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
- NCT07237100RecruitingPhase 2
Mirdametinib for advanced NF1-mutant melanoma
This trial tests an experimental oral drug called mirdametinib for people with advanced melanoma that has a specific gene change (NF1 mutation). To join, you must have already had immunotherapy, but not a MEK/ERK/RAF targeted drug before.
San Francisco, CaliforniaAges 18 years+ - NCT07234695RecruitingPhase 3
Levetiracetam to prevent seizures in adults with Down syndrome and Alzheimer's
This trial tests if levetiracetam, a seizure medication, can prevent seizures in adults with Down syndrome who also have Alzheimer's disease. It aims to help manage a common complication and improve quality of life.
Granada, AndalusiaAges 40 years+ - NCT07233421Enrolling by invitation
Blood sugar levels in children with Long QT syndrome on beta-blockers
This study looks at how often low blood sugar (hypoglycemia) happens in young children with Long QT syndrome who take beta-blocker medicine. It may help doctors understand if these children need extra monitoring.
HelsinkiAges Up to 7 years - NCT07235345Enrolling by invitation
Lung medication with different oxygen levels for premature babies
This study tests a lung medication called pulmonary surfactant, given with different oxygen levels, to help very premature babies (born before 32 weeks) who have breathing difficulties. The goal is to see which oxygen level works best with non-invasive breathing support.
Henan, ZhengzhouAges 6 months–7 months - NCT07237321Recruiting
Magnetic stimulation for flat feet: effects on arch and strength
This study tests whether magnetic stimulation of foot muscles can improve the arch of the foot and muscle strength in adults with flat feet. If you have flat feet without major leg or health problems, this trial may help find a new way to strengthen your feet.
Ljubljana, LjubljanaAges 18–50 - NCT07238465RecruitingPhase 3
Nervous system and heart function in Down syndrome
This study looks at how the nervous system controls heart rate and blood pressure in adults with Down syndrome. It involves some simple tests, like blood draws and placing a small tube in an arm vein, plus possibly some cold water or exercise tasks.
Aurora, ColoradoAges 18–50 - NCT07233408Recruiting
Online tool to help adults with neurofibromatosis 1 and low health literacy
This study tests an online platform designed to help adults with neurofibromatosis 1 (NF1) who have low health literacy. The goal is to make it easier to get evidence-based care. You may be a good fit if you have an upcoming checkup with your primary care doctor.
Boston, MassachusettsAges 18 years+ - NCT07239856Recruiting
Goat vs. Cow Milk: Which is easier to digest?
This study compares how well people digest goat milk versus cow milk. If you have trouble with cow milk but not goat milk (or can drink both), researchers want to see if goat milk is a better choice for you.
BobignyAges 18–65 - NCT07232459Recruiting
PET imaging for amyloidosis in the heart
This study tests a new PET imaging tracer called [18F]FT8 to see if it can better detect amyloid protein buildup in the heart. It may help doctors diagnose and monitor cardiac amyloidosis more accurately.
Hefei, AnhuiAges 18 years+ - NCT07232134RecruitingPhase 3
Treating AML in children with Down syndrome
This trial tests a treatment plan for children with Down syndrome who have been diagnosed with acute myeloid leukemia (AML) or a related bone marrow condition. The goal is to see how well the therapy works and to help guide doctors in choosing the best approach.
MoscowAges 1 day–18 years - NCT05463757Recruiting
Oral drugs for advanced or multiple basal cell skin cancer
This study registers people in the Netherlands who have certain types of basal cell skin cancer and take an oral hedgehog-blocking medicine. It may help doctors understand how well these tablets work and how patients do in real life.
Nijmegen, GelderlandAges Any age - NCT04774848Recruiting
Compare two breathing machines for newborns with diaphragmatic hernia
This study compares two types of ventilator support for newborns with congenital diaphragmatic hernia. The goal is to see which breathing approach works better and is safer when started very early after birth.
Salt Lake City, UtahAges Up to 1 day - NCT05201144RecruitingPhase 2
Test of sildenafil after diaphragm hernia surgery in newborns
This trial studies whether sildenafil (a medicine that helps relax blood vessels) can improve outcomes in babies born with a congenital diaphragmatic hernia (CDH) after they’ve had surgery. Babies are selected based on an ultrasound/echo of heart function after repair.
Salt Lake City, UtahAges Any age - NCT06911190Recruiting
10-year follow-up study for FSHD patients
This study follows FSHD patients for 10 years to see how the disease progresses. It includes people who were in earlier FSHD studies and some new patients with genetic confirmation of FSHD.
NijmegenAges 6 years+ - NCT07227857RecruitingPhase 1/Phase 2
Study of a new medicine for children with KCNT1-related epilepsy
This study tests a new medicine (S230815) given by lumbar puncture (spinal tap) to see if it helps reduce seizures in children with a specific genetic cause of epilepsy. It is for children aged 2 to 12 who have not responded well to other treatments.
Orange, CaliforniaAges 2–12 - NCT07229079Recruiting
Body awareness therapy for flat feet
This study tests whether basic body awareness therapy can help people with flexible flat feet (when the foot arch flattens under weight but returns when not standing). If you have flat feet without other foot problems, you may qualify.
Mardin, Mardi̇nAges 18–45 - NCT07224308Recruiting
Long-term effects of MFU-V on sagging skin
This study tests a non-invasive ultrasound treatment called MFU-V for sagging skin, fine lines, and crepey skin on the lower face, under the chin, and neck. It aims to see how long the results last and how well different treatment depths work.
New York, New YorkAges 25–65 - NCT07227116Recruiting
Finding transferable embryos in complex aneuploidy cases
This study aims to check if embryos previously considered too abnormal (with three or more chromosomal issues) might actually be usable for transfer. It may offer new options for patients who have only these types of embryos available.
Margate, FloridaAges Any age - NCT07230223RecruitingPhase 1/Phase 2
A study of Ev.FV on wound healing in dystrophic epidermolysis bullosa
This trial tests if a drug called Ev.FV can help heal wounds in people with dystrophic epidermolysis bullosa (DEB), a condition that causes fragile, blistering skin. It is for people with open wounds on their arms, legs, or trunk that are a specific size.
IsfahanAges 3–35 - NCT07226219RecruitingPhase 1
Medication to help attention in children with sickle cell disease
This study tests whether a common medication called methylphenidate (often used for ADHD) can help children with sickle cell disease who have attention or memory problems. If the child qualifies, the study may offer a new way to improve focus and daily functioning.
Memphis, TennesseeAges 8–17 - NCT07223255Recruiting
Mediterranean diet study for children with cystic fibrosis
This study tests whether a Mediterranean-style diet can improve gastrointestinal health in children with cystic fibrosis. It requires families to follow the diet for 6 months and attend regular clinic visits.
Lebanon, New HampshireAges 3–18 - NCT07226297Enrolling by invitationPhase 1/Phase 2
Personalized treatment for GARS1-related CMT2D
This trial tests a personalized medicine designed for a single person with a specific genetic mutation (GARS1) that causes Charcot-Marie-Tooth disease type 2D. The treatment is an antisense oligonucleotide, a custom-made molecule that targets the faulty gene.
Houston, TexasAges 13 years+ - NCT07227155Recruiting
HOPE Biobank for bone marrow failure patients
This study collects blood samples from patients with aplastic anemia, sickle cell disease, thalassemia, or other bone marrow failure conditions who are receiving a stem cell transplant or gene therapy. The samples will be stored and used for future research to better understand and treat these diseases.
Duarte, CaliforniaAges Any age - NCT07228962Recruiting
Skin clues for eczema in babies
This study looks at tiny skin samples from babies to find clues about eczema. It uses a gentle, painless device that sucks up a small piece of skin. If your baby has eczema or is healthy and under 6 months old, you may be able to join.
LondonAges birth–6 months
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.