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
- NCT06711666Recruiting
Maternal mental health and baby's brain in heart disease
This study looks at how a mother's mental health during pregnancy may affect the brain development of babies with a serious heart condition called critical cyanotic congenital heart disease (CHD). It aims to understand this link to improve support for families.
MarseilleAges Up to 50 years - NCT07089875RecruitingPhase 3
Testing dotinurad vs allopurinol for people with gout
This study compares a new gout medicine called dotinurad to the common medicine allopurinol. It is for people who have had gout for at least a year, have had at least 2 gout attacks in the last year, and are already taking a stable dose of allopurinol but still have high uric acid levels.
Birmingham, AlabamaAges 18–75 - NCT00246857Recruiting
Screening study for inherited immune system conditions
This study screens people who may have an inherited immune system condition (like problems with immune cell growth or death) and their blood relatives. The goal is to understand the condition better by collecting blood samples for testing.
Bethesda, MarylandAges 1 month–100 years - NCT07688746RecruitingPhase 1
Helping premature babies heal brain injury
This trial tests a treatment for brain injury in premature babies born before 32 weeks. It aims to protect and repair white matter, the part of the brain that helps nerve cells communicate.
San Francisco, CaliforniaAges 3 weeks–5 months - NCT07161544RecruitingPhase 1/Phase 2
Gene therapy for Stargardt disease
This trial tests a new gene therapy, AAVB-039, for people with Stargardt disease caused by a specific gene mutation. The treatment is given during eye surgery and aims to slow or stop vision loss.
Phoenix, ArizonaAges 8–55 - NCT05329350Recruiting
Device follow-up for a small hole in the heart
This follow-up study monitors how well and how safely the Occlutech mVSD device works to close certain muscular heart “holes” (ventricular septal defects). You may join if you already had the device placed and can return for scheduled checkups to help doctors learn long-term outcomes.
DublinAges 3 months–80 years - NCT07535034RecruitingPhase 2
Testing Dotinurad for Gout in People Who Can't Take Other Medications
This trial tests a new drug called dotinurad for gout patients whose standard medications either don't work or cause side effects. Dotinurad works differently by helping your body get rid of uric acid (the substance that causes gout attacks), and the study will see if it safely lowers uric acid levels.
Granada Hills, CaliforniaAges 18–75 - NCT07082725RecruitingPhase 3
Study of oral nizubaglustat for Niemann-Pick type C
This study tests an oral medication called nizubaglustat for people with Niemann-Pick type C disease who cannot take or are not satisfied with the current treatment miglustat. It aims to see if the drug can help with symptoms like coordination and movement problems.
Oakland, CaliforniaAges 4 years+ - NCT07220226Recruiting
Fontan circulation and venous compression study
This trial looks at how blood flow works in people with Fontan circulation, who have a unique heart surgery. It aims to understand vein compression and may help guide future care.
Dallas, TexasAges 18–45 - NCT07204938Enrolling by invitationPhase 3
Long-term study of navenibart for hereditary angioedema
This study tests navenibart over a longer period to see if it continues to prevent swelling attacks in people with hereditary angioedema. It is for people who were already in a previous navenibart study.
Scottsdale, ArizonaAges 12 years+ - NCT01500447Recruiting
Study of inherited causes of low puberty hormones
This study looks at families to understand inherited (genetic or family-related) reasons some people have delayed puberty or develop puberty abnormally. It may help doctors better figure out the cause of low sex hormone levels and related hormone problems.
Bethesda, MarylandAges 1 month–120 years - NCT07216027RecruitingPhase 2
A study of a new eczema medicine for adults
This trial tests a study drug (PF-08049820) for people with moderate to severe eczema who haven't found a suitable prescription treatment. It aims to see if the drug can improve skin symptoms and itching.
Huntington Beach, CaliforniaAges 18–64 - NCT03581318Recruiting
Heart MRI study for people with and without heart disease
This study uses magnetic resonance imaging (MRI) to take detailed pictures of the heart. It aims to improve how doctors use heart MRI to diagnose and manage heart conditions.
Bethesda, MarylandAges 7–100 - NCT07695701RecruitingPhase 4
Heart imaging study for a new ATTR-CM treatment
This trial uses heart scans to see how the drug acoramidis affects heart structure and function in people with a specific type of heart disease called ATTR cardiomyopathy. It may help doctors understand if the treatment is working.
St Louis, MissouriAges 18–80 - NCT07140614RecruitingPhase 1
Study of EDK060 for adults with CMT1A
This trial tests a new medicine called EDK060 for people with Charcot-Marie-Tooth disease type 1A (CMT1A). The study will check if the drug is safe and how the body processes it, with the goal of finding better treatments for this condition.
Ottawa, OntarioAges 18–60 - NCT04642729Enrolling by invitation
Fresh cornea lenticule implant for macular corneal dystrophy
This trial tests a new way to treat macular corneal dystrophy using a thin piece of clear cornea (a lenticule) from a donor. Instead of a full transplant, the surgeon uses a laser (RELEX SMILE) to insert this lenticule to improve vision.
PristinaAges 20–55 - NCT02447874RecruitingPhase 1/Phase 2
Arginine treatment for children’s sickle cell pain
This early-phase study tests whether arginine can help control pain in children with sickle cell disease. You may be considered if your child is having a sickle cell pain episode that needs stronger pain medicine given through an IV.
Atlanta, GeorgiaAges 7–21 - NCT02316314Recruiting
Study heart scan and function in people with Friedreich’s ataxia
This study uses heart tests (including ultrasound and MRI) to better understand how Friedreich’s ataxia affects the heart. You might benefit by getting detailed heart evaluation and contributing to research that could guide future care.
New York, New YorkAges 12–50 - NCT07298395RecruitingPhase 2
Study of ENV-294 for moderate-to-severe eczema
This trial tests an experimental cream or pill called ENV-294 in adults whose eczema is moderate to severe and hasn't gotten better with standard creams. It aims to see if ENV-294 can help control symptoms like itching and skin redness.
Birmingham, AlabamaAges 18–75 - NCT06667453RecruitingPhase 2
PGN-EDODM1 for myotonic dystrophy type 1
This study tests an experimental drug called PGN-EDODM1 for people with myotonic dystrophy type 1 (DM1). The goal is to see if it can help reduce symptoms like muscle stiffness (myotonia).
Calgary, AlbertaAges 16–65 - NCT07734415Enrolling by invitationPhase 2
Long-term safety of IMG-007 for moderate-to-severe eczema
This study checks the long-term safety and effectiveness of IMG-007 in people with moderate-to-severe atopic dermatitis (eczema) who have already finished a previous IMG-007 trial.
Lancaster, CaliforniaAges 18–75 - NCT07729787Recruiting
Autogenic drainage vs. chest percussion for kids with CF
This trial compares two breathing techniques to help clear mucus from the lungs of children with cystic fibrosis. It aims to see if one method (autogenic drainage) works better than another (mechanical percussion with a vest).
Multan Khurd, Punjab ProvinceAges 6–12 - NCT07732257Recruiting
Wireless button sensor study for premature infants
This study tests a small, wireless button-like sensor to monitor vital signs in premature infants in the NICU. It may offer a gentler way to track your baby's health.
Montreal, QuebecAges Any age - NCT07729982Recruiting
Study tracking vision changes in OPA1 optic atrophy
This study follows people with a genetic form of optic atrophy over time to understand how vision changes. It does not test a treatment but helps doctors learn more about the condition to improve care.
Munich, BavariaAges Any age
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.