Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,762 recruiting studies in this area right now.
By phase
- Not applicable2,682
- Phase 2280
- Phase 1239
- Phase 3209
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease133
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy36
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)29
Leading sponsors
- Assistance Publique - Hôpitaux de Paris57
- Mayo Clinic39
- Boston Children's Hospital38
- National Institute of Allergy and Infectious Diseases (NIAID)38
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Children's Hospital Medical Center, Cincinnati31
- Hospices Civils de Lyon29
- Children's Hospital of Philadelphia23
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
Where studies are running
- California324
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York84
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT06413368RecruitingPhase 2/Phase 3
Maralixibat for cystic fibrosis constipation
This trial tests a medicine called maralixibat to see if it helps children with cystic fibrosis who have chronic constipation that doesn't improve with standard treatments. The goal is to improve bowel movements and quality of life.
Los Angeles, CaliforniaAges 1–18 - NCT05226624Recruiting
Improving care for babies born with opioid withdrawal
This program helps participating hospitals improve care for newborns who may go through opioid withdrawal (also called neonatal abstinence syndrome). It mainly aims to make mother-baby support and treatment plans work better after delivery.
Edmonton, AlbertaAges Any age - NCT03055247RecruitingPhase 2
Try ibuprofen plus growth factor and stem-cell mobilizer for X-CGD
This trial tests a combination of medicines to help your body release (“mobilize”) stem cells for future treatment in people with X-linked chronic granulomatous disease (X-CGD). If it works well and is safe for you, it may make stem-cell collection easier and more successful.
Rome, LazioAges 18–45 - NCT06389877RecruitingPhase 1/Phase 2
Gene therapy for adults with alpha-1 antitrypsin deficiency
This study tests a one-time gene therapy called BEAM-302 that aims to fix the faulty gene causing alpha-1 antitrypsin deficiency (AATD), a condition that damages the lungs and liver. The goal is to improve AAT levels and slow or stop disease progression.
Birmingham, AlabamaAges 18–75 - NCT06379789RecruitingPhase 1/Phase 2
Gene therapy for severe hemophilia B
This trial tests a new gene therapy (REGV131-LNP1265) that aims to let your body produce its own factor IX, reducing or stopping the need for regular infusions. It is for children and adults with severe hemophilia B who are already using factor IX replacement therapy.
Phoenix, ArizonaAges 2 years+ - NCT06397911RecruitingPhase 2
Testing FB825 for moderate to severe eczema
This trial tests a new injectable medicine called FB825 for people with moderate to severe eczema that hasn't improved enough with standard creams. The study aims to see if FB825 can reduce itching and skin symptoms safely.
Fountain Valley, CaliforniaAges 18–65 - NCT06395285RecruitingPhase 1
Testing DF-003 for ROSAH syndrome with eye inflammation
This trial tests an oral medication called DF-003 for people with ROSAH syndrome who have eye inflammation (uveitis). The goal is to see if the drug is safe and tolerable. It might help reduce eye inflammation and other symptoms.
Bethesda, MarylandAges 18–65 - NCT04280510Recruiting
Study of immune-related gut illnesses in adults
This study looks at adults with immune-related stomach and intestine problems to better understand what may cause them. If you qualify, you can share information and consent to help researchers learn how these conditions work.
CréteilAges 18 years+ - NCT04927156Recruiting
Check safety and use of Balt medical devices after treatment
This post-marketing study follows people after they receive Balt medical devices to make sure the devices are safe, work well, and are easy to use in real life. You may be considered if you can consent and your procedure matches their device-use rules.
AngersAges 2 years+ - NCT06396546Recruiting
Indian Children with Glycogen Storage Disease Registry
This study is creating a registry (a database) to better understand glycogen storage diseases in Indian children. If your child has a confirmed genetic diagnosis, joining helps doctors learn more about the condition and improve care.
Guindy, ChennaiAges Up to 18 years - NCT06396988Enrolling by invitation
Novamin for weak enamel in children's teeth
This study tests a special toothpaste ingredient (Novamin) that may help repair weak enamel on permanent back teeth and front teeth in children and teens. The treatment is applied by a dentist and may strengthen the teeth and reduce sensitivity.
Pavia, LombardyAges 7–14 - NCT06382636Recruiting
Helping pregnant people make decisions about genetic testing
This study is for pregnant people who were offered prenatal genetic testing. It aims to help them make informed decisions about these tests, with the goal of improving pregnancy outcomes.
Cleveland, OhioAges 18–50 - NCT06396078RecruitingPhase 4
Antibiotics for Early Water Breaking Before Delivery
This study tests whether giving antibiotics to pregnant people with early water breaking (PPROM) before delivery improves outcomes. It aims to prevent infections and protect both parent and baby while waiting for delivery.
Columbus, OhioAges 18 years+ - NCT06380530Recruiting
Computer-assisted surgical planning for limb deformity correction
This trial tests a new computer-assisted planning method to help surgeons better correct bone deformities in the arms or legs. It may lead to more precise surgeries and faster recovery.
Bologna, BOAges 2–40 - NCT06399367Enrolling by invitation
New imaging for leg swelling conditions
This study uses a special imaging machine (MSOT) to look at different types of swelling in the legs, such as lipedema and lymphedema. It aims to help doctors better understand and tell these conditions apart.
Göttingen, Lower SaxonyAges 18–80 - NCT05853107Recruiting
AuTNA I implant study for retinitis pigmentosa with minimal vision
This pilot study tests an implant called AuTNA I in adults with retinitis pigmentosa who currently have no or only very limited light perception. The goal is to see whether the implant can work safely and potentially improve vision in a carefully selected group of people.
Shanghai, Shanghai MunicipalityAges 18–70 - NCT06372717RecruitingPhase 1
Testing APL-4098 alone or combined for AML or MDS
This trial tests a new drug called APL-4098, given alone or with another medicine, for people with certain blood cancers (AML or MDS) that have come back or not responded to treatment. The goal is to see if it is safe and works well.
Clayton, VictoriaAges 18 years+ - NCT06363760Enrolling by invitation
Long-term follow-up after EDIT-301 gene therapy
This long-term follow-up study monitors people with sickle cell disease or transfusion-dependent beta-thalassemia who have already received the gene-editing therapy EDIT-301. It helps researchers understand how safe and effective the treatment is over time.
Oakland, CaliforniaAges 12–50 - NCT06366464RecruitingPhase 3
Study of a drug for sleepiness in Prader-Willi syndrome
This trial tests a medication called pitolisant to see if it helps reduce excessive daytime sleepiness in people with Prader-Willi syndrome. You or your loved one may be able to join if they have a confirmed diagnosis and a caregiver who can regularly help with study tasks.
Los Angeles, CaliforniaAges 6 years+ - NCT06360406Recruiting
Real-world study of Koselugo (selumetinib) in South Korea
This study looks at how patients with certain conditions are doing while taking Koselugo (selumetinib) in real life. It's a way to gather information about the treatment's effectiveness and safety outside of a controlled clinical trial.
BusanAges 3–99 - NCT06371417RecruitingPhase 1
Testing RAY121 for several immune diseases (RAINBOW trial)
This trial tests a new medicine called RAY121 (an antibody) for people with certain autoimmune diseases. It aims to see if RAY121 is safe and how the body handles it. If you have one of these conditions and meet the basic health requirements, you might be able to join.
Orange, CaliforniaAges 18–85 - NCT06055361RecruitingPhase 1
Studying a new injection for moderate to severe eczema
This trial tests an investigational drug called BxC-I17e, given as an injection under the skin, for people with moderate to severe atopic dermatitis (eczema) that hasn't improved enough with standard creams. The goal is to see if it is safe and works well.
North Little Rock, ArkansasAges 18 years+ - NCT06367192Recruiting
School Readiness Program for Preschoolers with Sickle Cell
This study tests a program to help preschoolers with sickle cell disease get ready for school. It looks at whether a special learning program can help with skills like paying attention and getting along with others.
Memphis, TennesseeAges 3.5 years–6.5 years - NCT06374238Recruiting
Peer support for young people with sickle cell pain
This study tests whether having a peer mentor who also has sickle cell disease can help young people manage chronic pain. You would use a mobile app to connect with your mentor and track your pain.
Mobile, AlabamaAges 16–39
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,762 recruiting medical genetics studies from the U.S. registry right now, out of 5,482 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.