Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,858 recruiting studies in this area right now.
By phase
- Not applicable2,771
- Phase 2284
- Phase 1235
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- 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
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT06298071RecruitingPhase 1
Peguricase with methotrexate for uncontrolled gout
This early-phase trial tests a new injectable medication (Peguricase) combined with methotrexate to lower uric acid in people with gout that standard pills haven't controlled. You would first take a low dose of methotrexate to check your tolerance, then receive Peguricase infusions while stopping your usual gout medications.
Bengbu, AnhuiAges 18–70 - NCT06299891RecruitingPhase 2
Study of weight loss medication for youth with hypothalamic obesity
This study tests a combination of two medications, phentermine and topiramate, to see if they help young people with hypothalamic obesity lose weight safely. Hypothalamic obesity is rapid weight gain caused by injury to the hypothalamus, often from a brain tumor or its treatment.
Philadelphia, PennsylvaniaAges 6–28 - NCT06300697Recruiting
Food allergy and eczema study at University of Michigan
This study looks at how food allergies and eczema (atopic dermatitis) are related. You will have an oral food challenge (eating a small amount of a possible allergen in a safe setting) and give blood and skin samples to help researchers learn more.
Ann Arbor, MichiganAges 10–55 - NCT06300307RecruitingPhase 1/Phase 2
Study of ATX-01 for Myotonic Dystrophy Type 1
This trial tests an experimental drug called ATX-01 for people with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The study aims to see if ATX-01 can help reduce symptoms like muscle stiffness (myotonia) and improve daily function.
Los Angeles, CaliforniaAges 18–64 - NCT06301893Recruiting
Sickle cell surveillance study in Uganda
This study collects blood samples from up to 1 million people in Uganda to learn more about sickle cell disease. It helps doctors understand how common it is and how to better care for those affected.
KampalaAges Up to 1 year - NCT06302270Recruiting
Kaftrio during pregnancy and after birth
This study looks at how the cystic fibrosis medication Kaftrio works during pregnancy and after childbirth. It follows women with cystic fibrosis who are already taking Kaftrio and their newborn babies.
StockholmAges Up to 50 years - NCT06302985Recruiting
Inhaled dye for lung malformations in children
This trial tests a new way to use a dye called ICG (indocyanine green) that is inhaled like a mist. It may help doctors see and treat congenital lung malformations in children more clearly.
Wuhan, HubeiAges 3 months–14 years - NCT06305234Recruiting
Long-term immune response study for Palynziq PKU treatment
This long-term study checks how the immune system reacts to Palynziq (a treatment for PKU) over time. It involves extra blood tests for people already in another PKU study called 165-501.
Aurora, ColoradoAges Any age - NCT06306521Recruiting
Newborn screening for hundreds of genetic diseases by genome sequencing
This research study tests a new way to screen newborns for hundreds of genetic diseases using a sample of their blood or saliva. It aims to find conditions early so your baby can get the right care sooner.
San Diego, CaliforniaAges 1 day–4 weeks - NCT06309199Recruiting
Healthy Baby Digestive Health and Probiotics Study
This study looks at how common digestive problems are in healthy babies and whether probiotics can help. It involves following your baby from birth to age 1 with regular check-ups.
Bangkok, BangkokAges 3 days–1 year - NCT06311682RecruitingPhase 3
Testing a new shot plus steroid cream for children with eczema
This study tests an investigational shot (tralokinumab) plus a steroid cream for children with moderate-to-severe eczema that hasn't been controlled by creams alone. It aims to see if the combination is safe and works better than the cream alone.
Birmingham, AlabamaAges 6 months–11 years - NCT06311708Recruiting
PKP2-related heart condition natural history study
This study looks at how plakophilin 2 (PKP2)-related arrhythmogenic right ventricular cardiomyopathy (ARVC) progresses over time. You'll give blood samples to check for antibodies to a virus used in gene therapy, and help doctors understand the disease better.
San Francisco, CaliforniaAges 14–65 - NCT06313333Recruiting
National registry for moderate to severe adult atopic dermatitis
This registry tracks adults with moderate to severe atopic dermatitis (eczema) over time. It helps doctors learn more about the disease and how treatments work in real life.
Brescia, BresciaAges 18 years+ - NCT06313398RecruitingEarly Phase 1
Measuring red blood cell lifespan in sickle cell disease
This study uses a harmless vitamin (biotin) to label your red blood cells and track how long they survive. It aims to understand how different blood disorders affect red blood cell lifespan, which may help improve treatments.
Bethesda, MarylandAges 18–100 - NCT06313827Recruiting
Online program to prevent flare-ups in cystic fibrosis
This trial tests an online program designed to help people with cystic fibrosis avoid exacerbations (sudden worsening of symptoms). You participate from home using a device with internet, camera, and microphone.
ValenciaAges 6 years+ - NCT06314529Recruiting
Long-term follow-up of BRL-101 for thalassemia
This study follows people who have already received BRL-101 treatment for transfusion-dependent beta-thalassemia to check their health over time. It helps doctors understand how well the treatment works in the long run.
Nanning, GuangxiAges 5–35 - NCT06315348Recruiting
Gum shaping during braces with a vibration tool
This trial tests a new, less invasive surgical technique (using a special tool that vibrates, not a scalpel) to help reshape the gums while straightening teeth with braces. It is for people who need braces for mild to moderate crowding and have healthy gums.
MadridAges 16–70 - NCT06315556Recruiting
Study of Eylea for preterm babies with retinopathy of prematurity in the UK
This study collects information on how the medicine Eylea is given to preterm babies with retinopathy of prematurity (an eye problem in premature infants) using a special dosing device. The goal is to see how the treatment works in everyday hospital care.
Multiple LocationsAges Up to 1 year - NCT06316752Recruiting
Natural history study of sialidosis type I
This study follows people with sialidosis type I over time to understand how the condition progresses. Participants will have regular exams, blood draws, brain scans, and eye tests.
Taipei, TaipeiAges 12–80 - NCT06316778Recruiting
Pelvic floor training for women with myotonic dystrophy
This study tests whether pelvic floor muscle training (exercises to strengthen the muscles that control urine flow) can help women with myotonic dystrophy type 1 who have bladder leakage. It's a gentle, non-surgical approach that may improve quality of life.
Jonquière, QuebecAges 18 years+ - NCT06318260RecruitingPhase 1/Phase 2
Dobutamine for heart failure in wild-type ATTR amyloidosis
This trial tests whether dobutamine, a heart-strengthening medicine, can improve blood flow and symptoms in people with a specific type of amyloidosis that affects the heart and who are already on water pills.
AarhusAges 65 years+ - NCT06318143Recruiting
Increasing use of hydroxyurea for sickle cell in Nigeria
This study aims to help more people with sickle cell disease in Nigeria start taking hydroxyurea, a medicine that can prevent pain crises and other complications. It will work with clinics to make it easier for patients to get and stay on this treatment.
AbujaAges 1 year+ - NCT06319872RecruitingPhase 1
Study of disulfiram for vision in retinal degeneration
This trial tests whether disulfiram, a drug used for alcohol use disorder, can improve vision in people with retinal degeneration. You must have an alcohol use diagnosis and agree to avoid alcohol for 6 months.
Rochester, New YorkAges 18 years+ - NCT06320626RecruitingPhase 4
Personalized emicizumab dosing for hemophilia A
This trial tests whether adjusting emicizumab (Hemlibra) doses based on your body's drug levels can safely maintain good bleed control in people with hemophilia A who have been on the drug for at least a year.
Nijmegen, GelderlandAges 1 year+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,858 recruiting medical genetics studies from the U.S. registry right now, out of 5,764 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.