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 2284
- Phase 1235
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome55
- 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
- California341
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona78
- Texas72
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT05674487Recruiting
Balloon vs prostaglandin medicine to start labor in small babies
This trial compares two ways to help start labor by the vagina in pregnancies where the baby is measuring small. It aims to see which method works better and is safer for getting birth when needed.
BordeauxAges 18 years+ - NCT05674695Recruiting
Registry study for starting or switching atopic eczema medicines
This study follows people with atopic eczema when they start or switch to a specific medicine that affects the immune system. It helps doctors understand how these treatments work in real life, including for skin symptoms and daily quality of life.
Badalona, BarcelonaAges Any age - NCT05676099Recruiting
TSC or LAM blood and health information registry
This study sets up a database and collection of “biosamples” (like blood or other body samples) from people with tuberous sclerosis complex (TSC) or sporadic lymphangioleiomyomatosis (LAM). The goal is to better understand how these conditions change over time and support future research.
Birmingham, AlabamaAges Any age - NCT05677880Recruiting
Study for CADASIL using MRI, blood tests, and follow-up visits
This trial studies CADASIL (a genetic blood-vessel condition in the brain) by following people over time with MRI scans, blood draws, and questionnaires. It may help researchers better understand how the condition changes and how factors like medications affect outcomes.
Los Angeles, CaliforniaAges 18 years+ - NCT05677971RecruitingPhase 3
Tested medicine to treat liver scarring in PiZZ alpha-1 disease
This Phase 3 study tests fazirsiran for safety and whether it can help people with a specific genetic form of alpha-1 antitrypsin–related liver disease that causes liver scarring. You may be eligible if you have the PiZZ genetic type and moderate-to-severe fibrosis, with certain lung and liver conditions ruled out.
Birmingham, AlabamaAges 18–75 - NCT05678374Recruiting
Looking for immune signs linked to mental fatigue in Graves’ disease
This study looks at blood “immune” markers to understand why some people with Graves’ disease develop mental fatigue (feeling slowed down in thinking). It may help doctors better recognize and explain mental fatigue in Graves’ disease.
GothenburgAges 18–72 - NCT05678543Recruiting
Diabetes family registry for mothers, partners, and children
This study builds a registry to learn about diabetes in families over time. It includes mothers with diabetes, their partners, and their children, to help researchers understand how diabetes affects families.
AalborgAges 18 years+ - NCT05681624Recruiting
Extra oxygen during labor to support the baby
This trial studies whether giving extra oxygen to the pregnant person during labor helps the baby during “intrauterine resuscitation,” when monitoring suggests the baby needs support. It may help babies recover better while still in the womb, especially during continuous fetal monitoring.
St Louis, MissouriAges Any age - NCT05682378RecruitingPhase 3
Long-term follow-up for inclisiran in young people with inherited high cholesterol
This study follows people with HeFH or HoFH (inherited high cholesterol) who previously took inclisiran in specific pediatric ORION studies. It mainly checks long-term safety and how well the treatment continues to be tolerated over time.
Boca Raton, FloridaAges 12–100 - NCT05682144RecruitingPhase 1
Cell therapy for MPS I to improve immune function
This Phase 1 study tests an engineered cell therapy made to help the body in people with MPS I (Hurler-Scheie or Scheie). It is early-stage research, so the main goal is to learn about safety and the best way to treat.
Oakland, CaliforniaAges 10 years+ - NCT05683418RecruitingPhase 1
Study of a new drug for PIK3CA-mutated HR+ HER2- breast cancer
This early-stage study tests the safety and tolerability of TOS-358 in women with HR-positive, HER2-negative breast cancer that has a specific gene change (PIK3CA). It may help learn whether this targeted treatment is safe enough to move forward.
Chicago, IllinoisAges 18 years+ - NCT05686473Recruiting
Parent teaching program for children with prenatal alcohol exposure
This study offers a structured teaching program for parents of children who were exposed to alcohol before birth (prenatal alcohol exposure). It aims to help families better understand the child’s needs and how to support development and daily functioning.
ArendalAges 2–18 - NCT05687149Recruiting
Study of mouth and throat cancer patterns in Fanconi anemia
This study looks at how squamous cell carcinoma (a type of cancer) naturally develops over time in people with Fanconi anemia. It may help doctors better recognize warning signs and plan care in the future.
Bethesda, MarylandAges 8–90 - NCT05687292Recruiting
Decision tool to shorten breathing machine time in children
This study tests whether a computer “decision support” tool can help doctors safely reduce how long a child stays on a breathing machine after heart surgery. It may help children get off mechanical ventilation sooner and recover faster.
Boston, MassachusettsAges 1 day–12 years - NCT05688488RecruitingPhase 1/Phase 2
Curcumin to help prevent vocal cord scar adhesions after surgery
This trial tests whether curcumin (a natural compound) can reduce scar-like “adhesions” that can form between both vocal cords after endoscopy surgery. If you’re having a procedure on both vocal cords, this could help lower the chance of postoperative sticking and related voice problems.
Shanghai, Shanghai MunicipalityAges 18–75 - NCT05698277Recruiting
Ultrasound to track baby heart function in heart disease
This study uses automated ultrasound measurements to track how a baby’s heart is working during pregnancy. It compares babies who have a specific congenital heart problem with babies without it, to understand what ultrasound can detect.
Sydney, New South WalesAges 18 years+ - NCT05698901Recruiting
Testing heart blood tests and scans to monitor Fabry disease
This study checks whether certain heart-related blood tests and heart scans can help monitor people with Fabry disease. It may be useful for both people who have never received enzyme replacement therapy and those who have.
TaipeiAges 18 years+ - NCT05701514Recruiting
Test in babies with a prenatal lung cyst found on scans
This trial is for newborns who are found before birth to have a congenital lung cyst (CPAM) and who stay symptom-free. It tests an approach for babies with certain CPAM features to see if it’s safer and/or more effective than standard care.
Nijmegen, GelderlanfAges 1 month–1 year - NCT05702203Recruiting
Music therapy for newborns with congenital heart disease
This trial tests whether creative music therapy can help newborn babies who have congenital heart disease (CHD) while they are in intensive care. It focuses on babies who are otherwise very young and in the NICU or PICU, regardless of CHD severity.
ZurichAges 1 minute–4 weeks - NCT05702476Recruiting
Non-invasive 3D scan study for Marfan face features
This study uses a non-invasive 3D scan to better measure facial features in people with Marfan syndrome. It may help researchers describe face differences more accurately, without needles or surgery.
San Donato Milanese, LombardyAges Any age - NCT05704036RecruitingPhase 4
Estrogen use and bone health in women with cystic fibrosis
This observational study looks at how taking (or not taking) estrogen affects bone health in women with cystic fibrosis (CF). If you may have low estrogen, it also explores whether estrogen levels and symptoms relate to bone density over time.
Baltimore, MarylandAges birth–100 years - NCT05704010Recruiting
Video pill camera test for Lynch syndrome
This trial studies whether a swallowable camera (video capsule endoscopy) can help look inside the small intestine for people with Lynch syndrome. It may help doctors detect problems earlier or more clearly by improving how the small bowel is checked.
Milan, LombardyAges 18 years+ - NCT05704257Recruiting
Fetal surgery study for complex gastroschisis safety
This trial tests whether a specialized fetal operation is safe and practical for babies with a serious form of gastroschisis (bowel outside the belly) and swollen bowel. You may be considered if the pregnancy is otherwise free of major related problems and the family is ready for follow-up after birth.
Houston, TexasAges 18 years+ - 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
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 5,764 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.