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
- NCT07760194Recruiting
Fetal heart check in late pregnancy
This study monitors the baby's heart in the womb during late pregnancy to make sure it is developing well. It may help find any concerns early.
AsyutAges Any age - NCT07560618RecruitingPhase 4
Testing a new cream for itch relief in atopic dermatitis
This study tests whether roflumilast cream (a new anti-inflammatory medication applied to the skin) can reduce the intense itching caused by atopic dermatitis (eczema). You'll track your itching daily using a smartphone app and visit the clinic to see if the cream helps over time.
Sacramento, CaliforniaAges 12 years+ - NCT06921213RecruitingPhase 2
Oral antibiotics for newborns exposed to syphilis
This study tests whether giving oral antibiotics at home is as safe and effective as the usual injections for newborns exposed to syphilis during pregnancy. It is for babies who appear healthy at birth.
JakartaAges birth–1 week - NCT07265258RecruitingPhase 4
Teprotumumab for active thyroid eye disease
This study tests a drug called teprotumumab for people with active thyroid eye disease. It is designed to help reduce eye bulging and other symptoms, and may be an option if you have not had strong treatments before.
Shanghai, Shanghai MunicipalityAges 18–80 - NCT05016908RecruitingTeam says not enrolling
Testing blood and other fluid markers in Duchenne or Becker
This study looks for signals in blood and other body fluids that may help track Duchenne or Becker muscular dystrophy. It may be helpful for patients by improving how future monitoring tests are developed.
Boston, MassachusettsAges 5 years+ - NCT07420907Recruiting
Tracking heart health over time
This study uses a wearable patch to monitor heart-related signs in people with heart conditions. It aims to learn more about how these conditions progress, which could help improve future care.
Chicago, IllinoisAges 18 years+ - NCT06542120Recruiting
Child health body sounds study
This study looks at how well a computer system can check children's health by listening to body sounds, like those from the heart, lungs, or belly. It is testing an AI system to see if it works well as an extra check alongside regular medical care.
Wuhan, HubeiAges Up to 18 years - NCT07154550Recruiting
Comparing Mastisol bandage for clubfoot casting
This study compares two types of sticky bandage (Mastisol vs. standard) used during Ponseti casting for babies with clubfoot in both feet. It aims to see which one works better for keeping the casts on and preventing skin problems.
Madison, WisconsinAges birth–3 months - NCT06594393RecruitingPhase 2
Testing TCP-25 Gel for Epidermolysis Bullosa
This trial tests a gel called TCP-25 on wounds caused by a specific type of epidermolysis bullosa (DEB or JEB). It aims to see if the gel can help heal wounds and improve comfort.
Cincinnati, OhioAges 4 years+ - NCT06789913RecruitingPhase 2
Testing RLY-2608 for growth disorders linked to PIK3CA mutations
This trial tests a new medicine, RLY-2608, that targets the specific genetic change (PIK3CA mutation) causing overgrowth conditions. It aims to see if the drug can slow or stop the growth of affected tissues.
Phoenix, ArizonaAges 2 years+ - NCT06725550Recruiting
Acupuncture for pain in sickle cell disease
This trial tests whether adding acupuncture to standard pain treatments can help manage pain in people with sickle cell disease. It involves brain scans, questionnaires, and acupuncture sessions to see if this approach reduces pain and improves quality of life.
Cincinnati, OhioAges 14–80 - NCT07159841RecruitingPhase 2/Phase 3
Study of a new drug for children with classic CAH
This study tests a new medication for children with classic congenital adrenal hyperplasia (CAH). It aims to see if the drug helps better control hormone levels and reduce the need for high doses of current steroid medicines.
San Francisco, CaliforniaAges 1–17 - NCT07144163RecruitingPhase 3
Atumelnant for adults with classic CAH
This study tests a new drug, atumelnant, for adults with classic congenital adrenal hyperplasia (CAH) caused by 21-OHD. It aims to see if it can help control hormone levels while allowing lower doses of current steroid medications.
Los Angeles, CaliforniaAges 18–74 - NCT07713758RecruitingPhase 1
How the body handles repinatrabit with liver or kidney issues
This trial checks how repinatrabit is processed in people with moderate liver problems or severe kidney problems compared to healthy adults. It helps doctors understand if the dose should be adjusted for these conditions.
Miami, FloridaAges 18–65 - NCT07593391RecruitingPhase 3
NNZ-2591 Treatment for Children with Phelan-McDermid Syndrome
This study tests whether a medication called NNZ-2591 can help children with Phelan-McDermid syndrome, a rare genetic condition that affects brain development and learning. Researchers want to see if this drug can improve how children with this condition develop and learn.
Glendale, CaliforniaAges 3–12 - NCT07754968Recruiting
Probiotic for formula-fed babies with colic
This study tests whether adding a probiotic called Bifidobacterium bifidum to formula can help reduce colic symptoms in babies who are not breastfed. It may also help if the baby has eczema (atopic dermatitis).
Roma, RMAges 1 week–5 months - NCT05777993Enrolling by invitationPhase 4
Continued access to mitapivat for prior study participants
This study provides continued access to the drug mitapivat for people who have already been in a mitapativ study and are benefiting from it.
Anderlecht, Brussels CapitalAges 18 years+ - NCT07446400RecruitingPhase 1
Drug interaction study for repinatrabit
This trial tests how repinatrabit interacts with common medications like birth control, metformin, and others. It helps doctors understand safe dosing and potential side effects.
Melbourne, VictoriaAges 18–55 - NCT07587021RecruitingPhase 2
YOLT-203 treatment study for primary hyperoxaluria type 1
This study tests a new drug called YOLT-203 for people with primary hyperoxaluria type 1 (PH1), a rare genetic condition where the body produces too much oxalate, which can damage the kidneys. The trial aims to see if this drug can help reduce oxalate levels and prevent kidney damage.
Birmingham, AlabamaAges 6 years+ - NCT05535361Recruiting
Testing a bowel device for short bowel syndrome patients
This trial studies whether the Eclipse XL1 system is safe and may help people with short bowel syndrome, in both children and adults. It will check how well the approach works and watch for side effects over the study period.
Palo Alto, CaliforniaAges 3 months–65 years - NCT06153459Recruiting
Delayed cord clamping for babies with heart disease
This study looks at how long to wait before clamping the umbilical cord in babies born with a heart condition. It helps doctors understand the best timing for these newborns.
Birmingham, AlabamaAges 9 months–10 months - NCT06908226Enrolling by invitationPhase 3
LP352 for children and adults with DEE
This trial tests a medication called LP352 for children and adults with certain types of epilepsy (Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathies). It's for people who already completed a previous study of LP352 and may benefit from continued treatment.
Little Rock, ArkansasAges 2–66 - NCT07037901RecruitingPhase 2
Testing IMG-007 for moderate-to-severe eczema
This study tests a new medicine called IMG-007 for adults with moderate-to-severe eczema (atopic dermatitis) that hasn't gotten better with standard creams. The goal is to see if it safely reduces itching and skin symptoms.
Lancaster, CaliforniaAges 18–74 - NCT06862583Recruiting
Study of acute type A aortic dissection treatments
This trial is studying treatments for people with a sudden tear in the main artery from the heart (type A aortic dissection) or a similar condition. It aims to improve outcomes after this emergency.
StockholmAges 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.