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
- NCT06838143Recruiting
Ilaris real-world study for periodic fevers and sJIA
This study watches how people with certain periodic fever syndromes or a severe type of childhood arthritis (sJIA) do on the medicine Ilaris during their regular doctor visits. It collects information to see how well the medicine works in everyday care.
SeoulAges 2–100 - NCT06839235RecruitingPhase 1/Phase 2
Testing ABO-101 for primary hyperoxaluria type 1
This trial tests a new treatment called ABO-101 for people with primary hyperoxaluria type 1, a rare disease that causes high oxalate levels in urine. The study aims to see if ABO-101 can lower oxalate and protect kidney function.
Rochester, MinnesotaAges 6–64 - NCT06839456RecruitingPhase 1/Phase 2
Stem cell addback to prevent infections after transplant
This trial tests whether adding back certain immune cells after a stem cell transplant can help prevent serious viral or fungal infections. It's for children and young adults up to age 25 who need a transplant for a blood cancer or other curable disease.
Philadelphia, PennsylvaniaAges 1 month–25 years - NCT06839469Recruiting
Walking study for rare muscle and nerve conditions
This study looks at how children and teens with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk, by having them do walking tests while wearing sensors. The goal is to find better ways to track how these diseases progress and how treatments work over time, without needing blood tests or scans.
Palo Alto, CaliforniaAges 5 years+ - NCT06840496RecruitingPhase 3
Study of oral NA-921 vs placebo for girls and women with Rett syndrome
This trial tests an oral medication called NA-921 in females with Rett syndrome to see if it works better than a placebo. It may be an option for those who have a confirmed MECP2 gene mutation and stable seizure patterns.
Phoenix, ArizonaAges 5–20 - NCT06840717Recruiting
Genetic links to type 2 asthma in kids
This study looks at how certain genes might be connected to type 2 asthma in children. It aims to find out which kids are more likely to have asthma so doctors can better treat it.
Guangzhou, GuangdongAges 3–14 - NCT06847165Recruiting
Trigeminal nerve stimulation for kids with prenatal alcohol exposure
This study tests a gentle, non-invasive nerve stimulation device to see if it can improve attention and behavior in children aged 8-12 who were exposed to alcohol before birth and have ADHD. If it helps, it could offer a new way to manage symptoms without medication.
Los Angeles, CaliforniaAges 8–12 - NCT06843252Recruiting
Home-based brain stimulation for Huntington's disease symptoms
This study tests whether a gentle brain stimulation device (tDCS) used at home can help improve thinking and behavior in people with Huntington's disease. A caregiver is needed to be present during treatment and help with questionnaires.
San Antonio, TexasAges 18–85 - NCT06844123Recruiting
Robot-assisted corneal transplant surgery study
This study tests a new robot-assisted surgical technique for doing a full-thickness corneal transplant. The goal is to see if the robot can help make the surgery more precise and safer.
ReimsAges 18–60 - NCT06844214RecruitingPhase 1/Phase 2
Gene therapy trial for myotonic dystrophy type 1
This trial tests a one-time gene therapy (SAR446268) for people with non-congenital myotonic dystrophy type 1. It aims to see if the treatment is safe and can improve muscle symptoms like stiffness and weakness.
Gainesville, FloridaAges 10–55 - NCT06844734Recruiting
Study of ITB treatment for hereditary spastic paraplegia
This trial tests if a treatment called ITB (intrathecal baclofen) can help reduce muscle stiffness in people with hereditary spastic paraplegia (HSP). Participants will receive the treatment and be followed over time to see how well it works.
Shanghai, Shanghai MunicipalityAges 14–70 - NCT06844877Recruiting
Italian NCL Registry for Batten disease
This trial is creating a registry (a secure database) for people with a confirmed genetic diagnosis of NCL (Batten disease). The goal is to collect information over time to help researchers understand the disease better and plan future treatments.
PisaAges Any age - NCT06845371Recruiting
MELODY registry follow-up study
This study is for people who have already joined the MELODY registry. It will simply continue to collect information about their condition over time to help researchers better understand how things change.
TübingenAges Any age - NCT06845644Recruiting
MRI scans to track nerve damage in neuropathy patients
This study uses MRI scans to look at nerve and muscle changes over time in people with inherited or acquired nerve conditions. It may help doctors better understand how these diseases progress.
MarseilleAges 18 years+ - NCT06847282Recruiting
Motor skills study for children with FSHD
This study looks at how children with FSHD move and function over time. It uses walking tests and optional MRI scans to understand muscle changes. Your child's participation could help researchers learn more about FSHD progression in kids.
Palo Alto, CaliforniaAges 5–17 - NCT06849037Recruiting
Preventive antibiotics for meconium-stained fluid at birth
This trial tests whether giving a single dose of the antibiotic Zinacef (cefuroxime) to women with meconium-stained amniotic fluid during labor can reduce infections. You may be eligible if you have a full-term, single baby and your water is stained with meconium.
NahariyaAges 18 years+ - NCT06849635Recruiting
Cera ASD closure device long-term follow-up study
This study follows up with patients who already received the Cera ASD occluder device to close a hole in the heart (atrial septal defect). It looks at how well the device is working over time and checks for any late problems.
MyszkówAges Any age - NCT06849908RecruitingPhase 2
Baricitinib for treating intestinal Behçet's disease
This trial tests baricitinib, an oral medication, for people with intestinal Behçet's disease that hasn't improved with standard treatments. It aims to see if baricitinib can reduce symptoms and help heal gut ulcers.
Beijing, Beijing MunicipalityAges 18–65 - NCT06850194Recruiting
Comparing two jaw surgeries for severe underbite
This trial compares two surgical techniques to fix a severe underbite caused by a small lower jaw. One method uses a special cut in the jawbone and a piece of hip bone to lengthen it, while the other uses a different jawbone cut to move the jaw forward. It will help find which approach works better for people with this condition.
Beni-seuf, EgyptAges 18 years+ - NCT06850311RecruitingPhase 2/Phase 3
Herbal ointment for mild to severe eczema
This trial tests whether a special herbal ointment can help treat atopic dermatitis (eczema). It is for adults with mild, moderate, or severe eczema covering less than 10% of their body surface. The ointment is applied directly to the skin, and participants will also use a gentle, fragrance-free moisturizer.
New Taipei CityAges 18–65 - NCT06851338Recruiting
Inspire therapy study for teens with Down syndrome and sleep apnea
This study tests a device called Inspire that stimulates the airway during sleep to treat obstructive sleep apnea in teenagers with Down syndrome who cannot use a CPAP machine or had a tonsillectomy that did not work well.
Phoenix, ArizonaAges 13–18 - NCT06852612Recruiting
Dietary strategies for glycogen storage disease type I
This trial tests different diet strategies to help adults with GSDI (a metabolic condition also known as von Gierke disease) maintain stable blood sugars and avoid dangerous drops. It may help you learn how to better manage your condition through diet.
BernAges 18 years+ - NCT06853158Recruiting
Music therapy study for quality of life in sickle cell disease
This study tests if music therapy can improve quality of life for people with sickle cell disease who have chronic pain. You would join a music therapy group and share your feedback to help researchers see if this approach helps.
Cleveland, OhioAges 14 years+ - NCT06853743RecruitingPhase 2
Study of nicotinamide riboside for Huntington's disease
This trial tests if a vitamin B3-like supplement, nicotinamide riboside, can help people with early to mid-stage Huntington's disease. You will be randomly assigned to take the supplement or a placebo to see if it slows the disease.
OsloAges 18–80
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.