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 2283
- Phase 1235
- Phase 3216
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome54
- 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
- California340
- Maryland161
- Alabama132
- Massachusetts116
- New York84
- Arizona78
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- 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 - 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 - NCT07135167RecruitingPhase 3
Epi-ON corneal crosslinking study for people with Down syndrome
This study tests a special light treatment for cornea problems in people with Down syndrome. It aims to strengthen the front of the eye and may help prevent vision loss.
Atlanta, GeorgiaAges 8 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+ - 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 - NCT07137338RecruitingPhase 1
Gene therapy for BAG3-related dilated cardiomyopathy
This trial tests a one-time gene therapy called RP-A701 for people with dilated cardiomyopathy caused by a BAG3 gene mutation. The goal is to see if it is safe and can improve heart function.
San Diego, CaliforniaAges 18–65 - 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 - 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 - NCT07145138Recruiting
Multi-ethnic dilated cardiomyopathy registry
This study is a registry for people with dilated cardiomyopathy (DCM)—a condition where the heart's main pumping chamber becomes enlarged and weak. It aims to learn more about DCM, especially in people from different ethnic backgrounds, by collecting health information over time.
Chicago, IllinoisAges 18 years+ - NCT07145229RecruitingPhase 2
Testing a new drug for acute gout flares
This trial tests a new medication, ABP-745, for people having an acute gout flare. It aims to see if the drug is safe and helps reduce pain and inflammation.
Peoria, ArizonaAges 18–70 - NCT07145411Recruiting
Bone marrow transplant trial for patients 12 to 80
This trial provides bone marrow for transplant to patients who are already considered good candidates for a donor transplant by their own doctor. The goal is to see if using this bone marrow product is safe and helpful.
Birmingham, AlabamaAges 12–80 - NCT07146256Recruiting
OPMD Natural History Registry Study
This study is building a national registry (a database) for people with Oculo-Pharyngeal Muscular Dystrophy (OPMD) in Israel. Joining will help researchers understand how OPMD progresses over time and may lead to better care.
Ramat GanAges 18 years+ - NCT07148050RecruitingPhase 1
CAR T-cell therapy for children with solid tumors
This trial tests a new kind of immunotherapy for children and teens with solid tumors that have not responded to standard treatments. The therapy uses specially engineered T cells (CAR T cells) that target a protein called GPC3 found on many tumor cells, and it includes extra signals (IL-15 and IL-21) to help the T cells work better.
Seattle, WashingtonAges 1–26 - NCT07148583Recruiting
BioAmicus Complete for tummy troubles in babies
This study tests if a probiotic supplement called BioAmicus Complete can help babies with common digestive issues like colic, reflux, constipation, or loose stools. It might be a good option if your baby has these symptoms and you haven't used other probiotics or antibiotics recently.
Haiphong, Hai PhongAges birth–2 years - NCT07148739RecruitingPhase 4
Ensuring Best Access to CF Treatment
This study tests whether people with cystic fibrosis who are already on a triple combination CFTR modulator can stay on their treatment without interruptions or problems. It aims to make sure everyone gets the best, most consistent treatment for their CF.
Birmingham, AlabamaAges 3 years+ - NCT07150000Recruiting
Rheumatic disease research study for patients and healthy volunteers
This study is for people with certain rheumatic conditions (like arthritis, lupus, or vasculitis) and for healthy volunteers. It aims to learn more about these diseases and improve how they are treated.
Bonn, North Rhine-WestphaliaAges 18 years+ - NCT07150013RecruitingPhase 1
Vorinostat for Rett syndrome safety study
This trial tests whether vorinostat is safe and helpful for girls and young women with Rett syndrome. Participants must have a confirmed MeCP2 mutation and be in a stable phase of the condition.
MedellínAges 6–21 - NCT07151274Recruiting
Ehlers-Danlos syndrome and women's gynecological health
This study uses an online questionnaire to explore how Ehlers-Danlos syndrome (EDS) may affect gynecological health. It compares women with EDS to those without, to better understand any differences.
RouenAges 18–50 - NCT07152106Recruiting
Amniotic fluid and the preterm baby's gut
This study looks at the fluid around your baby (amniotic fluid) and how it might affect your baby's gut health if they are born early. It may help doctors understand how to better care for preterm infants.
AmsterdamAges 16 years+ - NCT07152197Recruiting
Resistance Exercise for Charcot-Marie-Tooth Disease
This trial tests whether resistance exercises (like lifting weights with your legs) can help people with Charcot-Marie-Tooth disease. It is for young adults aged 15 to 29 who can move their ankle enough to do strength training.
Temuco, La AraucaníaAges 15–29 - NCT07152392RecruitingPhase 4
Testing IBI311 for inactive thyroid eye disease symptoms
This trial tests a medication called IBI311 for people with inactive thyroid eye disease that still causes bothersome symptoms like double vision or trouble closing the eyes. The goal is to see if it helps improve these problems safely.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT07152366RecruitingPhase 4
Hormone and tetuzumab for active thyroid eye disease
This trial tests a new sequence of hormone therapy followed by a targeted drug (tetuzumab) for people with moderate to severe thyroid eye disease that is still active after steroid treatment didn't help enough.
Shanghai, Shanghai MunicipalityAges 18–80
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 5,779 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.