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
- NCT06364176RecruitingPhase 2
Adding losartan to improve Trikafta response in CF
This study tests whether adding the blood pressure medication losartan can help the standard CF drug Trikafta work better by reducing inflammation. It's for people with CF who still have high sweat chloride levels despite taking Trikafta.
Kansas City, KansasAges 12 years+ - NCT07156214Recruiting
Understanding RFC1-ataxia to find biomarkers
This study looks at a rare genetic condition called RFC1-ataxia (also known as CANVAS). It aims to find better ways to diagnose and track the disease. You may qualify if you have this condition, are between 18 and 80, and can agree to take part.
BolognaAges 18–80 - NCT07163403RecruitingPhase 1
Preventing cancer in Lynch syndrome carriers with dendritic cell vaccine
This trial tests whether a personalized vaccine made from your own immune cells can help prevent cancer in people with Lynch syndrome. You must have no history of cancer and be willing to have regular colonoscopies.
Barcelona, BarcelonaAges 18 years+ - NCT07159581Enrolling by invitation
Gene therapy for Wilson disease study with PET scans
This trial uses a special PET scan with a copper tracer to see how well gene therapy is working in people with Wilson disease. It may help doctors understand if the treatment is helping your body handle copper properly.
Aarhus, Aarhus NAges 18 years+ - NCT07162285Enrolling by invitation
Bubble CPAP for Preterm Babies with Breathing Problems
This trial tests a special breathing support called bubble CPAP, given right after birth in the delivery room, to help preterm babies (born before 34 weeks) who are having trouble breathing. It aims to see if starting this treatment early can improve their breathing and health.
DhakaAges 5 minutes–30 minutes - NCT07163780Recruiting
Home Therapy Program for Babies With Down Syndrome
This study tests a special therapy program called HEP (home early intervention program) for babies with Down syndrome. It looks at whether this program can help your baby's development.
Istanbul, BeykozAges 4 months–1 year - NCT07156318Recruiting
Drumming lessons for children with Down syndrome
This study tests whether drumming lessons can help children with Down syndrome. It may help with coordination, focus, or mood.
Nashville, TennesseeAges 7–15 - NCT07161544RecruitingPhase 1/Phase 2
Gene therapy for Stargardt disease
This trial tests a new gene therapy, AAVB-039, for people with Stargardt disease caused by a specific gene mutation. The treatment is given during eye surgery and aims to slow or stop vision loss.
Phoenix, ArizonaAges 8–55 - NCT07163260Enrolling by invitation
Study on haemophilia care in Asia and Pacific
This study looks at the experiences of people with haemophilia, their caregivers, and doctors in Asia and the Pacific. The goal is to understand current care and find ways to improve it. You may be asked about your treatment and daily life.
Bangalore, KarnatakaAges Any age - NCT07156578Enrolling by invitationPhase 2
Long-term bivamelagon for hypothalamic obesity
This long-term study tests a new drug, bivamelagon, for people with hypothalamic obesity. It's for those who already completed a similar study and tolerated the drug well.
Aurora, ColoradoAges 12 years+ - NCT07160634RecruitingPhase 3
Gene therapy for ambulant males with Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 in boys and men with Duchenne muscular dystrophy who are still able to walk. The treatment aims to deliver a working version of the dystrophin gene to help muscle cells produce the protein they are missing.
Little Rock, ArkansasAges 7–11 - NCT06591793RecruitingPhase 1/Phase 2
Gene therapy for Usher syndrome type 1B vision loss
This trial tests an experimental gene therapy given as an injection under the retina. It aims to slow or stop vision loss in people with Usher syndrome type 1B, a rare genetic condition that causes both hearing and vision problems.
NaplesAges 18–50 - NCT06692296Recruiting
Balance training with a stabilometric platform for Friedreich's ataxia
This trial tests if a special platform that measures balance can help people with Friedreich's ataxia stand more steadily. If you have Friedreich's ataxia, can walk at least 10 metres and stand without support, you may be eligible.
Pieve di Soligo, VenetoAges 14 years+ - NCT06955052Recruiting
Maze activity for balance and walking in Down syndrome
This study tests a fun activity program called 'Minds In Motion The Maze' to see if it helps improve balance and walking in children with Down syndrome.
Lahore, Punjab ProvinceAges 8–12 - NCT02257229Recruiting
Study for treating clubfoot in Spanish-speaking patients
This study looks at treatment for children with clubfoot (a foot that turns inward or upward). If you speak Spanish, the study may be easier to join and could help doctors improve how clubfoot is treated.
Dallas, TexasAges Up to 18 years - NCT06728787Recruiting
Robot-assisted walking for hereditary spastic paraplegia
This trial tests if robot-assisted walking therapy can help people with hereditary spastic paraplegia (a condition causing progressive leg stiffness and weakness) improve their walking ability. It may be an option if you can walk indoors on your own.
Bosisio Parini, Italy/leccoAges 4–70 - NCT07146256Recruiting
OPMD Natural History Registry Study
This study is building a national registry (a database) for people with Oculo-Pharyngeal Muscular Dystrophy (OPMD) in Israel. Joining will help researchers understand how OPMD progresses over time and may lead to better care.
Ramat GanAges 18 years+ - NCT07150013RecruitingPhase 1
Vorinostat for Rett syndrome safety study
This trial tests whether vorinostat is safe and helpful for girls and young women with Rett syndrome. Participants must have a confirmed MeCP2 mutation and be in a stable phase of the condition.
MedellínAges 6–21 - NCT07153939Recruiting
Study of a new device to create dialysis access
This trial tests a new device called the Velocity pAVF System that creates a connection between a vein and artery in your arm for hemodialysis. If you need dialysis now or within the next 6 months, this could be a new option for you.
Dothan, AlabamaAges 18–80 - NCT07145411Recruiting
Bone marrow transplant trial for patients 12 to 80
This trial provides bone marrow for transplant to patients who are already considered good candidates for a donor transplant by their own doctor. The goal is to see if using this bone marrow product is safe and helpful.
Birmingham, AlabamaAges 12–80 - NCT07150026RecruitingPhase 1
Testing vorinostat for Pitt Hopkins syndrome
This trial tests whether vorinostat, a medication that may affect gene activity, is safe and helpful for children and young adults with Pitt Hopkins syndrome. Participants take the drug by mouth or feeding tube.
MedellínAges 3–21 - NCT07145229RecruitingPhase 2
Testing a new drug for acute gout flares
This trial tests a new medication, ABP-745, for people having an acute gout flare. It aims to see if the drug is safe and helps reduce pain and inflammation.
Peoria, ArizonaAges 18–70 - NCT07145138Recruiting
Multi-ethnic dilated cardiomyopathy registry
This study is a registry for people with dilated cardiomyopathy (DCM)—a condition where the heart's main pumping chamber becomes enlarged and weak. It aims to learn more about DCM, especially in people from different ethnic backgrounds, by collecting health information over time.
Chicago, IllinoisAges 18 years+ - NCT07152392RecruitingPhase 4
Testing IBI311 for inactive thyroid eye disease symptoms
This trial tests a medication called IBI311 for people with inactive thyroid eye disease that still causes bothersome symptoms like double vision or trouble closing the eyes. The goal is to see if it helps improve these problems safely.
Shanghai, Shanghai MunicipalityAges 18–80
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.