Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,859 recruiting studies in this area right now.
By phase
- Not applicable2,773
- Phase 2283
- Phase 1236
- Phase 3215
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis128
- Congenital Heart Disease85
- 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
- Pennsylvania66
- Ohio57
Recruiting studies3,859 total
- NCT05500794Recruiting
Meditation for itch from psoriasis or eczema
This trial studies whether meditation can reduce itching and improve quality of life in adults with plaque psoriasis or atopic dermatitis (eczema). It may help by calming the mind and reducing discomfort related to inflammatory skin disease.
ParisAges 3 weeks+ - NCT05500807RecruitingPhase 1
Emicizumab study for severe von Willebrand disease or overlap disorder
This Phase 1 trial studies how well and safely emicizumab works for people with severe von Willebrand disease (VWD) or VWD plus hemophilia A. Emicizumab is a preventive medicine given regularly to reduce bleeding risk.
Orange, CaliforniaAges birth–90 years - NCT05500989Recruiting
Compare early pregnancy blood-flow markers in preeclampsia
This study looks at blood-flow and artery health signs during pregnancy to better understand why preeclampsia happens. It compares women with preeclampsia to women with an uncomplicated pregnancy, and may help improve early detection and care.
MaastrichtAges 18 years+ - NCT05501587Recruiting
Respiratory Health Registry measuring lung function in different groups
This registry study collects information and measures lung function to better understand respiratory health across people with cystic fibrosis, other lung diseases, and healthy volunteers. Your results may help researchers track lung health and improve future research and care.
Vancouver, British ColumbiaAges birth–99 years - NCT05502133Recruiting
Study genes in families affected by acute intermittent porphyria
This study looks at how genes may affect acute intermittent porphyria (AIP) in families. You may be asked to share health information and provide urine, blood, or saliva samples to help researchers understand what changes genes can make.
New York, New YorkAges 12 years+ - NCT05504837RecruitingPhase 1
Test KB407 inhaled treatment for adults with cystic fibrosis
This Phase 1 study tests the safety of KB407 and how it works in adults with cystic fibrosis (CF). It may help researchers understand whether this inhaled treatment could become a future option for CF.
Gainesville, FloridaAges 18 years+ - NCT05507723Recruiting
Smartphone-guided tight gout control vs usual care
This study tests whether a “tighter” gout treatment plan guided through a smartphone app can reduce gout flare-ups compared with usual care. It may help people whose uric acid levels are still high despite treatment.
EdinburghAges 18 years+ - NCT05508009RecruitingPhase 1/Phase 2
Kidney transplant with donor-matched stem cell treatment
This early-phase trial tests a stem cell transplant from a donor who is an extremely close match, given to people who are about to receive a kidney transplant from the same donor. The goal is to improve how well the new kidney works by resetting the immune system to better accept the transplant.
Palo Alto, CaliforniaAges 1–30 - NCT05508971Recruiting
Personalized tonsil surgery for sleep apnea in children with Down syndrome
This study tests whether a personalized surgical approach can treat moderate to severe obstructive sleep apnea (OSA) in children with Down syndrome. The goal is to improve breathing during sleep and overall sleep health.
Aurora, ColoradoAges 2–17 - NCT05508932Recruiting
Heart rhythm study for people with transfusion-dependent beta-thalassemia
This trial studies atrial fibrillation (an irregular heartbeat) in people with beta-thalassemia who need regular blood transfusions. It may help doctors better understand how this heart rhythm problem affects you and guide future care.
FerraraAges 18 years+ - NCT05509153RecruitingPhase 2
N-acetylcysteine trial for people at risk of Huntington’s disease
This Phase 2 study tests whether NAC (a supplement-like medicine) can delay symptoms in people who carry the Huntington’s disease gene expansion but do not yet have clear movement symptoms. You may be asked to take NAC and attend regular visits, scans, and blood tests to see if it changes how likely you are to develop Huntington’s disease within the next 10 years.
Westmead, New South WalesAges 18 years+ - NCT05512234Recruiting
Probiotic drops to reduce crying in breastfed baby colic
This trial tests whether probiotic Limosilactobacillus reuteri (a specific friendly gut bacteria) can shorten how long a baby cries when they have colic. If you join, you’ll keep feeding as usual and track crying in a diary across several visits.
CorkAges 3 weeks–2 months - NCT05514340RecruitingPhase 2
Test drug for brain injury after low oxygen at birth
This Phase 2 study tests whether sovateltide can improve safety and outcomes for babies whose brains may have been injured by low oxygen around birth. It focuses on babies who show signs of moderate to severe injury soon after delivery, and who receive standard supportive care.
Mangalore, KarnatakaAges Any age - NCT05517655RecruitingPhase 4
Cystic fibrosis blood/sweat check using MRI in children
This study looks at new “imaging biomarkers” (MRI measurements) in children with cystic fibrosis (CF). It mainly aims to see how MRI findings might relate to starting the recommended CF triple-combination therapy.
Kansas City, KansasAges 6–8 - NCT05518617Recruiting
Imaging study for inherited Parkinson’s using PET, SPECT, and spinal fluid
This trial tests how well special brain scans (PET/SPECT) and spinal fluid tests can show changes in people with inherited (genetic) Parkinson’s. It may help researchers understand what’s happening in the brain and improve how these conditions are measured.
Exeter, DevonAges 25–80 - NCT05519111RecruitingPhase 2
Cannabinoid treatment to lower inflammation and sickle pain
This Phase 2 study tests a cannabinoid medicine (dronabinol) to reduce inflammation and pain related to sickle cell disease. It may help you have less pain that interferes with daily life if you meet the study requirements and can safely avoid cannabis during the study.
New York, New YorkAges 18 years+ - NCT05521451Recruiting
Study for adults with heart rhythm problems
This trial is a “clinical cohort” study, meaning it follows adults with certain heart rhythm issues to understand them better. Your participation may help researchers learn what to expect and how best to support people over time.
HamburgAges 18 years+ - NCT05520359Recruiting
Testing spinal stimulation and devices to improve movement
This study tests spinal stimulation and mobility-related devices to see if they can improve how well you move. You would visit a lab, do simple movement tasks, and share feedback about comfort and what you experience.
Seattle, WashingtonAges 4–70 - NCT05521607Recruiting
Phone screening tool for newborn jaundice after birth
This trial tests whether a smartphone tool can help screen newborn jaundice (yellow skin) using skin measurements. It may help doctors identify jaundice sooner and decide if a blood test is needed.
Chicago, IllinoisAges 1 day–2 weeks - NCT05522374Recruiting
Study registry for people with suspected or confirmed NBIA
This trial is a research registry that collects information from people who have suspected or confirmed NBIA (a rare inherited brain movement/nerve condition). It helps researchers learn more about the condition and how it affects people, which may support future studies.
OttawaAges Any age - NCT05525039Recruiting
Vibration plus supplement trial for weak muscles and nerves
This trial studies whether a vibration treatment combined with an HMB supplement can improve weak muscle quality and reduce early nerve-to-muscle connection problems. It is mainly for older adults who already have signs of low muscle strength or function.
Hong KongAges 60 years+ - NCT05527093Recruiting
Brain-scan study mapping social thinking changes in epilepsy
This study uses special thinking and brain-scanning tests to understand how “social thinking” may change in people with certain types of epilepsy. You might benefit by having a detailed evaluation during your planned pre-surgery workup.
Gif-sur-YvetteAges 18–65 - NCT05528744Recruiting
Study genes and symptoms in ANKRD17-related CAGS
This study looks at how a specific gene change in ANKRD17 relates to the symptoms, brain/scan findings, and brain-cells in people with Chopra-Amiel-Gordon syndrome (CAGS). It may help families understand the condition better and connect gene results to what is seen clinically.
Boston, MassachusettsAges Any age - NCT05532813RecruitingPhase 3
Metformin for adult myotonic dystrophy type 1 muscle weakness
This Phase 3 trial tests whether metformin can improve symptoms and safety in adults with genetically confirmed myotonic dystrophy type 1 (DM1). It may help reduce muscle problems, but you must meet specific walking, breathing, heart, and kidney requirements.
GarchesAges 18–70
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,859 recruiting medical genetics studies from the U.S. registry right now, out of 5,745 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.