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
- NCT05636878Recruiting
Mobile mental health team for babies and toddlers
This study tests a mobile mental health support team for young children from birth to age 3. It may help families by bringing perinatal (around pregnancy and early parenting) psychiatric support closer to home.
LaxouAges 1 day–3 years - NCT05637814Recruiting
Screening for critical heart defects with extra blood flow checks
This trial tests if adding blood flow measurements to standard pulse oximeter screening better detects critical congenital heart disease in fetuses and newborns. It may help identify babies who need early heart care.
Davis, CaliforniaAges birth–3 weeks - NCT05641142Recruiting
Testing blood thinners in adults with inherited bleeding vessel disease
This study looks at people with Rendu-Osler (hereditary hemorrhagic telangiectasia) who have just started blood-thinning medicines. It aims to understand how antiplatelet and/or anticoagulant therapy affects safety and outcomes soon after starting.
AngersAges 18 years+ - NCT05641610RecruitingPhase 1/Phase 2
Study drug for adults with hemophilia B who still bleed
This Phase 1/2 study tests ZS801, a gene-related treatment, to see how safe it is and whether it helps prevent bleeding in adults with hemophilia B. You may be considered if you’ve had frequent bleeds or joint problems and meet several lab and health requirements.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT05643079Recruiting
Bone screw and tendon transfer for heel bone realignment
This trial studies a specific surgical technique for people who need heel bone realignment along with a tendon transfer. It tests whether using a human donor bone screw helps the bone heal and the tendon work after surgery.
ViennaAges 18 years+ - NCT05643209Recruiting
Mapping and ablation to treat abnormal heart electrical signals
This study uses detailed 3D heart mapping to find where abnormal electrical activity starts in Brugada syndrome and then performs ablation (a targeted scar or block). It’s mainly for adults who have had concerning symptoms and already have an implanted defibrillator (ICD).
Cotignola, RavennaAges 18 years+ - NCT05643872RecruitingPhase 3
Test gel QTORIN for adults with pachyonychia congenita
This Phase 3 study tests the safety and how the body absorbs (pharmacokinetics) of a rapamycin gel called QTORIN 3.9% in adults with pachyonychia congenita. It may help determine whether this gel is safe and works for people with this rare skin nail condition.
New Brighton, MinnesotaAges 16 years+ - NCT05644522Recruiting
At-home brace study for leg support and walking
This study tests an at-home wearable activity monitor and brace-related walking support for people who already use a knee-ankle-foot brace. It may help researchers understand how brace use and day-to-day movement relate to walking ability.
Chicago, IllinoisAges 18–89 - NCT05647213RecruitingPhase 1
Skin biopsy study of stem-cell therapy for congenital heart disease
This early study takes a small skin sample to make a personalized type of heart stem cells in the future. It is testing whether this approach is safe and feasible for people with complex single-chamber congenital heart disease and advanced heart failure.
Rochester, MinnesotaAges 18–40 - NCT05649098RecruitingEarly Phase 1
Testing dupilumab for itchy inherited genetic skin disorders
This early trial tests whether dupilumab can reduce itching in people with certain inherited (genetic) inflammatory skin conditions. It may help by calming skin inflammation that causes ongoing itch and sleep problems.
Chicago, IllinoisAges 6 months+ - NCT05651113Recruiting
Your experience during screening for SCID
This study looks at what it feels like for people to go through screening for SCID (a rare immune condition). It may help improve the screening process and support for patients and families.
LondonAges 18 years+ - NCT05651347RecruitingPhase 3
Melatonin in pregnancy to protect babies with slow growth
This trial studies whether taking melatonin during pregnancy can help protect the baby’s brain and organs in cases of poor fetal growth. It focuses on pregnancies at a specific gestational age with signs that the placenta may not be working well.
Camperdown, New South WalesAges 18 years+ - NCT05654025Recruiting
Study of eyesight outcomes after congenital lens dislocation
This follow-up study looks at how vision changes over time in children and adults with congenital lens dislocation, and what complications may occur. It may help doctors better predict outcomes and prevent problems after diagnosis.
Guangzhou, GuangdongAges 3 years+ - NCT05654272Recruiting
Testing CIRC imaging technology for people with heart problems
This study is developing and testing CIRC technologies using heart imaging. You may be able to join if you’re an adult with cardiovascular (heart) disease and can safely have the MRI scans.
Cleveland, OhioAges 18 years+ - NCT05656365Recruiting
Study of genes and immunity in PFAPA and other tonsil problems
This study looks at genetic markers and immune system patterns in people with PFAPA (periodic fevers with mouth sores and throat/neck gland symptoms) or similar tonsil problems. It may help researchers understand why these conditions happen and how they might be better diagnosed or treated.
Washington D.C., District of ColumbiaAges 1 month–99 years - NCT05658575RecruitingPhase 2/Phase 3
Tablets to treat sudden gout flare pain
This trial studies dapansutrile tablets (OLT1177) to see how well they work for people in the first 4 days of an acute gout flare. It may help reduce pain and inflammation in the specific joint where your gout is flaring.
Birmingham, AlabamaAges 18 years+ - NCT05659901Recruiting
Study tracks symptoms and test results in PLP1-confirmed PMD
This study follows children with Pelizaeus-Merzbacher disease (PMD) who have a specific genetic change (PLP1 duplication). It aims to learn which body measurements (“biomarkers”) and symptoms change over time, to better understand disease progression.
Atlanta, GeorgiaAges 6 months–17 years - NCT05664412Recruiting
Brain stimulation trial to improve thinking in 22q11.2 deletion
This trial tests a non-invasive brain stimulation approach to help improve executive function—skills like planning, attention, and self-control—in people with 22q11.2 deletion syndrome. If you qualify, you would be asked to participate and allow study procedures to see whether this treatment helps.
Geneva, Canton of GenevaAges 14–25 - NCT05664867Recruiting
Program study for hereditary breast or colon cancer counseling in clinics
This study helps clinics roll out cancer genetic counseling services for people who may have inherited cancer risk. If you may need genetic testing for hereditary breast or colon cancer, you can be interviewed about your experience with these services.
Chicago, IllinoisAges 25 years+ - NCT05666986Recruiting
Sports and activity check-in for teens after diaphragmatic hernia surgery
This study looks at how adolescents who had diaphragmatic hernia surgery as babies engage in physical activities and sports. It will use questionnaires and an optional interview by video call to learn what helps or gets in the way—so care teams can better support families.
LilleAges 12–18 - NCT05669950RecruitingPhase 1/Phase 2
Study drug Lu AG13909 for congenital adrenal hyperplasia
This early study tests Lu AG13909 in people with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. It aims to see how the drug affects hormone levels and whether it is safe for different CAH hormone patterns.
Ann Arbor, MichiganAges 18–70 - NCT05671666Recruiting
Testing urea processing in healthy people and urea cycle disorder
This study measures how your body breaks down protein-related waste and makes urea. It includes both healthy volunteers and people with urea cycle disorders, which may help researchers better understand the condition and improve testing.
Zurich, Canton of ZurichAges Any age - NCT05672849Recruiting
Testing devices used during fetal surgery for spina bifida
This trial looks at how safe and effective certain surgical devices are when doctors perform a minimally invasive fetal procedure to repair a neural tube defect. If you choose this specific type of fetal surgery, you may be able to join the study to help improve care for future patients.
Houston, TexasAges 18 years+ - NCT05673499Recruiting
Monitoring oxygen and brain signals during major newborn surgery
This study checks how well newborn babies’ brains are getting oxygen and how brain activity changes while they have certain major surgeries. It may help doctors better monitor brain safety during surgery in newborns with specific conditions.
New York, New YorkAges Up to 1.1 years
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.