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
- NCT05705999Recruiting
TMS treatment plus CBIT study for Tourette’s tics
This trial tests whether a treatment called TMS, added to an evidence-based therapy called CBIT, can reduce Tourette’s tics more than CBIT alone. You may be assigned to different study groups, and they will also use brain imaging to measure changes.
Morgantown, West VirginiaAges 18–100 - NCT04908319Recruiting
Studying liver tissue in urea cycle disorders
This study looks at liver tissue (under a microscope) in people with urea cycle disorders to better understand liver changes from the condition. It may help clinicians learn what to watch for and how to manage liver health, especially around liver biopsy or transplant.
Washington D.C., District of ColumbiaAges Any age - NCT06175104Recruiting
HeartGPS: Support for parents after a prenatal heart diagnosis
This study tests a counseling program for parents whose unborn baby has a serious heart condition called single ventricle. The program aims to help parents manage stress and support their baby's development before and after birth.
Los Angeles, CaliforniaAges 18 years+ - NCT05871216Recruiting
Study of joint instability in people with loose joints
This trial is testing how to assess and manage joint instability in people who have unusually loose (hypermobile) joints. If you have moderate to severe loose joints, you may be able to join after screening checks like movement tests and health history.
BolognaAges 18–60 - NCT06789445RecruitingPhase 1/Phase 2
Cell therapy for inherited retinal disease (CLARICO)
This study tests a new cell therapy (OpCT-001) for adults with inherited retinal diseases that affect the light-sensing cells. It aims to see if the treatment is safe and possibly helps improve vision.
Los Angeles, CaliforniaAges 18 years+ - NCT06790394RecruitingPhase 1
Test-Retest PET Scan Study for Heart Amyloidosis
This study uses a special PET scan tracer to see how consistent the images are in people with heart amyloidosis (a buildup of abnormal protein in the heart) and in people without the condition. The goal is to improve diagnosis and monitoring.
LondonAges 40 years+ - NCT06788990RecruitingPhase 2/Phase 3
Study of ficerafusp alfa with pembrolizumab for advanced head and neck cancer
This study tests a new drug called ficerafusp alfa (BCA101) combined with the immunotherapy pembrolizumab for people with advanced head and neck cancer that has not been treated before. The goal is to see if adding ficerafusp alfa helps shrink tumors better than pembrolizumab alone.
Birmingham, AlabamaAges 18 years+ - NCT06789913RecruitingPhase 2
Testing RLY-2608 for growth disorders linked to PIK3CA mutations
This trial tests a new medicine, RLY-2608, that targets the specific genetic change (PIK3CA mutation) causing overgrowth conditions. It aims to see if the drug can slow or stop the growth of affected tissues.
Phoenix, ArizonaAges 2 years+ - NCT04686253Recruiting
Study how a heart opening and its flow link to stroke
This study looks at the detailed flow pattern through a patent foramen ovale (PFO)—a small opening in the heart—and how it may relate to stroke, TIA, or migraine. Doctors use special ultrasound testing with contrast to better understand who is most affected.
Beijing, Beijing MunicipalityAges 16–65 - NCT03358693Recruiting
Study of inflammation patterns in chronic skin diseases
This study looks at molecular (body-level) signatures in people with chronic inflammatory skin conditions. You may help researchers understand what drives inflammation, while receiving standard (already approved) systemic treatment through routine care.
KielAges Any age - NCT03361215Recruiting
Tracking skin disease patterns and anti-inflammatory response
This study looks at how atopic dermatitis (eczema), psoriasis, or other autoimmune skin diseases change over time and how the body responds to anti-inflammatory signals. Your information and—if eligible—samples help researchers find patterns that could guide better treatments.
KielAges birth–100 years - NCT06049082RecruitingPhase 1
A study of KB408 for alpha-1 antitrypsin deficiency
This study tests a new gene therapy called KB408 for people with alpha-1 antitrypsin deficiency (AATD). It aims to see if KB408 can help your body produce enough of the missing protein to protect your lungs and liver.
Gainesville, FloridaAges 18–70 - NCT05929976Recruiting
Study of gut and blood chemistry in children with new leukemia
This study looks at the gut microbiome (tiny organisms in the gut) and metabolome (small chemicals in the body) in children and teens with newly diagnosed ALL. It may help researchers understand what is happening in the body early in treatment and could guide future, more personalized care.
New York, New YorkAges 3–18 - NCT06783725Recruiting
Sleep help for people with Down syndrome
This study tests a sleep intervention to improve quality of life for people with Down syndrome who have trouble sleeping. A caregiver will help track sleep and take part in activities to improve sleep habits.
Birmingham, AlabamaAges 1–30 - NCT06782230Recruiting
A biobank for rare disease samples and data
This study creates a collection of biological samples and health information from people with rare diseases and their family members. It aims to build a shared resource that can help future research on rare conditions.
San Donato Milanese, MilanAges Any age - NCT06786754Enrolling by invitation
Skin cell study for Marfan syndrome and aortic aneurysms
This study looks at skin cells (fibroblasts) from people with Marfan syndrome or other genetic conditions that cause thoracic aortic aneurysms. The goal is to understand how these cells behave, which may help improve future treatments.
San Donato Milanese, MilanAges 18 years+ - NCT05219617RecruitingPhase 3
Carisbamate for frequent drop seizures in Lennox-Gastaut
This Phase 3 study tests whether carisbamate can reduce drop seizures in people with Lennox-Gastaut syndrome (LGS). It also checks whether the treatment is safe when added to existing seizure medicines.
Palo Alto, CaliforniaAges 4–55 - NCT06347276Recruiting
PFO closure and brain microemboli study
This trial looks at whether closing a small hole in the heart (PFO) can reduce tiny clots traveling to the brain. It includes people who have had a stroke or migraine, and also those without symptoms but with high-risk features.
Beijing, Beijing MunicipalityAges 18–65 - NCT06775704Recruiting
Studying outcomes of the ROMAX hip resurfacing system
This study tracks how well the ROMAX hip resurfacing system works over time, focusing on patient recovery and X-ray results. It is designed for adults who need hip resurfacing and are generally healthy enough for surgery.
LilleAges 18–65 - NCT06775860RecruitingPhase 3
Testing ICP-332 for moderate to severe eczema
This trial tests an oral medication called ICP-332 for adults with moderate to severe eczema (atopic dermatitis) who haven't gotten enough relief from topical creams. It aims to see if the drug can reduce skin inflammation and itching.
Hefei, AnhuiAges 18–75 - NCT06776822Enrolling by invitation
DNS therapy for asymmetry after heart surgery in children
This trial tests whether a special physical therapy called DNS (Dynamic Neuromuscular Stabilization) can help children aged 6 to 18 who have body asymmetry (uneven movement or posture) after surgery for a congenital heart defect. Participation may improve their balance and muscle control.
PragueAges 6–18 - NCT06772402Enrolling by invitation
Gene therapy for children with type 2 SMA
This trial tests a new gene therapy called GCB-001 for children with type 2 spinal muscular atrophy who can sit but not walk. It aims to improve muscle function and overall health.
Hangzhou, ZhejiangAges 2–12 - NCT06779916Recruiting
Placental cell health in pregnancy complications
This study looks at how cells in the placenta handle stress in pregnancies with complications like preeclampsia or poor fetal growth. It aims to understand why some placentas don't work well, which could lead to better treatments.
NîmesAges 18 years+ - NCT06776848RecruitingPhase 1/Phase 2
Lithium for TBR1-related neurodevelopmental disorder
This trial tests whether taking lithium for 2 years can improve symptoms in people with a TBR1 gene change that causes neurodevelopmental challenges. It aims to see if lithium is safe and helpful for this condition.
DijonAges 6 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.