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 applicable2,772
- 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 studies3,858 total
- NCT06262282Enrolling by invitation
Phage therapy for stubborn NTM infection in cystic fibrosis
This trial tests whether a special virus therapy (phage) can help clear stubborn NTM lung infections in people with cystic fibrosis who have been on antibiotics for over a year without success. Participants will continue their antibiotics while receiving the phages.
Birmingham, AlabamaAges 6 years+ - NCT06261333Enrolling by invitation
Quality of life study for HHT patients
This study looks at how hereditary hemorrhagic telangiectasia (HHT) affects your day-to-day life and well-being. By joining, you'll answer questions to help doctors better understand the impact of the condition.
Essen, North Rhine-WestphaliaAges 18 years+ - NCT06253507Enrolling by invitationPhase 1/Phase 2
Gene therapy for p47 chronic granulomatous disease
This trial tests a new gene therapy for people with a specific type of chronic granulomatous disease (p47 CGD). The treatment uses your own blood stem cells, which are modified in a lab to help your body fight infections better. It is for people who do not have a matched stem cell donor and have had serious infections.
Bethesda, MarylandAges 3–65 - NCT06222632Enrolling by invitation
MIND diet and forest bathing for heart and mind
This study tests whether a brain-healthy diet (MIND diet) combined with relaxing walks in nature (forest bathing) can help protect your heart and improve your mood. It is for Chinese adults aged 50–75 who have high cholesterol and some anxiety.
Hong KongAges 50–75 - NCT06212050Enrolling by invitation
A new valve for severe aortic stenosis in bicuspid valves
This trial tests a special heart valve (ACURATE neo2) in people with severe aortic stenosis who also have a bicuspid aortic valve, a common birth difference. The goal is to see if the valve is safe and works well for these patients.
Bad NauheimAges 18 years+ - NCT06158490Enrolling by invitationPhase 2
JYP0061 tablet for moderate to severe eczema
This trial tests a new tablet called JYP0061 for people with moderate to severe atopic dermatitis (eczema) that has lasted at least a year. It aims to see if the drug is safe and effective in reducing symptoms.
Shanghai, Shanghai MunicipalityAges 18–65 - NCT06148311Enrolling by invitationPhase 2
Dexmedetomidine film for autonomic crises in Familial Dysautonomia
This study tests a new dissolvable film containing dexmedetomidine (a medicine that calms overactive stress responses) for people with Familial Dysautonomia who experience sudden episodes called autonomic crises. It aims to treat these crises at home without needing a hospital visit.
New York, New YorkAges 16–80 - NCT06144957Enrolling by invitation
SLC13A5 deficiency natural history study (US only)
This study follows people with SLC13A5 deficiency (a rare genetic condition that affects brain development) to learn more about the disease over time. It collects health information, assessments, and samples to help understand the condition and plan future treatments.
Palo Alto, CaliforniaAges Any age - NCT06132048Recruiting
Testing a smartphone app for cystic fibrosis
This study tests a smartphone app called MuCopilot that helps you track your cystic fibrosis symptoms at home. Researchers want to see if the app is easy to use and can give accurate information about your condition.
RouenAges 18 years+ - NCT06112327Enrolling by invitation
Long-term follow-up for a gene editing heart therapy
This study follows people who already received an experimental gene editing treatment for cardiovascular disease in a prior Verve trial. The goal is to monitor their health and any long-term effects over time.
AucklandAges 18 years+ - NCT06103487Enrolling by invitation
Long-term follow-up for RGX-111 treatment
This study follows participants who previously received the RGX-111 treatment to monitor their health over time. It helps researchers understand the long-term effects and safety of the therapy.
Orange, CaliforniaAges Any age - NCT06101940Enrolling by invitation
Chinese study tracking myotonic dystrophy type 1 outcomes
This study follows people with myotonic dystrophy type 1 (DM1) over time to learn more about how the disease progresses. It collects health information and samples to help improve care.
Beijing, Beijing MunicipalityAges Any age - NCT06075537Enrolling by invitationPhase 2/Phase 3
Long-term safety study of a MPS II treatment
This study is for people with MPS II (Hunter syndrome) who have already completed a previous Denali study. It will test a drug called tividenofusp alfa (DNL310) over a longer period to see how safe it is and how well it works.
Oakland, CaliforniaAges Up to 18 years - NCT06072079Enrolling by invitation
Structural chromosome changes and brain disorders
This trial is for people who have or might have a rare disease or a change in their chromosomes. The study aims to learn more about how these changes relate to brain disorders.
StockholmAges Any age - NCT06066580Enrolling by invitationPhase 2
Long-term sevasemten study for Becker muscular dystrophy
This trial offers continued treatment with sevasemten (EDG-5506) for people with Becker muscular dystrophy who already completed a previous study of this drug. It tests the long-term safety and effects of taking sevasemten over more time.
Little Rock, ArkansasAges Any age - NCT06041906Enrolling by invitation
International registry for congenital portosystemic shunt
This registry is gathering information from people who have a rare liver blood vessel problem they were born with (congenital portosystemic shunt). By joining, you help researchers learn more about this condition and improve future care.
New Haven, ConnecticutAges 1 day+ - NCT06034392Enrolling by invitation
CHAMP app study for kids with complex heart conditions
This study tests whether using a mobile app to monitor children with complex health conditions at home can help their care team. Families will use the app to share health information instead of only in clinic visits.
Washington D.C., District of ColumbiaAges Up to 18 years - NCT05967351Enrolling by invitationPhase 3
Long-term follow-up for Duchenne patients treated before
This study checks how people with Duchenne muscular dystrophy are doing long-term after receiving delandistrogene moxeparvovec (SRP-9001) in an earlier trial. It helps researchers understand the long-term safety and effects of that treatment.
Little Rock, ArkansasAges Any age - NCT05962398Enrolling by invitation
Follow-up study for men previously treated with CSL222
This study checks long-term safety and outcomes in men with hemophilia B who previously received CSL222. It may help your care team better understand how well the treatment lasts over many years.
Phoenix, ArizonaAges 18 years+ - NCT05962346Recruiting
Pregnancy balloon procedure for severe left diaphragmatic hernia
This trial studies whether placing a small balloon in the fetus’s airway during pregnancy (a procedure called FETO) can improve lung development and outcomes for babies with severe left congenital diaphragmatic hernia. You may be considered if the condition is isolated, very severe, and you can meet specific timing and follow-up requirements.
Rochester, MinnesotaAges 18–50 - NCT05925075Enrolling by invitation
Lung ultrasound check for breathing problems in very premature babies
This trial tests whether doing a bedside lung ultrasound can help doctors assess and manage breathing problems in very premature infants. It may help guide care decisions for infants who are struggling to breathe, especially those needing breathing support.
Phoenix, ArizonaAges birth+ - NCT05921162Enrolling by invitation
Long-term follow-up after a vision gene eye injection
This study checks how people who previously received a gene therapy eye injection (vMCO-I) are doing over the long term. It may help researchers understand long-lasting effects and safety.
Cuttack, OdishaAges 18 years+ - NCT05911139Enrolling by invitation
Study how anesthesia affects brain injury markers in craniosynostosis surgery
This study looks at how general anesthesia may affect “brain injury marker” levels during and after surgery for single-suture craniosynostosis in infants. It may help doctors better understand anesthesia timing and safety for babies having corrective surgery.
BratislavaAges Up to 18 years - NCT05876169Enrolling by invitation
Study how jaw surgery changes neck and jaw movement
This study looks at how orthognathic (jaw alignment) surgery affects how your jaw and neck muscles move. It may help doctors better understand recovery and movement changes after surgery.
UmeåAges 16–50
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 5,795 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.