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
- NCT07429240RecruitingPhase 1/Phase 2
Gene editing trial for Duchenne muscular dystrophy (ages 2-7)
This trial tests a new gene editing treatment called PBGENE-DMD for boys with Duchenne muscular dystrophy (DMD) whose genetic mutation falls within a specific range of the dystrophin gene (exons 45-55). The goal is to see if it is safe and can help improve muscle function.
Little Rock, ArkansasAges 2–7 - NCT07429188Recruiting
Study of upper-limb assistive device users
This study looks at how well upper-limb assistive devices (like robotic arms) work for people who use them and for their caregivers. You and your caregiver can help by sharing your experiences in video interviews.
PerpignanAges 18 years+ - NCT07431632Recruiting
Nerve freezing vs. epidural for pain after pectus excavatum repair
This study compares two ways to manage pain after pectus excavatum surgery: freezing the nerves during surgery (cryoanalgesia) versus an epidural block. The goal is to see which method provides better pain relief with fewer side effects.
São Paulo, São PauloAges 13 years+ - NCT07432867RecruitingPhase 1/Phase 2
Gene therapy for severe sickle cell disease in teens and adults
This trial tests a new gene therapy for sickle cell disease. It uses your own blood stem cells (collected from bone marrow) that are modified in a lab to help produce healthy red blood cells, then given back to you after a short chemotherapy. The goal is to reduce or stop severe complications like pain crises and lung problems.
Paris, Île-de-France RegionAges 12–35 - NCT07435168Recruiting
Chest pain and hidden cardiac amyloidosis study
This study looks for signs of a condition called cardiac amyloidosis in people who have chest pain but no blocked heart arteries. It aims to find hidden cases that might be missed with standard tests.
CopenhagenAges 65 years+ - NCT07436039Recruiting
Toothpaste for sensitivity and color improvement in MIH
This study tests a new toothpaste containing nano-hydroxyapatite, a substance that mimics natural enamel, to see if it can help reduce sensitivity and improve the color of teeth affected by Molar-Incisor Hypomineralization (MIH)—a condition where first molars and sometimes front teeth have white, yellow, or brown spots. It is for children aged 6 to 12 who have these spots on at least one permanent molar or baby molar.
JeddahAges 6–12 - NCT07436312Recruiting
Effect of ultra-processed foods on cancer risk in high-risk people
This trial looks at whether eating less ultra-processed foods can lower cancer risk in people who are already at high risk for cancer. It is part of a larger program called Interception that helps monitor and reduce cancer risk.
AvignonAges 18 years+ - NCT07436351Recruiting
ACT With CF Self-Help Toolkit
This trial tests an online self-help program based on acceptance and commitment therapy (ACT) for adults with cystic fibrosis who are feeling depressed or anxious. It aims to help you manage these feelings and improve your well-being.
Philadelphia, PennsylvaniaAges 18 years+ - NCT07437105RecruitingPhase 1
Healthy volunteer study of VX-272 safety
This study tests a new investigational medicine called VX-272 in healthy volunteers. Researchers want to check its safety and how it moves through the body, so you would not have the condition the medicine is being developed for.
Tempe, ArizonaAges 18–55 - NCT07437404RecruitingPhase 3
Testing SCT800 for previously untreated severe hemophilia A
This study tests a new medicine called SCT800 for boys with severe hemophilia A who have never been treated with factor VIII before. The goal is to see if it works safely to prevent bleeding.
Beijing, Beijing MunicipalityAges Up to 6 years - NCT07438691Recruiting
Comparing surgical methods for children with anorectal malformations
This study compares different surgical techniques for children with a specific type of anorectal malformation (perineal or vestibular fistula). It aims to see which surgery leads to better outcomes.
Florence, FlorenceAges birth–18 years - NCT07439679Recruiting
Anxiety Before Double Jaw Surgery and Your Recovery
This trial studies how anxiety before double jaw surgery affects your body during surgery and your recovery (pain, swelling, and ability to open your mouth). Researchers will use a simple anxiety questionnaire to see if feeling anxious changes your surgical experience.
IstanbulAges 18 years+ - NCT07439887RecruitingPhase 1/Phase 2
Study of an injection for Stargardt disease
This trial tests a single injection of a new medicine, RTx-021, into the eye for people with Stargardt disease. The goal is to see if it is safe and to find the right dose.
Bakersfield, CaliforniaAges 16 years+ - NCT07441395RecruitingPhase 2
Study of soquelitinib for moderate to severe eczema
This trial tests an experimental pill called soquelitinib for people with moderate to severe eczema (atopic dermatitis). It aims to see if the drug can reduce symptoms like itching and skin inflammation.
South San Francisco, CaliforniaAges 18 years+ - NCT07442851Recruiting
SickleFit exercise and nutrition study for sickle cell
This study looks at whether a personalized exercise and nutrition program can help people with sickle cell disease feel better and have fewer pain crises. It is for adults 35 and older with any type of sickle cell disease.
Durham, North CarolinaAges 35 years+ - NCT07444008Recruiting
BioAmicus Complete for infant digestive and skin issues
This study tests if BioAmicus Complete, a probiotic blend, can help babies with colic, reflux, diarrhea or constipation, and food allergy-related skin problems. Eligible babies are 1 to 4.5 months old, breastfed or formula-fed, and whose parents speak Russian.
Saint PetersburgAges 1 month–5 months - NCT07444580RecruitingPhase 1
RNA drug study for prion disease safety
This study tests an RNA-based drug designed to turn off the gene that causes prion disease. It aims to see if the drug is safe in people with early-stage symptoms.
Boston, MassachusettsAges 18 years+ - NCT07444723RecruitingPhase 2/Phase 3
New imaging to find parathyroid tumors before surgery
This study tests two advanced imaging techniques (PET/MR and PET/CT) to find overactive parathyroid glands or tumors that need surgery. Participants get a special scan before surgery to help surgeons locate the problem more accurately.
Bethesda, MarylandAges 18–100 - NCT07444801Recruiting
Tralokinumab for eczema with genital symptoms
This trial tests if tralokinumab, a drug already approved for moderate-to-severe eczema, helps people who also have eczema in the genital area. It aims to understand how well the drug works for this specific symptom.
Augsburg, MünchenAges 18 years+ - NCT07445919RecruitingPhase 2
Testing SM17 for moderate-to-severe eczema
This trial tests an investigational drug called SM17 for people with moderate-to-severe atopic dermatitis (eczema). It aims to see if SM17 can reduce itching and skin symptoms when topical treatments haven't worked well enough.
BeijingAges 18–70 - NCT07447648Recruiting
Evinacumab for plaque buildup in HoFH
This observational study looks at how adding evinacumab to standard cholesterol-lowering therapy affects plaque in the heart arteries of people with homozygous familial hypercholesterolemia (HoFH). It compares those who started evinacumab within the last 2 years to those on standard therapy alone, using CT scans taken before and after treatment.
ParisAges 12 years+ - NCT07448181Recruiting
Daily tracking of hereditary angioedema burden
This study uses a smartphone app to track how hereditary angioedema affects your daily life over 8 weeks. It aims to better understand the real-world burden of the condition and may help improve care.
MilanAges 18 years+ - NCT07448935Recruiting
Understanding a gene's role in AL amyloidosis and POEMS
This study looks at how a specific gene (IGLV1-44) affects two rare diseases: AL amyloidosis and POEMS syndrome. It involves a blood draw and possibly a bone marrow sample to learn more about how the gene works in these conditions.
PaviaAges 18–99 - NCT07450547RecruitingPhase 2
A trial of a new medicine for cystic fibrosis and pancreatic problems
This trial tests a new medicine (ANG003) for people with cystic fibrosis who have severe pancreatic problems. It looks at how safe and effective the medicine is, and whether it helps with digestion.
Long Beach, CaliforniaAges 12 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.