Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,856 recruiting studies in this area right now.
By phase
- Not applicable2,770
- Phase 2282
- Phase 1235
- Phase 3215
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 353
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis129
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome54
- 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
- California340
- Maryland161
- Alabama132
- Massachusetts116
- New York84
- Arizona79
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,856 total
- NCT07224880Recruiting
Skin tightening and wrinkle treatment study for adults 30-65
This trial tests a micro-coring device called Ellacor® to improve both facial wrinkles and abdominal skin looseness. It is open to people of all skin types and includes those taking certain diabetes/weight-loss medications (GLP-1 agonists).
Dublin, CaliforniaAges 30–65 - NCT07226219RecruitingPhase 1
Methylphenidate for Attention in Sickle Cell Disease
This trial tests if a medication called methylphenidate can help children with sickle cell disease who have trouble with attention, focus, or executive function. It aims to see if it can improve daily life and school performance.
Memphis, TennesseeAges 8–17 - NCT07226726RecruitingPhase 1
Stem cell exosome treatment for congenital myasthenic syndrome
This trial tests a treatment made from stem cell exosomes (tiny particles that may help repair cells) for people with congenital myasthenic syndrome, a muscle-weakening condition. It aims to see if this treatment is safe and could improve symptoms.
Naples, FloridaAges 18 years+ - NCT07228793Recruiting
A study tracking EYS-related retinitis pigmentosa over time
This study follows people with retinitis pigmentosa caused by changes in the EYS gene over 4 years. It collects data on how the disease progresses and does not test any new treatments.
MoscowAges 14–100 - 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 - NCT07229430Recruiting
Ultrasound treatment for loose neck and chin skin
This study tests a high-intensity focused ultrasound (HIFU) device to lift and tighten loose skin under the chin and neck. If you are in good health and want a non-surgical option for mild to moderate skin laxity, this trial may be for you.
Los Angeles, CaliforniaAges 30–65 - 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 - NCT07230483RecruitingPhase 2
Long-term safety of CM512 for atopic dermatitis
This study is for people who have already completed a previous study of CM512 for atopic dermatitis. It will check if the medicine is safe and works well over a longer period.
Beijing, Beijing MunicipalityAges 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 - 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+ - NCT07233265Recruiting
Comparing two surgical methods for undescended testicle in boys
This study compares a newer single-incision scrotal surgery with the standard two-incision inguinal surgery to fix an undescended testicle in boys under 10 years old. The goal is to see which method works better.
Faisalābad, Punjab ProvinceAges 1–10 - 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+ - NCT07235709Recruiting
Study of agalsidase alfa for heart inflammation in Fabry disease
This trial tests whether agalsidase alfa can reduce heart inflammation in people with Fabry disease who have heart problems. You may qualify if you have not had this treatment in the past year.
SeoulAges 15–75 - 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+ - 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
Exploring nervous system function in Down syndrome
This trial studies how the nervous system works in people with Down syndrome. It may help understand differences in body functions and improve future care.
Aurora, ColoradoAges 18–50 - 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 - NCT07240844RecruitingPhase 4
Enavogliflozin for amyloid heart disease
This study tests a drug called enavogliflozin to see if it helps people with a specific type of heart failure caused by amyloid buildup. It may improve symptoms and daily function.
SeoulAges 19 years+ - NCT07240896RecruitingEarly Phase 1
Testing a new mRNA therapy for Wilson disease
This trial tests an experimental mRNA treatment (DSL101) for adults with Wilson disease. It's for people whose condition is stable on current therapy and who have certain genetic markers. The goal is to see if the new treatment can help the body process copper better and reduce the need for standard medicines.
Hefei, AnhuiAges 18 years+ - NCT07241234RecruitingPhase 1
Study of an experimental drug for phenylketonuria (PKU)
This trial tests an investigational drug (AG-181) for people with PKU who have a specific genetic change (R408W mutation). It aims to check if the drug is safe and how it works in your body.
Indianapolis, IndianaAges 18–69 - NCT07242638RecruitingPhase 2
Abrocitinib for skin and hair loss in Down syndrome
This study tests if the pill abrocitinib can help people with Down syndrome who have atopic dermatitis (eczema) or alopecia areata (patchy hair loss). The goal is to see if it improves their skin or hair with manageable side effects.
New York, New YorkAges 12 years+ - NCT07244133Recruiting
Powerball exercise for kids with Down syndrome
This trial tests whether using a Powerball (a small exercise ball) can improve hand strength and coordination in children with Down syndrome. It may help with daily tasks and motor skills.
Lahore, Punjab ProvinceAges 6–10 - NCT07244172Recruiting
Mud walking for kids with flexible flat feet
This study tests whether walking on mud can improve foot posture and balance in children with flexible flat feet. It may help strengthen the feet without surgery or special equipment.
Lahore, Punjab ProvinceAges 6–11 - NCT07244614Recruiting
Study of ustekinumab or apremilast for Behçet's disease symptoms
This study tests if ustekinumab or apremilast can help control Behçet's disease symptoms like mouth and genital ulcers, skin sores, and joint pain. It may be an option if standard treatments haven't worked well.
BordeauxAges 18 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,856 recruiting medical genetics studies from the U.S. registry right now, out of 5,791 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.