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
- NCT07189208RecruitingPhase 2
Preventing syphilis during pregnancy
This study tests whether taking an antibiotic (penicillin) during pregnancy can prevent syphilis infection in women who are at higher risk. If you are pregnant and have certain risk factors, this trial may help keep you and your baby safe from syphilis.
Duque de Caxias, Rio de JaneiroAges 18–45 - NCT07186179Recruiting
Stem cell mobilization for diamond blackfan anemia
This trial tests whether a medication can help move your own blood stem cells from your bone marrow into your bloodstream, so they can be collected. This is a first step toward possibly using those cells in a future treatment for diamond blackfan anemia.
New Hyde Park, New YorkAges 3–30 - NCT07188415Recruiting
CMT Gait, Mobility, and Balance Study
This study looks at how CMT affects walking, balance, and movement. It compares two groups of people with CMT: those who use an ankle-foot orthosis (AFO) and those who have had surgery to correct a foot deformity. The goal is to understand which approach works better for improving mobility.
North Liberty, IowaAges 25–55 - NCT07192679Recruiting
MAGNIFY Study: New MRI for cystic fibrosis lung health
This study tests a new type of MRI scan that uses either special gas (xenon) or regular MRI to take detailed pictures of the lungs in people with cystic fibrosis. The goal is to see how well these scans can track lung health over time, without using X-ray radiation.
SheffieldAges 1 year+ - NCT07188766Recruiting
Different IV fluid amounts for sickle cell pain crisis
This study compares two ways of giving IV fluids (lighter vs. more fluids) to adults with sickle cell disease who are in the hospital for a painful crisis. The goal is to see which approach helps you feel better sooner.
Dammam, Eastern ProvinceAges 18–60 - NCT07186764Recruiting
Quality of life in women with MRKH syndrome
This study tracks quality of life and follow-up care in women with Mayer-Rokitansky-Küster-Hauser (MRKH) syndrome, a condition where the uterus and upper vagina are underdeveloped or absent. It compares their experiences to women without MRKH to improve care.
ToulouseAges 18–50 - NCT07188285Enrolling by invitation
Immune system study for active autoimmune diseases
This study uses a blood test called mass spectrometry to look at the immune system in people with active autoimmune diseases. It aims to find patterns that could help doctors better understand and treat these conditions.
Beijing, Beijing MunicipalityAges 18 years+ - NCT07184476Recruiting
Measuring small blood vessels in kids having heart surgery
This study looks at tiny blood vessels in children with specific heart defects during surgery. It may help doctors better understand circulation and improve care.
Boston, MassachusettsAges 1 day–17 years - NCT07187206RecruitingPhase 3
Fetoscopic surgery for severe diaphragmatic hernia
This trial tests if a special surgery (FETO) done while the baby is still in the womb can improve survival for babies with a severe form of congenital diaphragmatic hernia (CDH). The surgery places a small balloon in the baby's windpipe to help the lungs grow. We are looking for pregnant women whose baby has a left-sided CDH with a lung-to-head ratio (LHR) less than 30%, or a right-sided CDH with LHR less than 45%, and whose liver is herniated into the chest.
Cincinnati, OhioAges 18–50 - NCT07193134RecruitingPhase 1/Phase 2
Gene-modified skin graft for RDEB
This trial tests a new skin graft made with your own cells that have been genetically modified to produce a missing protein (collagen 7). It's for people with RDEB, a severe blistering condition, and aims to see if the graft can heal wounds safely.
Toronto, OntarioAges 7 years+ - 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+ - NCT06946563Recruiting
Fetoscopic surgery for spina bifida before birth
This trial tests a special fetal surgery (fetoscopic repair) to fix spina bifida before birth. It might help reduce spine and brain damage in babies with this condition.
Portland, OregonAges 18 years+ - NCT07183462RecruitingPhase 4
Antibiotic treatment for late preterm water breaking
This trial tests whether a combination of two antibiotics (azithromycin and ampicillin) can help pregnant people whose water has broken between 34 and 36 weeks of pregnancy. The goal is to see if this treatment improves outcomes for both the parent and baby without causing early labor or infection.
Ramat Gan, IsraelAges 18–50 - 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 - NCT07175051RecruitingPhase 2
Testing a kidney-protecting drug in sickle cell disease
This study tests whether empagliflozin, a medicine that protects the kidneys in people with diabetes, can also protect the kidneys in people with sickle cell disease. The goal is to see if it can reduce protein in the urine, which is a sign of kidney stress.
Chicago, IllinoisAges 18–60 - NCT07183423Recruiting
New skin barrier product vs. Vaseline for dry, irritated skin
This study tests a new skin barrier product against a standard moisturizer (petrolatum) to see which works better for mild to moderate skin problems like eczema, psoriasis, or dry skin. It may help improve your skin's barrier and reduce symptoms.
Makati City, National Capital RegionAges 18 years+ - NCT07183579Recruiting
Finding the right dose of burosumab for XLH
This study looks at how young people with X-linked hypophosphataemia (XLH) respond to different doses of burosumab. The goal is to find the best dose to manage the condition.
NottinghamAges 2–18 - NCT07182474Recruiting
Turkish version of a baby neurological exam
This study is testing a Turkish version of a brief exam for babies with a high risk of cerebral palsy (CP). The goal is to see if the exam can help doctors spot brain development problems early, so your baby can get the right support sooner.
Kahramanmaraş, OnikişubatAges 3 months–1 year - NCT07178873Recruiting
Online support program for adults with spina bifida
This study tests an online support program for adults with spina bifida to help with emotional and social well-being. It is done from home through video calls.
Bilbao, BizkaiaAges 18–75 - NCT07177300RecruitingPhase 4
Hydroxyurea for sickle cell disease in children
This trial tests different ways to start hydroxyurea for children with sickle cell anemia. The goal is to find the safest and most effective approach to help manage symptoms and prevent complications.
Cincinnati, OhioAges 6 months+ - NCT07183644Recruiting
Laser treatment for port-wine birthmarks in children
This study tests how well laser therapy works for children with port-wine stains (red or purple birthmarks). It aims to find the best way to use lasers to lighten or remove these marks safely.
Dallas, TexasAges 2 months–18 years - NCT07180355RecruitingPhase 1
Gene therapy trial for Friedreich's ataxia
This trial tests a gene therapy called SGT-212 for people with Friedreich's ataxia. It aims to help treat the condition by delivering a working copy of the gene.
Los Angeles, CaliforniaAges 18–40 - NCT07180511Enrolling by invitationPhase 3
A study of QY201 tablets for moderate to severe eczema
This trial tests a new pill called QY201 for people with moderate to severe atopic dermatitis (eczema) that hasn't responded well to creams. It aims to see if QY201 can help control itching and skin inflammation.
Hangzhou, ZhejiangAges 18–75 - NCT07181837RecruitingPhase 1/Phase 2
Testing MVX-220 for Angelman Syndrome
This study tests an experimental medicine called MVX-220 to see if it is safe and effective for people with Angelman Syndrome. It includes both children (ages 4-8) and adults (ages 18-50) who can walk independently and have stable seizure medications.
Los Angeles, CaliforniaAges 4–50
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.