Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,859 recruiting studies in this area right now.
By phase
- Not applicable2,773
- Phase 2283
- Phase 1236
- Phase 3215
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis128
- Congenital Heart Disease85
- 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
- Pennsylvania66
- Ohio57
Recruiting studies3,859 total
- NCT04713800Recruiting
Dental implant bridge comparison with two implant types
This trial tests two ways of making a small back teeth bridge (a “cantilever” dental implant crown) using different materials. It may help show which material works best when replacing missing back teeth with implant support.
GenevaAges 22 years+ - NCT04720690Recruiting
Monitoring body water and blood flow in newborns
This study tests how well simple, harmless sensors and cuff checks work to monitor circulation and body water in newborns. It may help clinicians understand what “normal” looks like in both term and preterm babies, including some smaller babies.
LondonAges Up to 6 months - NCT04723862RecruitingEarly Phase 1
Spironolactone to test sleep hormone patterns in teen girls
This early-phase study tests whether spironolactone can “normalize” a key hormone timing pattern during sleep and wake cycles in mid-to-late pubertal girls who have high male-hormone levels. The goal is to learn how this medicine may affect hormone rhythms related to conditions like PCOS.
Charlottesville, VirginiaAges 10–17 - NCT04728841Recruiting
Gene therapy trial for Chinese hemophilia A
This trial tests a one-time gene therapy treatment (GS001) to help people with hemophilia A make more of the clotting factor they lack. If it works and is safe for you, it could improve how well blood clots form over time.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT04731272RecruitingPhase 2
Weekly GLP-1 medicine for blood sugar issues in adults with CF
This Phase 2 trial tests a weekly GLP-1 medicine (dulaglutide) to improve blood sugar control in adults with cystic fibrosis (CF) who have trouble managing glucose. It may help people with CF-related prediabetes or diabetes by improving how their body handles sugar.
Aurora, ColoradoAges 18 years+ - NCT04732910Recruiting
Study of CFTR-modulator effects using body samples
This study looks at how well CFTR-modulator medicines are working in people with cystic fibrosis, by checking specific “biomarkers” (measurable signals) in samples. Your care team may use the results to better understand how these medicines help, especially for certain CFTR changes.
Berlin, State of BerlinAges 6 months+ - NCT04741906Recruiting
Dental implants in people taking antiresorptive medications
This study looks at how well dental implant treatment works in people with cancer or osteoporosis who take antiresorptive medicines. It may help your team understand what to expect for healing and outcomes in your situation.
Copenhagen, DanmarkAges Any age - NCT04746066Recruiting
Study of thalassemia, sickle cell, and inherited anemia with COVID
This observational study looks at people with thalassemia, sickle cell disease, or other inherited anemia who have COVID-19. It aims to learn how COVID affects these conditions and may help improve care planning.
GenovaAges Any age - NCT04750928RecruitingPhase 1/Phase 2
Abemaciclib for NF1 tumors using pills and body scans
This trial studies whether the medicine abemaciclib can shrink or slow the growth of “atypical neurofibromas” in people with NF1. Participants take pills and undergo regular check-ups, including MRI scans to measure the tumor.
Bethesda, MarylandAges 12 years+ - NCT04763109Recruiting
MRI scan with AI to spot early changes in NF1 children
This study uses special MRI scans plus computer (AI) analysis to look for early, possibly cancer-related skin or tissue changes in children with neurofibromatosis type 1 (NF1). If it works, it could help doctors detect concerning changes earlier and plan care sooner.
Los Angeles, CaliforniaAges 5–17 - NCT04766866Recruiting
Check two blood markers to help prevent preeclampsia
This trial tests whether measuring two pregnancy blood markers (sFlt1 and PlGF) and planning delivery timing can help prevent preeclampsia as you reach term. It may help reduce risk for mother and baby by guiding when to deliver based on test results.
LiègeAges 18 years+ - NCT04770519Recruiting
Family genetic study for eye misalignment and nystagmus
This study looks at genetics in families where eye misalignment (strabismus) and/or constant eye shaking (nystagmus) runs. It may help researchers understand the causes and guide future testing or treatments.
Boston, MassachusettsAges Any age - NCT04771156RecruitingPhase 4
Ketorolac pain relief during cleft palate repair
This trial tests whether ketorolac (a pain medicine similar to other NSAIDs) helps control pain during palatoplasty, the surgery to repair a cleft palate. It may help make recovery more comfortable for people having their first palate repair.
Houston, TexasAges 6 months+ - NCT04772963Recruiting
Genetics study for brain blood vessel malformations
This study looks at genetic (inherited DNA) factors in people with certain abnormal blood vessel connections in the brain or spinal cord. It may help researchers understand why these malformations happen and how they might behave over time.
ParisAges Any age - NCT04774536RecruitingPhase 1/Phase 2
CRISPR gene-edited bone marrow transplant for severe sickle cell
This Phase 1/2 study tests a CRISPR-edited bone marrow (stem cell) transplant to treat severe sickle cell disease. It aims to reduce dangerous sickle-related complications by changing the patient’s stem cells before transplant.
Los Angeles, CaliforniaAges 12–35 - NCT04774848Recruiting
Compare two breathing machines for newborns with diaphragmatic hernia
This study compares two types of ventilator support for newborns with congenital diaphragmatic hernia. The goal is to see which breathing approach works better and is safer when started very early after birth.
Salt Lake City, UtahAges Up to 1 day - NCT04776824Recruiting
Swiss registry for people with amyloidosis and possible heart involvement
This trial is a registry that collects health information from people with amyloidosis, including cases where the heart may be involved. Sharing your medical details can help researchers learn how this condition affects patients and may guide future care.
BaselAges 18 years+ - NCT04781790Recruiting
Registry for bone marrow failure patients
This study is a national registry that collects health information and some blood or tissue samples from people diagnosed with bone marrow failure. It helps researchers understand causes and long-term outcomes, and may improve care by building a large patient database.
ParisAges Any age - NCT04782232Recruiting
Study of a new blood tube for heart pump patients
This registry study checks whether a new venous blood tube used with a heart pump is safe and practical for people whose right heart is failing and who may need a heart transplant. If you qualify, your care team would monitor how the device works over time.
MunichAges Any age - NCT04784052RecruitingPhase 1/Phase 2
Briquilimab with reduced donor stem cell transplant for Fanconi anemia
This study tests whether a reduced-intensity donor stem cell transplant (with briquilimab as part of the conditioning) can safely treat bone marrow failure in people with Fanconi anemia. It may help by lowering how much intensive treatment is needed before the transplant, while still helping the donor cells grow.
Stanford, CaliforniaAges 2 years+ - NCT04784364Recruiting
Study of immune treatments at Mount Sinai
This study follows people who are receiving immune-related medications (called biologics or immune modulators) at Mount Sinai. It aims to better understand how these treatments affect the immune system and health over time, which may help improve future care.
New York, New YorkAges 12 years+ - NCT04793867Recruiting
MRI study of lung differences in cystic fibrosis and bronchiectasis
This study uses MRI scanning to better understand how the lungs differ in people with cystic fibrosis (CF) and in people with non-CF bronchiectasis. The results may help doctors understand disease changes over time and guide future care, especially for children and teens.
Cincinnati, OhioAges 5–100 - NCT04795843Recruiting
Exercise and education versus usual care for hip dysplasia
This trial compares a structured exercise and patient education program with standard care in people with hip dysplasia. It may help improve hip and groin pain and function, especially if you are not getting the usual surgery (periacetabular osteotomy, or PAO).
Aarhus NAges 18–50 - NCT04798950Recruiting
Bronchial Dilation Study in Polynesian Patients
This is a retrospective study (looking back at medical records) examining bronchial dilation—a condition where airways in the lungs become abnormally enlarged—in Polynesian patients. Researchers want to understand this condition better by reviewing past medical records from 2010–2020.
PapeeteAges 18 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,859 recruiting medical genetics studies from the U.S. registry right now, out of 5,745 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.