Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,863 recruiting studies in this area right now.
By phase
- Not applicable2,776
- Phase 2282
- Phase 1237
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis129
- Congenital Heart Disease85
- Cystic Fibrosis78
- Down Syndrome55
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease39
- Hemophilia A39
- 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 Human Genome Research Institute (NHGRI)23
Where studies are running
- California339
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona77
- Texas72
- Minnesota67
- Pennsylvania66
- Ohio57
Recruiting studies3,863 total
- NCT04849754Recruiting
Monitoring heart amyloidosis with a special scan
This study looks at how a type of heart condition called transthyretin cardiac amyloidosis changes over time, using a special imaging test called a bone scan. The goal is to see if the scan can help track the disease and potentially improve care.
LilleAges 18 years+ - NCT05136859Recruiting
Predicting fistula success with blood tests and vessel checks
This study looks at whether blood markers and the condition of your blood vessels before surgery can predict how well a new dialysis access (fistula) will work. It aims to help doctors identify patients who may need extra care to ensure their fistula matures properly.
TaipeiAges 20 years+ - NCT06494540Recruiting
TDC treatment for advanced non-squamous lung cancer
This study is looking at how well a new treatment called TDC (which includes durvalumab and chemotherapy) works for people with a certain type of advanced lung cancer that has spread and has specific genetic features. It aims to see if this treatment is effective and safe in the real world, and it might be an option if you are starting this therapy anyway.
Bad HomburgAges 18–120 - NCT07776444Recruiting
Measuring brain blood flow with light and MRI
This study uses a special light-based device and MRI to measure how blood vessels in the brain respond to changes. It may help researchers better understand blood flow in people with and without sickle cell disease.
St Louis, MissouriAges 6–70 - NCT07310849Recruiting
Digital health support for older adults with diabetes
This study tests a digital care program using the LINE messaging app to help older adults with diabetes and other chronic conditions manage their health. It may be a good fit if you are 50 or older, have type 2 diabetes plus another condition like high blood pressure, and are comfortable using a smartphone.
New Taipei City, Wugu DistrictAges 50 years+ - NCT07780188Recruiting
Lean beef in a healthy meal plan for teens with PCOS
This study tests whether adding lean beef to a healthy meal plan can help teens with PCOS improve their health. You'll follow a special diet and wear a small monitor to track your blood sugar, and the study may help you feel better and manage your condition.
College Station, TexasAges 11–17 - NCT06761560RecruitingPhase 2/Phase 3
Hydroxyurea dosing for children with sickle cell anemia
This study looks at whether adjusting the dose of hydroxyurea based on drug levels in the blood works better than the standard approach for treating sickle cell disease in children. It aims to reduce side effects and improve treatment outcomes.
Montreal, QuebecAges 6 months–18 years - NCT07778836RecruitingPhase 3
Study of daily injections for Friedreich's ataxia
This study tests an investigational drug given as a daily injection to see if it can improve walking and balance in people with Friedreich's ataxia.
Chicago, IllinoisAges 12–40 - NCT07417137Recruiting
Long-term study of Angelman syndrome
This study follows people with Angelman syndrome over time to learn more about the condition. It does not test any new treatments, but you need a parent or caregiver to come with you to all visits.
Lexington, MassachusettsAges 1 year+ - NCT07643844RecruitingPhase 1
Gene therapy for propionic acidemia in infants
This trial tests a new gene therapy treatment designed to help the body process certain amino acids better in infants with propionic acidemia. The therapy uses a modified virus to deliver a working copy of a faulty gene, potentially reducing dangerous acid buildups and hospital visits.
Rochester, MinnesotaAges 6 months–2 years - NCT07441876RecruitingPhase 2/Phase 3
Comparing a new drug to vosoritide for children with achondroplasia
This trial tests a new medicine, BMN 333, against an existing one (vosoritide) to see if it helps children with achondroplasia grow. It's for kids who haven't used these treatments before.
Oakland, CaliforniaAges 2–17 - NCT06226051Recruiting
Growing Little PEAPODS Preterm Baby Study
This study follows the growth and body composition of very premature babies (born between 22 and 32 weeks) to learn how they develop. It uses a special device called the PEAPOD to measure body fat, which may help improve care for preemies.
Madison, WisconsinAges 5 months–7 months - NCT07227207RecruitingPhase 1/Phase 2
Study of TSRA-196 for adults with Alpha-1 Antitrypsin Deficiency
This trial tests a new medicine called TSRA-196 for people with Alpha-1 Antitrypsin Deficiency who have the PiZZ genetic type. The study looks at whether TSRA-196 can help with lung or liver problems related to this condition.
Boston, MassachusettsAges 18–70 - NCT04667494RecruitingPhase 4
Ultrasound treatment for heart amyloidosis
This trial studies whether ultrasound therapy can help people with amyloidosis that affects the heart. It’s aimed at improving heart-related outcomes in AL or transthyretin (ATTR) cardiac amyloidosis.
Boston, MassachusettsAges 18–90 - NCT05883748Enrolling by invitationPhase 2/Phase 3
Long-term study of bitopertin for EPP safety and benefit
This trial studies how safe and well bitopertin works over the long term for people with EPP. It mainly includes people who already finished an earlier bitopertin study, to keep tracking side effects and overall progress.
Birmingham, AlabamaAges 12 years+ - NCT04563520RecruitingPhase 3
Testing the safety of aPCC after emicizumab in hemophilia A
This study looks at whether aPCC (a clotting medicine) is safe to use for bleeding safety in people with hemophilia A who are already taking emicizumab. It mainly checks side effects and safety after switching to or using aPCC when needed.
Atlanta, GeorgiaAges 6 years+ - NCT05066217RecruitingPhase 3
Study tests clemizole for Lennox-Gastaut seizures
This Phase 3 trial studies whether clemizole helps reduce seizures in people with Lennox-Gastaut syndrome (LGS) and checks safety. If you have LGS and meet the seizure and heart-safety requirements, this could be an option to explore.
Little Rock, ArkansasAges 2–55 - NCT07450664Enrolling by invitation
Observational study of VYKAT XR for Prader-Willi syndrome
This study follows people with Prader-Willi syndrome who are already taking or starting VYKAT XR, including those switching from an earlier version of the drug (DCCR). It aims to understand how the treatment works in everyday life.
Phoenix, ArizonaAges 4 years+ - NCT04278404Recruiting
Drug safety study for children already on certain medicines
This study measures how certain medicines move through the body and checks their safety in children. It may help doctors better understand dosing and side effects in kids who are already receiving these treatments, or in some kids who recently tested positive for COVID-19.
Phoenix, ArizonaAges birth–20 years - NCT07003425RecruitingPhase 2
Long-term safety and effectiveness of APG777 for eczema
This study looks at the long-term safety and how well APG777 works for people with eczema (atopic dermatitis). It is for people who have already completed treatment with APG777 in an earlier study and may benefit from continuing it.
Fountain Valley, CaliforniaAges 18 years+ - NCT07550218Recruiting
Surfactant Delivery Methods for Premature Babies with Breathing Problems
This study looks at different ways doctors give a life-saving medication called surfactant to premature babies who are struggling to breathe. Researchers want to understand which delivery methods work best and help improve care.
GothenburgAges Any age - NCT07769957Recruiting
Brain scans for babies with slow growth in the womb
This study uses a special type of MRI (magnetic resonance imaging) to look at the brain development of babies who are growing slowly in the womb. The goal is to see if this scan can help doctors better understand this condition and how to care for these babies.
IstanbulAges 18–45 - NCT07694440RecruitingPhase 2
Study of MZE782 in Adults With PKU
This study tests a new oral medication (MZE782) for adults with PKU. It looks at how safe and effective the drug is, and it requires you to keep your diet stable for the entire study.
Oakland, CaliforniaAges 18–75 - NCT04292574Recruiting
UK Spinal Muscular Atrophy patient registry
This trial is a registry that collects health information from people with spinal muscular atrophy (SMA). It helps researchers understand the condition better and may support future studies and treatments.
Newcastle upon TyneAges Any age
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,863 recruiting medical genetics studies from the U.S. registry right now, out of 5,730 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.