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
- NCT07254000RecruitingPhase 2
Anakinra dosing study for preterm infants
This trial tests different doses and ways of giving a medicine called anakinra to preterm babies. It aims to find the safest and most effective dose to help reduce inflammation.
Clayton, VictoriaAges 6 months–7 months - NCT07255131Recruiting
Online talk therapy for cystic fibrosis
This study tests an online cognitive behavioral therapy (a type of talk therapy) program designed for people with cystic fibrosis. It aims to help improve mental health and coping skills through 13 video sessions.
AthensAges 18–70 - NCT07259343Recruiting
Cream with milk peptide for children's mild eczema
This trial tests if a special cream made with a milk protein peptide can improve mild eczema symptoms like itching and reduce bacteria on the skin in children aged 2 to 12.
Aguascalientes, AguascalientesAges 2–12 - NCT07253038Recruiting
Testing social skills in Huntington's disease
This study tests three different ways to measure social skills (like understanding emotions or reading social cues) in people with Huntington's disease. The goal is to find which test works best for future research and care.
LundAges 18–75 - NCT07259356Enrolling by invitation
Blood-flow-restriction training for hemophilia patients
This trial tests whether a special type of exercise that limits blood flow is safe and possible for people with hemophilia. It might help strengthen muscles without causing joint bleeds.
Bonn, North Rhine-WestphaliaAges 18 years+ - NCT07252050RecruitingPhase 1/Phase 2
Ruxolitinib and half-matched transplant for severe sickle cell disease
This trial tests a new approach using the drug ruxolitinib to help a half-matched bone marrow transplant work better for people with severe sickle cell disease. It aims to reduce complications and improve outcomes.
Aurora, ColoradoAges 12–45 - NCT07250906Recruiting
Oral health and life quality in children with enamel defects
This study measures how a child's daily life and happiness are affected by a condition called Amelogenesis Imperfecta, which affects tooth enamel. It focuses on children who need veneers for their front teeth, and the results will help dentists improve care.
IstanbulAges 11–14 - NCT07250945Recruiting
Laser treatment for birthmarks that are at least 2.5 cm across
This trial tests a laser treatment for vascular birthmarks (like port-wine stains). It might help if your birthmark is on an area of your body that is not the center of your face and you have light to medium skin.
Szeged, Csongrád-Csanád VármegyeAges 7–65 - NCT07255144Recruiting
Ultrafast ultrasound imaging for children's heart conditions
This study tests a new, faster ultrasound technique to see heart structure and blood flow in children with or without congenital heart disease (CHD). It aims to improve imaging without risk, using a short scan during a regular check-up.
Pessac, FranceAges Any age - NCT07250854RecruitingEarly Phase 1
Using a dye to help diagnose blocked bile ducts in newborns
This study tests if a special dye (ICG) and a camera can help doctors see blocked bile ducts in newborns with jaundice, so they can diagnose biliary atresia faster and without extra procedures.
Birmingham, AlabamaAges 2 weeks–4 months - NCT07253051Recruiting
Study for women with BRCA gene mutations
This study creates a platform for women who carry a BRCA1 or BRCA2 gene mutation to be followed over time. It aims to better understand and manage cancer risks associated with these mutations.
AvellinoAges 18 years+ - NCT07251790Recruiting
Better breathing support for very preterm babies
This trial tests different ways to help very preterm babies when they are first taken off a breathing machine and switched to CPAP. It aims to find the safest and most effective breathing support to help them breathe on their own.
Melbourne, VictoriaAges birth+ - NCT07250997Recruiting
PALLAS laser study for skin conditions
This trial tests a new laser (PALLAS) that delivers targeted UVB light to treat skin conditions like psoriasis or eczema. It may help if your doctor thinks your skin problem could improve with UVB therapy.
Szeged, Csongrád-Csanád VármegyeAges 18–65 - NCT06529874Recruiting
Pain, balance, and strength in children with sickle cell
This study looks at how children (ages 9-14) with sickle cell anemia or who are healthy handle pressure, balance, and strength. It aims to understand pain and movement better to help improve care.
GizaAges 9–14 - NCT07239947RecruitingPhase 1
Testing BBT001 for healthy volunteers and people with eczema
This trial tests a new medicine called BBT001, first in healthy people and then in adults with moderate to severe eczema (atopic dermatitis). It aims to see if the drug is safe and how well it works for treating eczema that hasn't improved with topical treatments.
Hefei, AnhuiAges 18–65 - NCT07247188RecruitingPhase 3
Pneumococcal vaccine study for children with sickle cell disease
This trial tests a single dose of a new pneumococcal vaccine (21-valent) in children and teens with sickle cell disease. It aims to see if the vaccine can help protect against serious infections caused by pneumococcus bacteria, which are especially dangerous for people with SCD.
Atlanta, GeorgiaAges 2–17 - NCT07241104RecruitingPhase 1
Study of a new drug for dilated cardiomyopathy with a specific genetic change
This trial tests an experimental drug called AZD4063 for people with dilated cardiomyopathy caused by a specific genetic mutation (R14del in the PLN gene). The goal is to see if the drug can improve heart function and symptoms.
AmsterdamAges 18–80 - NCT07241234RecruitingPhase 1
Study of an experimental drug for phenylketonuria (PKU)
This trial tests an investigational drug (AG-181) for people with PKU who have a specific genetic change (R408W mutation). It aims to check if the drug is safe and how it works in your body.
Indianapolis, IndianaAges 18–69 - NCT07240896RecruitingEarly Phase 1
Testing a new mRNA therapy for Wilson disease
This trial tests an experimental mRNA treatment (DSL101) for adults with Wilson disease. It's for people whose condition is stable on current therapy and who have certain genetic markers. The goal is to see if the new treatment can help the body process copper better and reduce the need for standard medicines.
Hefei, AnhuiAges 18 years+ - NCT06190210Recruiting
Brain blood flow study in infants with heart defects
This study measures how well blood flows to the brain in infants with serious heart defects. The results may help doctors understand and protect brain development before and after heart surgery.
Leuven, Vlaams-BrabantAges Up to 6 months - NCT06900049RecruitingEarly Phase 1
Testing LE051 for boys with Duchenne muscular dystrophy (exon 51)
This study tests a new medicine called LE051 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change that allows 'exon 51 skipping.' The goal is to see if it is safe and can help improve muscle function. Boys who join will be closely monitored for heart and lung health.
Shanghai, Shanghai MunicipalityAges 4–8 - NCT06905678Recruiting
Cleft palate cost and emotion study
This study looks at how having a cleft palate affects your family's finances and emotions. You may be able to join if you are 5, 12, or 18 years old and have a cleft lip or palate.
GhentAges 4–22 - NCT07247058RecruitingPhase 4
Testing Isturisa for mild cortisol excess from adrenal tumors
This study tests whether a drug called Isturisa (osilodrostat) can safely lower mild cortisol excess in people with adrenal tumors. It aims to help patients avoid the long-term effects of too much cortisol.
Baltimore, MarylandAges 18 years+ - NCT07248267Recruiting
Vojta therapy for children with Down syndrome
This study is testing a therapy called Vojta therapy to see if it helps children with Down syndrome move better and walk more easily. It compares the therapy to other supportive care.
Lahore, Punjab ProvinceAges 2–10
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.