Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,863 recruiting studies in this area right now.
By phase
- Not applicable2,776
- Phase 2282
- Phase 1237
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease142
- Atopic Dermatitis129
- Congenital Heart Disease85
- Cystic Fibrosis78
- Down Syndrome55
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease39
- Hemophilia A39
- 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 Human Genome Research Institute (NHGRI)23
Where studies are running
- California339
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona77
- Texas72
- Minnesota67
- Pennsylvania66
- Ohio57
Recruiting studies3,863 total
- NCT07425769Recruiting
Adapting a program for adults with Down syndrome and caregivers
This study tests a program to help adults with Down syndrome and their caregivers become more active. It aims to improve health and well-being for both.
Kansas City, KansasAges Any age - NCT06096688Recruiting
Finding new ways to lower colorectal and endometrial cancer risk
This study looks for new ways to lower the risk of colorectal and endometrial cancers. It collects tissue and information from people who are getting certain procedures or treatments at Weill Cornell Medicine.
New York, New YorkAges 18 years+ - NCT04753970RecruitingPhase 1/Phase 2
Study uses retina changes to assess blood vessel health
This study looks at the back of your eyes (the retina) to see if it reflects blood vessel health in the brain. It may help doctors find easier ways to track conditions related to small blood vessel disease.
Jacksonville, FloridaAges 18 years+ - NCT07740915Enrolling by invitation
Pain relief after heart surgery in children
This study compares two types of pain blocks for children having heart surgery for the first time. The goal is to see which block provides better pain relief after surgery, which could help your child recover more comfortably.
Diyarbakır, DiyarbakırAges birth–18 years - NCT06164730RecruitingPhase 1
Study of VERVE-102 for high cholesterol or early heart disease
This trial tests a new medicine called VERVE-102 for people with a specific type of high cholesterol (HeFH) or early coronary artery disease. It aims to see if it can help lower cholesterol levels.
Dothan, AlabamaAges 18–70 - NCT07179640RecruitingPhase 1/Phase 2
Testing ALE1 for hypophosphatasia in healthy people and patients
This trial tests a new medicine called ALE1 for hypophosphatasia (HPP), a rare bone condition. It first checks safety in healthy volunteers, then tests if the medicine helps people with HPP.
WürzburgAges 18–50 - NCT06239272RecruitingPhase 1/Phase 2
Personalized treatment for NRSTS in children and young adults
This trial tests a personalized treatment plan for children and young adults with a type of soft tissue sarcoma (NRSTS). It uses a combination of surgery, targeted radiation, and two drugs (pazopanib and selinexor) to fight cancer while reducing side effects. The study is looking for people who have not been treated before for this sarcoma.
Atlanta, GeorgiaAges Up to 30 years - NCT07319091Recruiting
Cystinosis and Mitochondrial Energy Study
This study looks at how cystinosis affects the energy centers (mitochondria) in your cells. It may help doctors understand how cysteamine treatment works in patients with kidney cystinosis.
BronAges 2 years+ - NCT07585097Recruiting
Long-term Safety Study of Odevixibat for Alagille Syndrome
This study follows patients with Alagille syndrome who are taking odevixibat to see how safe the medicine is over time. Alagille syndrome is a rare liver disease that affects bile flow, and odevixibat is a treatment that may help reduce itching and improve liver function.
BronAges 6 months+ - NCT07623525RecruitingPhase 1/Phase 2
DIAG723 study for hereditary hemorrhagic telangiectasia
This study tests a new drug called DIAG723 in adults with hereditary hemorrhagic telangiectasia (HHT), a genetic condition that causes abnormal blood vessels and bleeding. Depending on which part of the study you qualify for, the drug may help reduce nosebleeds, improve anemia, or help with lung blood vessel problems caused by HHT.
Camperdown, New South WalesAges 18 years+ - NCT06850038Recruiting
Long-term study of odevixibat in Alagille syndrome
This study follows people with Alagille syndrome who are already taking or starting odevixibat (Bylvay). It looks at how well the medicine works and how safe it is over a long period of time.
Los Angeles, CaliforniaAges Any age - NCT05697861Enrolling by invitation
Follow-up study for people previously treated with NTLA-2001
This study checks how people are doing long-term after receiving NTLA-2001 in an Intellia clinical trial. If you previously took part and agree to be followed, this may help the researchers understand lasting safety and effects.
ParisAges 18 years+ - NCT06943469Recruiting
Study of special formula for babies with allergy risk
This study tests a special baby formula (partially broken-down protein with added prebiotics and probiotics) to see if it helps strengthen babies' skin barrier and reduce allergy risk. It's designed for newborns who have a family history of allergies.
BrugesAges 1 day–2 weeks - NCT07081711Recruiting
Testing new heart MRI technology in volunteers
This study is testing a new way to take pictures of the heart using MRI. It is looking for healthy volunteers to help make sure the technology works correctly.
Cleveland, OhioAges 18 years+ - NCT06325709RecruitingPhase 1/Phase 2
Gene editing trial for X-linked chronic granulomatous disease
This trial tests a new gene-editing treatment for men with a specific genetic mutation that causes X-linked chronic granulomatous disease (CGD). The goal is to fix the faulty gene in your own blood stem cells to help your immune system fight infections better.
Bethesda, MarylandAges 18–75 - NCT07701694Recruiting
Driving study for people with urea cycle disorders
This study evaluates how people with urea cycle disorders perform during a driving task. It may help researchers understand if the condition affects driving ability.
Memphis, TennesseeAges 16–40 - NCT07587684RecruitingPhase 1
Testing IN026 for Hard-to-Treat Gout
This study tests a new drug called IN026 for people with refractory gout—gout that doesn't respond well to current treatments and causes severe symptoms. The goal is to see if IN026 can lower uric acid levels and reduce gout attacks.
WuhanAges 18–75 - NCT06910813RecruitingPhase 1/Phase 2
Gene therapy trial for children with cystinosis
This trial tests a new gene therapy called DFT383 for children aged 2 to 5 with nephropathic cystinosis. It aims to see if the treatment is safe and can help improve kidney function.
Phoenix, ArizonaAges 2–5 - NCT06560463Recruiting
CGM and glucose test for cystic fibrosis related diabetes
This study tests if a continuous glucose monitor (CGM) along with a standard glucose drink test can better detect diabetes in people with cystic fibrosis. It is for adults who do not yet have diabetes and are generally healthy.
Milwaukee, WisconsinAges 18 years+ - NCT07037446Enrolling by invitation
Occlutech leak device for mitral or aortic paravalvular leaks
This registry tracks the safety and effectiveness of a device designed to close leaks around heart valves (mitral or aortic). It may be an option for people who have significant symptoms like heart failure or anemia from these leaks and are not good candidates for surgery.
KatowiceAges 18 years+ - NCT07615686Recruiting
Spinal Cord Stimulation to Help Children with Spina Bifida Walk
This trial tests a treatment called transcutaneous spinal cord stimulation (tSCS)—gentle electrical pulses applied to the skin over the spine—to help children with spina bifida walk better and manage bladder control. The study includes children ages 4–17 who can walk short distances with help or devices.
Pittsburgh, PennsylvaniaAges 4–17 - NCT04419584RecruitingPhase 2/Phase 3
Modified Qing-Ying decoction for children with eczema
This trial tests a traditional Chinese herbal formula (Modified Qing-Ying Decoction) for children ages 4 to 17 with moderate-to-severe eczema (atopic dermatitis). The study aims to see if this herbal treatment can improve skin dryness, redness, and itching, especially for those with dry, scaly patches.
Hong KongAges 4–17 - NCT04657068RecruitingPhase 1/Phase 2
Study of ART0380 for advanced solid tumors
This study tests a new drug called ART0380 for people with advanced solid tumors that have not responded to standard treatments. It may be especially helpful for those with certain DNA repair gene changes (like ATM or BRCA). The drug will be given alone or with chemotherapy (gemcitabine or irinotecan) depending on the part of the trial.
Birmingham, AlabamaAges 18 years+ - NCT07187973RecruitingPhase 1
Study of DISC-3405 for people with sickle cell disease
This trial tests an investigational drug called DISC-3405 in people with sickle cell disease (SCD) to see if it is safe and how it affects your body. It may be an option if you have certain SCD complications or lab results.
Birmingham, AlabamaAges 18 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,863 recruiting medical genetics studies from the U.S. registry right now, out of 5,730 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.