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
- NCT07050160Enrolling by invitation
Long-term follow-up after gene therapy for arrhythmogenic cardiomyopathy
This study follows participants who have already received the gene therapy LX2020 for a genetic heart condition called arrhythmogenic cardiomyopathy. It tracks their long-term safety and health outcomes, with no new treatment given.
Ann Arbor, MichiganAges 18–67 - NCT07054515RecruitingPhase 3
Study of oral nizubaglustat for rare genetic disorders
This trial tests a new oral medicine, nizubaglustat (AZ-3102), for children and adults (4+) with late-infantile or juvenile Niemann-Pick type C, GM1, or GM2 gangliosidosis. It aims to see if the drug is safe and helps slow disease progression.
Oakland, CaliforniaAges 4 years+ - NCT07047144RecruitingPhase 2
Apitegromab for infants with SMA under 2 years old
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
Phoenix, ArizonaAges Up to 2 years - NCT07052266Recruiting
Combined genetic screening for pregnancy and cancer risk
This trial offers a combined genetic test for both common inherited conditions and hereditary cancer risks to pregnant women or those planning pregnancy. It aims to see if adding cancer screening to routine pregnancy testing is helpful.
Brooklyn, New YorkAges 18–55 - NCT07049900Recruiting
Abdominal binder study for premature babies on breathing support
This study tests whether a soft abdominal binder can help premature babies who need breathing support breathe more comfortably and may reduce the time they need the support. It is for babies born very early who are expected to need CPAP for at least two weeks.
Minneapolis, MinnesotaAges 5 months–7 months - NCT07048106Recruiting
Study on eye changes in people with PXE
This study uses special eye imaging to track changes caused by PXE, a rare genetic condition. It may help researchers understand how PXE affects the eyes over time.
Bonn, North Rhine-WestphaliaAges Any age - NCT07053813Enrolling by invitation
Preventive genetic medicine trial for cancer risk
This trial tests whether a special program can help people with cancer-related genetic changes get the right screening and prevention care. It aims to improve how doctors manage your health based on your genes.
Denver, ColoradoAges 18–75 - NCT07048418Recruiting
Nose job results in adults born with a cleft lip and palate
This trial asks adults who had a nose job as teens or children, after being born with a cleft lip and palate, to rate how they look and feel about their nose. Their answers will help doctors understand how these surgeries affect patients long-term.
MontpellierAges 18–30 - NCT07049042Recruiting
Hormone study in genetic brain development conditions
This study looks at whether certain genetic brain development conditions affect hormone production in the body. It may help find out if people with these conditions have low sex hormones.
Sheffield, SelectAges birth–99 years - NCT07050810Enrolling by invitationEarly Phase 1
Bath system for chronic skin conditions
This study tests a special bath system (Thera-Clean Microbubbles) to see if it helps people with certain skin conditions like psoriasis, eczema, epidermolysis bullosa, or ichthyosis. It's designed for people whose skin is stable and who can follow a home bathing routine.
Chicago, IllinoisAges 2 years+ - NCT07045545Enrolling by invitation
Comparing two layers in hypospadias repair for young boys
This trial tests two ways to add an extra layer during hypospadias repair surgery in young boys. One method uses a material made from your child's own blood (platelet-rich fibrin), and the other uses a small flap of tissue from the penis. The goal is to see which method helps the healing better.
Tashkent, CityAges Any age - NCT07053969Recruiting
Does a protein gel thicken gum tissue around implants?
This study tests whether a protein-based gel (enamel matrix derivative) can make the gum tissue thicker during a second-stage implant procedure. It may help people getting dental implants achieve better healing and gum support.
San Antonio, TexasAges 18–18 - NCT07048574Recruiting
Mood and behavior in children with cystic fibrosis
This study looks at mood and behavior in children with cystic fibrosis (CF) under 12. By following children over time, researchers hope to better understand and help with depression, anxiety, and behavior challenges that may come up.
Orange, CaliforniaAges 1.5 years–11 years - NCT07044804Recruiting
Brain stimulation to improve language in Down syndrome
This trial tests a non-invasive brain stimulation technique to help adolescents and young adults with Down syndrome improve their language skills. It aims to see if this safe method can boost word-finding and speaking ability.
RomeAges 12–21 - NCT07050381Recruiting
Risk factors for ruptured aneurysms in brain AVM
This study looks at why certain aneurysms tied to a brain AVM tear more often. If you have a brain AVM with aneurysms that bled, your data may help doctors understand how to prevent future ruptures.
Nanjing, JiangsuAges Any age - NCT07049588Recruiting
Finding early signs of Charcot-Marie-Tooth 1A
This trial looks for new markers of Charcot-Marie-Tooth 1A (CMT1A) early in the disease. Researchers hope to find signs that can help diagnose and track the disease better, which could lead to better treatments in the future.
MarseilleAges 10–30 - NCT06991400Recruiting
A study of a new surgical approach for TTTS and TAPS
This trial tests a new surgical technique for twin-to-twin transfusion syndrome (TTTS) or twin anemia-polycythemia sequence (TAPS). It places a stitch through the amniotic sac during surgery to see if it helps. The study is for pregnant people between 18 and 23 weeks with healthy cervical length and a safe access point for surgery.
Cincinnati, OhioAges 18 years+ - NCT06629376RecruitingPhase 1
Testing a new gout drug for safety and effect on uric acid
This trial tests an experimental drug, SSS11, to see if it is safe and how it affects uric acid levels in people with gout and high uric acid. You may not take other gout medicines during the study.
ShanghaiAges 18–65 - NCT06825520Recruiting
Studying urges before tics during habit reversal training
This study tests whether a therapy called 'habit reversal training' can help reduce the strong urges that happen before tics. It is for children and teens (ages 8–17) who have at least 3 tics and feel a warning urge before each tic.
Salt Lake City, UtahAges 8–17 - NCT06861998Recruiting
Sleep help before heart surgery for infants and toddlers
This trial tests if helping young children with sleep problems before heart surgery can reduce confusion or agitation after surgery. It is for children ages 0 to 3 who have sleep issues and are scheduled for heart repair surgery.
BeijingAges 1 day–3 years - NCT07042126RecruitingPhase 3
Testing 611 for Teen Eczema (moderate to severe)
This trial tests a new injectable medicine (611) for teenagers with moderate to severe eczema that hasn't been helped enough by creams. It aims to see if the drug can safely reduce itching and skin symptoms.
Beijing, Beijing MunicipalityAges 12–17 - NCT07036536Recruiting
Non-Euploid Embryo Transfer Registry
This registry is collecting information from people who plan to transfer an embryo that is not genetically normal (non-euploid) at a fertility clinic in The Prelude Network. It will help doctors learn more about outcomes with these embryos.
Houston, TexasAges Any age - NCT07038200RecruitingPhase 3
Study of Del-brax (AOC 1020) for People with FSHD
This study tests an experimental medicine called Del-brax (AOC 1020) for people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to see if it can help improve muscle strength and slow the disease. You may be able to join if you have a confirmed FSHD diagnosis and can walk at least 10 meters on your own.
Orange, CaliforniaAges 16–70 - NCT07038824RecruitingPhase 1/Phase 2
A study of ENTR-601-45 for Duchenne MD (exon 45 skipping)
This trial tests an experimental drug called ENTR-601-45 for people with Duchenne muscular dystrophy that can be treated with 'exon 45 skipping.' The goal is to see if it's safe and helps improve muscle function. It's for males between 4 and 20 who can still walk and have enough arm strength.
GhentAges 4–20
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.