Clinical trials
Congenital Disorders clinical trials
Below are recruiting congenital disorders clinical trials, each written for real people, not researchers. We’re tracking 56 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT04199000Recruiting
Study for congenital glycosylation disorders and NGLY1 deficiency
This trial is for people who have a confirmed genetic condition related to how the body builds certain sugar-like structures (CDG) or NGLY1 deficiency. It aims to better understand these conditions through clinical and basic science research, which may help improve future care.
San Diego, CaliforniaAges Any age - NCT02089789Recruiting
Study of inherited glycosylation disorders for diagnosis
This study helps doctors diagnose congenital (from birth) glycosylation disorders, which are inherited conditions that affect how the body builds certain sugar-related proteins. You (or your child) may have clinic visits, blood or other samples, and possibly genetic testing—especially if there are family members with a known or suspected condition.
Bethesda, MarylandAges 1 month–80 years - NCT06891443RecruitingPhase 3
Study of Sepofarsen for LCA type 10
This study tests an investigational RNA therapy called sepofarsen for people with Leber Congenital Amaurosis (LCA) type 10, a rare inherited eye disease that causes severe vision loss from birth. The goal is to see if it can slow or improve vision loss.
San Francisco, CaliforniaAges 6 years+ - NCT07572825RecruitingPhase 1
Safety study of NMN supplement for DHDDS-CDG
This trial tests whether a supplement called NMN is safe and well-tolerated in children and adults with a rare genetic condition called DHDDS-CDG. NMN may help support cellular energy and protein production, which are affected by this condition.
New York, New YorkAges 4 years+ - NCT03016806Recruiting
Umbilical cord blood transplant from unrelated donors
This trial studies transplanting healthy blood-forming cells from an umbilical cord blood donor who is not related to you. It may help people with certain blood cancers, immune disorders, or other serious diseases when a standard transplant plan is needed.
Rochester, New YorkAges 2 months–75 years - NCT06818760Recruiting
Remote monitoring for pregnant women with heart conditions
This study tests if a wrist wearable can safely monitor pregnant women with congenital heart disease. It aims to improve care during pregnancy, delivery, and after birth by tracking health data remotely.
Cleveland, OhioAges 18 years+ - NCT05658965Recruiting
EEG monitoring after early heart surgery in infants
This study looks at whether early brain-wave testing (EEG) around the time of heart surgery in very young babies can predict later learning and development issues. It may help doctors identify children who need extra support sooner.
Lille, Hauts-de-FranceAges 1 day–1 year - NCT06442592Recruiting
Understanding development in kids with heart defects
This study looks at how children with complex congenital heart defects develop, especially their brain and learning skills. It will help doctors provide better support for these children.
Brest, Brittany RegionAges 3–11 - NCT06690151Recruiting
Babies with heart defects and development support
This study looks at how a serious heart condition before birth may affect a baby's brain development. It follows babies and their parents to better understand and support these children as they grow.
Nantes, Loire AtlantiqueAges Any age - NCT01403402Recruiting
Study of congenital muscle disease patients and their families
This trial collects health information from patients with certain inherited (genetic) muscle diseases and from their families. It may help researchers better understand the disease and improve care by learning what symptoms and treatments patients experience over time.
Lakewood, CaliforniaAges Any age - NCT05749419Recruiting
Vaccines for children with disabilities and long-term conditions
This study looks at how vaccines work in children who have disabilities, long-term (chronic) illnesses, or conditions they were born with. It may help improve vaccine care for kids who need extra support.
JerusalemAges Any age - NCT07477197Recruiting
Heart Institute Biobank for Adult Congenital Heart Disease
This study collects blood and tissue samples and health information from adults with or at risk for congenital heart disease or related heart conditions. It aims to help researchers better understand these conditions and improve future treatments.
Cincinnati, OhioAges 16 years+ - NCT04405700Recruiting
Tracking birth outcomes and birth defects after delivery
This study follows pregnant people and their babies to better measure adverse outcomes and possible congenital (from birth) conditions. It may help improve how birth risks and birth defects are identified and recorded at the clinic.
EldoretAges Any age - NCT06584877Enrolling by invitation
Understanding how childhood tumors and birth conditions start
This study looks at leftover tissue samples from children who have had tumors or birth conditions. Researchers want to learn more about how these conditions develop, and you may help if you or a relative fits the criteria.
CambridgeAges Any age - NCT06828822Recruiting
Study of children with congenital nevi over time
This study follows babies and toddlers with medium to large birthmarks to understand how they change over time and plan the best care. It will help doctors give better advice to families.
Nice, Alpes-MaritimesAges birth–2 years - NCT03059420Recruiting
Genetic study of strabismus and certain birth eye-movement disorders
This study looks for genetic (inherited DNA) causes of strabismus (eyes that don’t line up) and certain birth conditions that affect eye movement and brainstem/cranial nerves. Your participation may help researchers better understand these conditions and guide future testing and care.
Boston, MassachusettsAges 1 day+ - NCT04661059Recruiting
Study of how a fetus develops late in pregnancy
This study looks at how a baby’s organs develop in late pregnancy, using situations where someone is already being evaluated for a possible fetal abnormality and chooses to end the pregnancy. The goal is to better understand late-stage fetal development and related causes of abnormalities.
LondonAges 18 years+ - NCT05673499Recruiting
Monitoring oxygen and brain signals during major newborn surgery
This study checks how well newborn babies’ brains are getting oxygen and how brain activity changes while they have certain major surgeries. It may help doctors better monitor brain safety during surgery in newborns with specific conditions.
New York, New YorkAges Up to 1.1 years - NCT06970158Recruiting
Smart scan for babies with one-sided cleft lip and palate
This study tests using a smart camera (artificial intelligence) to help plan surgery for newborns with a single-sided cleft lip and palate. Your baby will have a quick and painless 3D scan of their mouth to guide their doctors, and you'll be closely supported throughout.
ChennaiAges 1 day–6 months - NCT00001403Recruiting
Understanding Proteus Syndrome and Related Growth Disorders
This study investigates Proteus syndrome, a rare condition that causes abnormal overgrowth of body parts, and related disorders. Researchers want to understand how these conditions develop, progress over time, and affect people of different ages—including how they may change into adulthood.
Bethesda, MarylandAges 1 month–99 years - NCT06190210Recruiting
Brain blood flow study in infants with heart defects
This study measures how well blood flows to the brain in infants with serious heart defects. The results may help doctors understand and protect brain development before and after heart surgery.
Leuven, Vlaams-BrabantAges Up to 6 months - NCT07092436Recruiting
Testing a new device for arm rehab in children
This trial tests a new device called PhiCube that helps children with movement problems improve arm and hand function through fun, interactive activities. It may be an option for children with conditions like cerebral palsy or coordination difficulties.
Bosisio Parini, LCAges 4–18 - NCT06431269Recruiting
Screening for developmental disorders in children with heart disease
This study is looking for children aged 1 to 5 years who have congenital heart disease and had heart surgery or a procedure in their first year. A special nurse will check for early signs of developmental delays, to see if this kind of screening is helpful for these children.
MontpellierAges 1–5 - NCT07409857Recruiting
Dengue infection in congenital bleeding disorders registry
This study creates a registry to track how dengue infection affects people with congenital bleeding disorders, such as hemophilia. It aims to learn more about managing dengue in this group.
BangkokAges Any age
Hear when a new Congenital Disorders trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Common questions
- Are there clinical trials for congenital disorders?
- Yes. Clin2 currently lists 56 recruiting congenital disorders studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a congenital disorders trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a congenital disorders trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.