tests a new medicine, PRX-115, given through a vein, to see if it lowers uric acid levels in people with gout who haven't had success with standard treatments. Some participants will also take methotrexate, and the results are compared to a placebo (a dummy infusion).
Glendale, ArizonaAges 18 years+ - NCT06884046Recruiting
Supporting parents of babies with heart conditions
This study offers support to parents whose baby is diagnosed with a heart condition before birth and will need surgery shortly after birth. The goal is to help parents manage stress during this difficult time.
Pessac, FranceAges birth–4 weeks - NCT06853743RecruitingPhase 2
Study of nicotinamide riboside for Huntington's disease
This trial tests if a vitamin B3-like supplement, nicotinamide riboside, can help people with early to mid-stage Huntington's disease. You will be randomly assigned to take the supplement or a placebo to see if it slows the disease.
OsloAges 18–80 - NCT07284641RecruitingPhase 2
Stem cell transplant for immune disorders
This trial tests a stem cell transplant to treat common variable immunodeficiency (CVID) and other immune problems. It aims to replace a faulty immune system with a healthy one from a donor.
Pittsburgh, PennsylvaniaAges 5–40 - NCT07280585RecruitingPhase 3
Testing a diabetes drug to slow kidney disease progression
This study tests whether a common diabetes medication (SGLT2 inhibitor) can help people with autosomal dominant polycystic kidney disease (ADPKD) maintain kidney function longer. If you have fast-growing kidney cysts and are between 18 and 60 years old, this trial may be an option.
FeldkirchAges 18–60 - NCT07280104Recruiting
Infants with congenital hypothyroidism and development
This study looks at how babies with primary congenital hypothyroidism (low thyroid hormone from birth) grow and develop after being treated. It helps doctors understand if treatment helps them reach normal milestones.
NiğdeAges 6 months–1.5 years - NCT07285720Recruiting
Language Development in Williams Syndrome
This study looks at how children and adults with Williams syndrome learn language. Researchers want to understand how sounds and word patterns affect language skills in this condition.
AmiensAges 6 years+ - NCT07286565Recruiting
Remote motor development study for early-diagnosed DMD or SMA
This study remotely tracks motor development in children with Duchenne muscular dystrophy or spinal muscular atrophy who were diagnosed at birth or before symptoms started. It aims to see if early monitoring helps improve long-term care and outcomes.
LiègeAges 4 months+ - NCT07287241Recruiting
Heart health study for people with osteogenesis imperfecta
This study looks at heart structure and function in children and adults with osteogenesis imperfecta (OI), a condition that affects bones. Researchers want to understand how OI may impact the heart.
Bologna, Emilia-RomagnaAges 5–80 - NCT07282821RecruitingPhase 2
Bempedoic acid for polycystic kidney disease
This trial tests if the cholesterol-lowering drug bempedoic acid can slow kidney function loss in people with autosomal dominant polycystic kidney disease (ADPKD). It may offer a new option to protect your kidneys.
Baltimore, MarylandAges 18–60 - NCT07280468RecruitingPhase 4
Personalized sleep apnea treatment for Down syndrome
This study tests if a treatment chosen based on your specific type of sleep apnea works better for people with Down syndrome. It looks for participants who have sleep apnea but are not already using a CPAP machine regularly.
Tucson, ArizonaAges 6 years+ - NCT07279883Recruiting
Nerve block for pain after cleft palate surgery in children
This study looks back at the medical records of children who had cleft palate surgery in 2017 or 2018. It compares how long they stayed in the hospital and their pain needs after surgery, between those who got standard pain medicine alone and those who also got a nerve block to help with pain.
StockholmAges 3 months–15 years - NCT07280091Recruiting
Skin and gut bacteria in Netherton syndrome
This study looks at the bacteria on the skin and in the gut of people with Netherton syndrome, a condition that affects the skin barrier and often causes allergies. It also compares these findings to healthy volunteers to understand how the microbiome might be different.
Paris, Île-de-France RegionAges 10 years+ - NCT07286773Recruiting
Biofeedback exercises for flexible flat feet
This study tests if special exercises with biofeedback (a tool that helps you see muscle activity) can improve flexible flat feet. It may help you if you have flat feet that are not fixed in position.
Istanbul, BüyükçekmeceAges 18–45 - NCT05643079Recruiting
Bone screw and tendon transfer for heel bone realignment
This trial studies a specific surgical technique for people who need heel bone realignment along with a tendon transfer. It tests whether using a human donor bone screw helps the bone heal and the tendon work after surgery.
ViennaAges 18 years+ - NCT05825183Recruiting
Ultrasound-guided vacuum procedure study after early pregnancy loss
This trial studies a medicine-product made from conception tissue (from an ultrasound-guided manual vacuum aspiration procedure) for people with an early miscarriage or termination. It may help researchers understand whether this approach is safe and potentially beneficial for recovery, but eligibility depends on specific medical and procedural factors.
Hong KongAges 18 years+ - NCT06885307Recruiting
Building a research network for children with heart conditions
This study is building a network of hospitals across China to help children with congenital heart disease. If your child was born with a heart problem and is under 18, they may be able to join and help improve care for kids like them.
Wuhan, HubeiAges Up to 18 years
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,762 recruiting medical genetics studies from the U.S. registry right now, out of 5,482 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.