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
- NCT07224360RecruitingPhase 2
Safety of a new drug for sickle cell pain
This study tests a new medicine called anumigilimab to see if it is safe for people with sickle cell disease. The study focuses on those who have had pain crises recently, to help manage and prevent them.
Little Rock, ArkansasAges 18 years+ - NCT07746427Recruiting
Mindfulness meditation for teens with eczema
This study tests whether a smartphone-based mindfulness meditation program can help teenagers with moderate to severe eczema (atopic dermatitis) that hasn't improved with standard creams. Participants will learn relaxation techniques to possibly reduce itching and skin inflammation.
New York, New YorkAges 12–17 - NCT07748169Recruiting
Observational study of skin disease patients
This study looks at the health records of people with skin diseases to learn more about their care and outcomes. It may help improve future treatments.
Winston-Salem, North CarolinaAges Any age - NCT07375602Recruiting
AI tool to predict complications after VSD closure in children
This study uses artificial intelligence to predict complications in children who have had a specific type of hole in the heart (called a perimembranous ventricular septal defect) repaired with a catheter-based procedure. The goal is to help doctors better identify which children might have problems after the procedure.
Shanghai, Shanghai MunicipalityAges Up to 18 years - NCT06674109RecruitingPhase 4
Testing a diabetes drug for gout prevention
This study tests if a type 2 diabetes medication (SGLT2 inhibitor) can help people with frequent gout flares. It aims to lower uric acid levels and reduce flare-ups.
Boston, MassachusettsAges 18–80 - NCT07228962Recruiting
Skin clues for eczema in babies
This study looks at tiny skin samples from babies to find clues about eczema. It uses a gentle, painless device that sucks up a small piece of skin. If your baby has eczema or is healthy and under 6 months old, you may be able to join.
LondonAges birth–6 months - NCT07566494RecruitingPhase 1
Testing VAS-101 for sickle cell disease
This early-phase trial tests a new drug called VAS-101 for people with sickle cell disease. It aims to find a safe dose and see how the drug affects the body.
Bethesda, MarylandAges 18–90 - NCT07298447RecruitingPhase 3
Donidalorsen for children with hereditary angioedema
This study tests a new medicine, donidalorsen, to prevent swelling attacks in children ages 2 to 11 with hereditary angioedema (HAE). If your child has HAE type 1 or 2, this trial may help find a treatment option.
Santa Monica, CaliforniaAges 2–11 - NCT07434037Recruiting
Trust and the brain in Down syndrome and Fragile X
This study explores how people with Down syndrome or Fragile X syndrome learn to trust others. It uses simple tasks and a brain scan (MRI) to compare their responses with those of typically developing children and young adults.
BronAges 3–29 - NCT06902558RecruitingPhase 2
Study of ABBV-CLS-628 for adults with polycystic kidney disease
This trial tests a new drug, ABBV-CLS-628, for people with autosomal dominant polycystic kidney disease (ADPKD). The goal is to see if it slows kidney function decline and is safe to use.
Birmingham, AlabamaAges 18–55 - NCT05596539Recruiting
Study of adults with low alkaline phosphatase
This is a long-term observational study (not a drug trial) that follows adults with signs of hypophosphatasia, a condition linked to very low alkaline phosphatase levels and sometimes a gene change. It aims to better understand the condition over time and how symptoms look and progress.
Bordeaux, FranceAges 18 years+ - NCT06396546Recruiting
Indian Children with Glycogen Storage Disease Registry
This study is creating a registry (a database) to better understand glycogen storage diseases in Indian children. If your child has a confirmed genetic diagnosis, joining helps doctors learn more about the condition and improve care.
Guindy, ChennaiAges Up to 18 years - NCT07681089RecruitingPhase 2
Foscenvivint for HIV/HCV cirrhosis with hemophilia
This trial tests a drug called foscenvivint for people with hemophilia and liver cirrhosis caused by both HIV and hepatitis C (already treated). The study aims to see if it can help improve liver health.
Sapporo, HokkaidoAges 18–79 - NCT06379789RecruitingPhase 1/Phase 2
Gene therapy for severe hemophilia B
This trial tests a new gene therapy (REGV131-LNP1265) that aims to let your body produce its own factor IX, reducing or stopping the need for regular infusions. It is for children and adults with severe hemophilia B who are already using factor IX replacement therapy.
Los Angeles, CaliforniaAges 2 years+ - NCT07594236RecruitingPhase 1
Experimental Cell Therapy for Retinal Degeneration
This early-stage study tests a new cell-based treatment (C.001) for inherited retinal diseases that affect the back of the eye. The treatment may help slow vision loss or improve sight in people with geographic atrophy or Stargardt disease.
Beverly Hills, CaliforniaAges 18 years+ - NCT07656415RecruitingPhase 3
Study of mitapivat for sickle cell disease patients who need blood transfusions
This study tests whether a daily pill called mitapivat can reduce the number of blood transfusions you need. It is for people with sickle cell disease who have had at least one transfusion in the past year and are not on regular transfusion therapy.
Indianapolis, IndianaAges 12 years+ - NCT06708338Recruiting
Bevel direction during dialysis fistula puncture
This study looks at which way the needle bevel should face when starting dialysis through a new fistula. It might help reduce pain or damage to the fistula.
Le Puy-en-VelayAges 18–85 - NCT06563895RecruitingPhase 3
Testing acoramidis to prevent ATTR amyloidosis in gene carriers
This trial tests a daily pill called acoramidis to see if it can prevent ATTR amyloidosis in people who carry a gene mutation but have no symptoms yet. It aims to stop the disease before it starts.
La Jolla, CaliforniaAges 18–75 - NCT06244212Recruiting
Atopic dermatitis treatment with steroid cream trial
This trial tests a common steroid cream (triamcinolone) for atopic dermatitis (a type of eczema). It aims to see how well it works in people who have active skin inflammation and can come back for a follow-up visit one week later.
Winston-Salem, North CarolinaAges 9 years+ - NCT06994520Enrolling by invitationPhase 2
Topical cannabis extract for eczema itch
This study tests a cannabis-based cream on eczema-related itch over 4 weeks. It may help reduce itching if other treatments haven't worked, but requires pausing other skin products during the trial.
Khon Kaen, Changwat Khon KaenAges 25 years+ - NCT05586243Recruiting
MRI lab measurements in adults with type 3 Gaucher disease
This study looks at MRI-based lab measurements (similar to a special type of scan) to find markers that reflect health in people with type 3 Gaucher disease. It may help researchers track the disease over time and improve future treatments.
Minneapolis, MinnesotaAges 18–80 - NCT07226206RecruitingPhase 1/Phase 2
Gene therapy for adults with severe hemophilia A
This trial tests a new gene therapy called SPK-8011QQ for adults with severe or moderately severe hemophilia A. The goal is to see if a single infusion can help your body produce its own factor VIII, reducing or stopping the need for regular factor VIII injections.
Orange, CaliforniaAges 18 years+ - NCT01060371Recruiting
Study of genetic causes of spinocerebellar ataxia
This study follows people with certain types of spinocerebellar ataxia (a group of conditions that cause progressive movement problems) to understand how the disease changes over time and how genes might influence it. It may help researchers learn more about these conditions and support future treatments.
Los Angeles, CaliforniaAges 6 years+ - NCT07742046Enrolling by invitation
Heart blood flow scan for congenital heart disease
This study tests a new MRI technique to measure blood flow in the heart without invasive procedures. It may help doctors better understand and manage congenital heart conditions.
LausanneAges 16 years+
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.