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
- NCT07143201RecruitingPhase 2
Precision dosing of ibuprofen for PDA in premature babies
This study tests a personalized way to give ibuprofen to very premature babies with a heart condition called PDA. It aims to see if adjusting the dose can help close the heart opening more safely.
Hamilton, OntarioAges Up to 6 months - NCT07138274RecruitingPhase 2
Metyrapone study for mild cortisol excess
This study tests if a medication called metyrapone can help people with mild autonomous cortisol secretion (MACS), a condition where your body makes too much cortisol. It looks at whether the drug improves related health problems like high blood pressure, diabetes, or bone issues.
Rochester, MinnesotaAges 18 years+ - NCT07138963Recruiting
Gene study in children with congenital muscle weakness
This study looks at how genes affect symptoms in children with congenital myopathies or congenital muscular dystrophies. It may help doctors better understand these conditions.
CairoAges 1–18 - NCT07137286Recruiting
Genes and high LDL on a ketogenic diet
This study looks at how genes might affect why some people have a big rise in LDL cholesterol when they start a ketogenic diet. If your LDL is over 170 mg/dL on keto, you may be able to join a blood draw to help find out.
Rochester, MinnesotaAges 18 years+ - NCT07142135Enrolling by invitation
Long-term follow-up for Klinefelter syndrome patients
This study follows patients with Klinefelter syndrome over time to learn more about their health. It may help doctors better understand and care for people with this condition.
CopenhagenAges Any age - NCT07142629Enrolling by invitation
Cavity removal methods for MIH-affected molars
This trial compares two ways of removing tooth decay in children with a condition called MIH (which makes enamel weak and spotted). It tests whether selective or non-selective drilling works better for deep cavities, aiming to save the tooth and keep the nerve healthy.
IrbidAges 6–13 - NCT07136844Recruiting
Walking and arm movement study for nerve and muscle conditions
This study uses motion analysis to understand walking and arm function in adults with certain neurological or metabolic diseases. The goal is to find patterns that help doctors better manage these conditions.
LiègeAges 18 years+ - NCT07136857RecruitingPhase 2
Eptacog beta for bleeding in Glanzmann's thrombasthenia
This trial is testing a medication called eptacog beta (a clotting factor) to see if it helps control or prevent severe bleeding episodes in people with Glanzmann's thrombasthenia, a rare inherited bleeding disorder.
Atlanta, GeorgiaAges Any age - NCT06552052Recruiting
Natural history study of genetic blood vessel disease
This study tracks the health of people with a confirmed ACTA2 gene change over time to better understand the disease. It may help researchers learn how to improve care for this condition.
Boston, MassachusettsAges 4 weeks+ - NCT06877715Recruiting
Sensory and autism traits in children with Prader-Willi syndrome
This study looks at autism-like symptoms and sensory profiles in children with Prader-Willi syndrome (PWS). It aims to better understand these traits to improve care and support.
ToulouseAges 3–16 - NCT07129343RecruitingPhase 3
Study of a new VWF treatment for von Willebrand disease in China
This Phase 3 trial tests an recombinant version of the von Willebrand factor (rVWF) for people with severe von Willebrand disease (VWD) living in China. It aims to see if the drug can safely control bleeding and help those who need regular clotting factor treatments.
BeijingAges 18 years+ - NCT07135050RecruitingPhase 1/Phase 2
Gene therapy study for Pitt Hopkins syndrome
This trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins syndrome. The therapy is given as a single injection into the fluid around the brain to help replace the missing or faulty TCF4 gene.
Oakland, CaliforniaAges 2–25 - NCT07127978Recruiting
Study of Givinostat in people with Duchenne muscular dystrophy
This study is for people with Duchenne muscular dystrophy (DMD) who are taking Givinostat, a daily oral medicine. Researchers will look at real-world data to see how the medicine works in everyday life, including lab results and how it's being used.
Little Rock, ArkansasAges 6 years+ - NCT07126262RecruitingPhase 2
Vosoritide for growth in infants with hypochondroplasia
This study tests a daily injection called vosoritide to see if it helps children with hypochondroplasia grow taller. It is for babies and toddlers up to 35 months old who are shorter than average for their age.
Phoenix, ArizonaAges birth–3 years - NCT07135427RecruitingPhase 4
Genes and vaccines in alpha-1 antitrypsin deficiency
This study looks at how genetic differences in alpha-1 antitrypsin deficiency affect immune response to a pneumonia vaccine. It may help find better ways to prevent lung infections.
Birmingham, AlabamaAges 19 years+ - NCT07133464Recruiting
CDH1 gene and BCDS syndrome registry
This registry studies people with changes in the CDH1 gene who may have blepharocheilodontic syndrome (BCDS). It collects information to better understand the condition and improve care.
Columbus, OhioAges Any age - NCT07134621Enrolling by invitation
Blood vessel damage in repaired aortic narrowing with exercise-related high blood pressure
This study checks if people with repaired coarctation of the aorta who develop high blood pressure only during exercise have any damage to their blood vessels or organs. It aims to understand how exercise affects long-term health after aortic repair.
LjubljanaAges 18 years+ - NCT07130565Recruiting
Pan-Amyloid PET/CT Scan for Amyloid Heart Disease
This trial is testing a special PET/CT scan (Pan-Amyloid) that finds abnormal protein deposits (amyloid) in the heart and body. It is for people who have or may have cardiac amyloidosis and are already planning to get this scan.
Hefei, AnhuiAges 18–80 - NCT07129954Recruiting
Fear of Falling in Muscular Dystrophy Trial
This trial tests a treatment to help people with certain types of muscular dystrophy who are afraid of falling. It aims to improve confidence and walking safety.
Verona, veronaAges 16–65 - NCT07131293Recruiting
European registry study on lymphatic treatments
This study is for people of any age with a confirmed central lymphatic disorder (a problem in the main lymphatic vessels, often linked to heart conditions or occurring on its own). Researchers want to collect information from different hospitals to learn more about these disorders and the best ways to treat them.
CopenhagenAges Any age - NCT07129538Recruiting
Breathing exercise for teens with FMF
This trial tests whether a special breathing exercise can help teens with FMF breathe better and feel stronger. It is for adolescents aged 13 to 17 who have FMF and are otherwise healthy.
Giza, DokkiAges 13–17 - NCT07134049Recruiting
Sensory therapy for kids with brachial plexus injury
This study tests whether sensory integration therapy helps young children with a brachial plexus injury (nerve damage in the arm from birth) improve their development. It may be a good option for children who have not had other therapies recently and do not have other major health issues.
AnkaraAges 7 months–2.9 years - NCT07133789Recruiting
Better understanding chromosome changes in children with developmental disorders
This study uses a new technique called optical mapping to better understand chromosome changes found in children with neurodevelopmental disorders. It aims to give clearer answers to families about these genetic findings.
Paris, ParisAges 2 years+ - NCT07134387Recruiting
Long-term effects of BPD on family quality of life
This study looks at how bronchopulmonary dysplasia (BPD), a lung condition in premature babies, affects the long-term health and quality of life of children and their families. Parents or caregivers of children born early with or without BPD can join to compare experiences.
München, BavariaAges birth–18 years
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.