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 applicable2,772
- Phase 2282
- Phase 1235
- Phase 3215
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 353
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis129
- 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
- Arizona79
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT02132741Recruiting
Eye imaging study in people with kidney disease
This study uses a painless eye scan to learn more about how kidney disease may be linked to eye changes. It may help improve understanding and future care for people with chronic kidney disease and/or high blood pressure.
Edinburgh, MidlothianAges 18–80 - NCT02795052Recruiting
Stem cell treatment trial for nerve injury
This study tests whether bone-marrow stem cells can improve nerve function in people with nerve damage that usually doesn’t recover with current care. It also checks whether the treatment is safe and whether people can be followed over time with repeated nerve exams.
Westport, ConnecticutAges 18 years+ - NCT02505464Recruiting
Pregnancies with baby birth differences
This study looks at pregnancies that are considered high-risk because the baby is affected by a difference found during pregnancy or at birth. It may help researchers understand and improve care for families dealing with these fetal (baby) anomalies.
Memphis, TennesseeAges 1 minute–50 years - NCT02735824Recruiting
Genetic study of immune problems in children
This study looks for new genetic causes of immune system conditions that can run in families. If you join, researchers will collect health information (and usually samples) from children with immune concerns and from close relatives for comparison.
Zurich, Canton of ZurichAges Any age - NCT02797366RecruitingPhase 2
Proton beam radiation for adult brain and spine tumors
This trial studies whether proton radiotherapy can treat primary brain (and sometimes spine) tumors in adults. You may join because your tumor type and situation fit specific diagnoses, including some cases needing a re-treatment plan.
GothenburgAges 18 years+ - NCT02617966Recruiting
Test of how rod and cone vision work in eye disease
This study measures how your eyes use two types of light-sensing cells (rods for dim light and cones for clearer detail). It may help researchers better understand retinal (retina) diseases and how well vision is functioning.
Bethesda, MarylandAges 5–100 - NCT02829684Recruiting
Register for people with Prader-Willi syndrome
This is a patient registry that collects information about people with Prader-Willi syndrome. It may help researchers better understand the condition and plan future studies.
ToulouseAges Any age - NCT02399748Recruiting
Long-term study of outcomes in Pompe disease
This study follows people with Pompe disease over a long period to learn how they do over time, especially those receiving enzyme replacement therapy. Your results could help doctors better understand expected outcomes and refine care.
TaipeiAges Any age - NCT02315521Recruiting
Pregnancy care study for babies with LUTO
This study tests a standardized plan of prenatal (during pregnancy) care for pregnancies where the baby has LUTO, regardless of when the diagnosis was made. It may help families by testing whether consistent prenatal monitoring and management improves outcomes for babies with this condition.
Houston, TexasAges 3 months–2 years - NCT02143830RecruitingPhase 2
Stem cell transplant chemotherapy for Fanconi anemia
This Phase 2 study tests a stem cell transplant approach using adjusted chemotherapy doses for people with Fanconi anemia. It may help replace damaged bone marrow and treat blood problems like severe aplastic anemia, MDS, or leukemia.
New York, New YorkAges 3 months+ - NCT02432079Recruiting
Study genetics in heterotaxy and certain heart defects
This study looks at the genes behind heterotaxy and related congenital (from-birth) heart defects. You may be able to join if you have this condition—or if you are a close family member of someone who does.
Indianapolis, IndianaAges Any age - NCT02419365Recruiting
PCD registry for people with suspected or confirmed ciliadisease
This registry collects information from people with primary ciliary dyskinesia (PCD), a condition that affects how the lungs and airways clear mucus. It helps researchers learn more about who has PCD, how it looks in real life, and any other health problems that may go along with it.
Münster, North Rhine-WestphaliaAges Any age - NCT02497521Recruiting
Registry for adults with ADPKD starting tolvaptan
This registry study collects information about adults with autosomal dominant polycystic kidney disease (ADPKD) who are starting or already taking tolvaptan. It may help researchers understand how tolvaptan is used in real life and what outcomes look like over time.
Müllheim, Baden-WurttembergAges 18 years+ - NCT02551081Recruiting
Genetic testing to guide care for newborn birth defects
This trial uses genetic testing to better understand the cause of a newborn’s birth defect and to help doctors choose more personalized treatment. It may help families get clearer answers and more targeted care.
Shanghai, Shanghai MunicipalityAges Up to 4 weeks - NCT02194816Recruiting
Online survey study for people with Parkinson’s or Parkinsonism
This study looks at how changing certain factors over time relates to symptoms in Parkinson’s disease and related conditions. You may help researchers by completing longer online surveys every 6 months for 5 years.
Kenmore, WashingtonAges 19 years+ - NCT02321423Recruiting
Research registry for pachyonychia congenita patients
This study builds an international database of people with pachyonychia congenita (or a closely related condition). Sharing your information may help researchers understand the condition better and improve future treatments.
Salt Lake City, UtahAges Any age - NCT02297724Recruiting
MRI checks placental health in twin or single growth restriction
This trial uses an MRI scan to look at placental health in pregnancies where the baby (or one twin) is not growing as expected. The goal is to better understand placental problems and how they may affect baby growth.
Boston, MassachusettsAges 18–45 - NCT02356653RecruitingEarly Phase 1
Stem cell transplant help for children lacking a matching sibling
This expanded-access study offers a specific type of stem cell transplant preparation that removes certain immune cells (CD3 and CD19) from donor stem cells. It is for children who need a transplant but do not have a fully matched sibling donor and whose team cannot use the hospital’s usual matching device protocol.
Philadelphia, PennsylvaniaAges Up to 30 years - NCT02824471Recruiting
Testing a chip technology for sickle cell samples
This trial tests a “biochip” device using samples from people with sickle cell disease. If it works, it may help doctors study sickle cell biology more accurately using patient samples.
Cleveland, OhioAges 12 years+ - NCT02593565Recruiting
Pregnancy blood-vessel disease registry study
This is a pregnancy registry that collects information from people with certain blood-vessel inflammatory diseases during pregnancy. It helps researchers better understand these conditions in pregnancy and may guide future care.
Tampa, FloridaAges 18–50 - NCT02804269Recruiting
Ages 16 to 90 for heart disease information collection
This is a repository program that collects health and imaging-related information to better understand heart (cardiovascular) health and disease. You may be able to join if you are in the age range and share your data for research use.
Singapore, SingaporeAges 16–90 - NCT02258724Recruiting
Adults with congenital heart disease in Swiss GUCH centers
This study is a national registry that collects health information from adults (age 18+) with congenital heart disease. It may help researchers better understand care and outcomes in people treated at specialized Swiss centers.
BaselAges 18 years+ - NCT02841553Recruiting
Online registry for Wolfram syndrome worldwide
This study builds an international online registry to learn how Wolfram syndrome (and related WFS1 conditions) changes over time. It may help researchers understand the disease course and improve future care by collecting health and family history data.
St Louis, MissouriAges birth+ - NCT02453542Recruiting
Testing how blood-clotting is managed in hemophilia with inhibitors
This study looks at different ways to help blood clot in people with hemophilia who have developed inhibitors (proteins that stop standard clotting treatments from working). It may help doctors choose the safest, most effective clotting approach for these situations.
Solna, Stockholm CountyAges 7 years+
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 5,795 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.