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
- NCT05987449RecruitingPhase 1/Phase 2
Study of NXT007 for severe or moderate hemophilia A
This trial tests a new medicine called NXT007 to see if it is safe and effective for people with severe or moderate hemophilia A, including those with inhibitors. It may help reduce bleeding episodes.
Sacramento, CaliforniaAges 2–59 - NCT05689151Recruiting
Abrocitinib for adult moderate to severe atopic eczema
This study tests how well abrocitinib (a prescription medicine) works for adults with moderate to severe atopic dermatitis (atopic eczema). You may join if you meet the study’s health and consent requirements and your doctor believes abrocitinib is an appropriate option for you.
Amiens, SommeAges 18 years+ - NCT07685210RecruitingPhase 1
GenSci144 tablet safety study for healthy volunteers
This trial tests the safety of a new tablet called GenSci144 in healthy adults. It helps doctors see if the drug is safe and how the body handles it.
Jinan, ShandongAges 18–45 - NCT06576323Recruiting
Support for mothers with opioid use disorder and babies in the NICU
This study aims to improve care for mothers with opioid use disorder and their babies with neonatal abstinence syndrome. Researchers will interview healthcare providers and mothers to develop better support programs.
Philadelphia, PennsylvaniaAges 18 years+ - NCT07686341Recruiting
Precision oxygen monitor study for infants
This trial tests a new type of oxygen monitor (pulse oximeter) in babies under 1 year old. It helps doctors better track oxygen levels in critically ill infants to improve care.
Memphis, TennesseeAges birth–1 year - NCT06389136RecruitingPhase 3
Study of upadacitinib for eczema after dupilumab didn't work
This trial tests if a pill called upadacitinib (a JAK inhibitor) can help adults with moderate-to-severe atopic dermatitis (eczema) that didn't improve enough with dupilumab. You may be able to join if your eczema is still active despite using dupilumab for at least 4 months.
Birmingham, AlabamaAges 18–63 - NCT07684898RecruitingPhase 3
Long-acting factor VIII for severe hemophilia A
This trial tests a new long-acting factor VIII treatment (FRSW107) for people with severe hemophilia A. It aims to prevent bleeding episodes with fewer injections, and you must have already used factor VIII treatments many times before.
Tianjin, Tianjin MunicipalityAges 12–65 - NCT07682519Recruiting
Home vs. in-person rehab for hemophilia pain
This study compares a rehabilitation program you can do at home using video calls (telerehabilitation) with in-person rehabilitation sessions. The goal is to see if the home-based option works as well as office visits for reducing chronic joint pain in adults with hemophilia.
Santiago, Santiago MetropolitanAges 18–60 - NCT05901077Recruiting
European cystinosis patient follow-up study
This study follows people with confirmed cystinosis to better understand the condition. It mainly checks that you have the right diagnosis and can join voluntarily.
Paris, Île-de-France RegionAges Any age - NCT07682883Enrolling by invitation
Mouth screening to prevent oral cancer
This study checks how well routine mouth exams can find early signs of oral cancer. It is for adults who do not already have a condition called OPMD (a mouth problem that can lead to cancer) and are not being treated for it.
Warsaw, Masovian VoivodeshipAges 18 years+ - NCT07569887Recruiting
Brain Imaging Study of Arteriovenous Malformations Before and After Surgery
This research study uses special brain imaging scans to understand how an unruptured arteriovenous malformation (an abnormal tangle of blood vessels in the brain) affects blood flow and thinking abilities before and after surgical treatment. The goal is to learn how surgery impacts brain function and cognition in patients like you.
Montpellier, FranceAges 18 years+ - NCT07598383RecruitingPhase 2
Gel treatment for mild-to-moderate atopic dermatitis
This study tests a new gel called VC005 to see if it safely reduces itching, redness, and irritation in people with atopic dermatitis (a chronic skin condition). You'll be randomly assigned to either the new gel or a placebo (inactive gel) and apply it to affected areas over several weeks.
Hangzhou, ZhejiangAges 18 years+ - NCT07277660RecruitingPhase 2
Study of galvokimig for adults with moderate-to-severe eczema
This trial tests whether a new medication, galvokimig, can help adults with moderate-to-severe atopic dermatitis (eczema) that hasn't improved enough with creams or ointments. The study will measure how well the drug works, its safety, and how it behaves in your body.
Oceanside, CaliforniaAges 18 years+ - NCT07048574Recruiting
Mood and behavior in children with cystic fibrosis
This study looks at mood and behavior in children with cystic fibrosis (CF) under 12. By following children over time, researchers hope to better understand and help with depression, anxiety, and behavior challenges that may come up.
Orange, CaliforniaAges 1.5 years–11 years - NCT01666080Recruiting
Second bone marrow transplant with gentler chemotherapy
This trial studies a “reduced intensity” second transplant using donated blood-forming cells, for people whose first transplant did not fully and reliably restart blood production. It aims to improve donor blood cell growth while using a less harsh conditioning approach.
Minneapolis, MinnesotaAges Up to 55 years - NCT07592585RecruitingPhase 3
Testing VC005 cream for mild-to-moderate eczema in teens and adults
This study tests a new medication called VC005 to treat atopic dermatitis (eczema)—a skin condition causing itching, redness, and inflammation. Researchers want to see if VC005 can safely reduce symptoms in people aged 12 and older with mild-to-moderate cases.
Nanjing, JiangsuAges 12 years+ - NCT06789445RecruitingPhase 1/Phase 2
Cell therapy for inherited retinal disease (CLARICO)
This study tests a new cell therapy (OpCT-001) for adults with inherited retinal diseases that affect the light-sensing cells. It aims to see if the treatment is safe and possibly helps improve vision.
Los Angeles, CaliforniaAges 18 years+ - NCT06708429Recruiting
Lynch syndrome and gut immune system study
This study looks at how the immune system interacts with the gut lining in people with Lynch syndrome and those without. It may help us better understand how Lynch syndrome affects the body.
Monrovia, CaliforniaAges 18 years+ - NCT06708468Recruiting
Personalized training for rare neuromuscular disorders
This study tests a personalized exercise program for people with rare neuromuscular diseases like FSHD, DM1, or CMT to see if it improves their physical function and quality of life.
BergenAges 18–70 - NCT06708572Recruiting
GCSF after Kasai surgery for biliary atresia
This study tests a medicine called GCSF (helps make white blood cells) in babies who have had Kasai surgery for biliary atresia. It aims to see if GCSF can improve liver function and overall outcomes.
Cairo, Menofia GovernorateAges 3 weeks–1 year - NCT06712095Recruiting
Video Capsule for Lynch Syndrome Screening
This study tests whether a video capsule (a pill-sized camera you swallow) can help find polyps in people with Lynch syndrome, without needing a colonoscopy. It may be an easier way to screen for colon cancer.
London, (Select)Ages 18 years+ - NCT06713993Recruiting
Esophageal Cancer Study: Using Noninvasive Tests to Guide Care
This study is looking for people newly diagnosed with esophageal cancer or cancer at the stomach-esophagus junction who are planning to have surgery. The goal is to see if certain noninvasive tests (like scans) can help predict how the disease will progress.
BudapestAges 18 years+ - NCT06716931Recruiting
Exercise study for myotonic dystrophy type 2
This study tests if a structured exercise program is safe and helpful for people with myotonic dystrophy type 2 (DM2). It aims to improve strength and daily function.
Boston, MassachusettsAges 18–70 - NCT06716814RecruitingPhase 3
Continuing efanesoctocog alfa after earlier trial
This study provides continued access to the clotting factor treatment efanesoctocog alfa for people who have already been getting it in a previous trial. It helps ensure you can keep using the medicine if it is working for you.
PlovdivAges 6 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 5,779 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.