Congenital limb malformation

This study tests a new drug called BNT326, given alone or with other cancer immunotherapies, in about 980 adults with advanced solid tumors that have spread or come back after prior treatment. The goal is to find the best dose and see if it is safe and effective. It is an early-s…

This study tests two experimental drugs, BNT326 and BNT327, in people with advanced non-small cell lung cancer that has spread or come back. The trial has three parts to find the best dose and check safety, and will compare the combination to standard treatments like pembrolizuma…

This study compares two common surgeries for correcting clubfoot in children aged 2-5 who have not been treated before. The goal is to see which method better improves ankle movement and has fewer complications like bleeding or infection. About 44 children will take part, and res…

This trial compares two surgical tools—a standard surgical blade and a special ophthalmic knife—for cutting the Achilles tendon in children aged 2 to 5 with clubfoot. The goal is to see which tool improves ankle movement more and causes fewer problems like bleeding or infection. …

Club foot is a common birth defect where a baby's foot is twisted out of shape. The standard treatment uses weekly plaster casts to gradually correct the foot. This study tests whether changing the cast twice a week instead of once works just as well or better. About 144 babies u…

This early-stage trial is testing a new cancer vaccine called BNT116, alone or with other drugs, in people with advanced non-small cell lung cancer. The main goal is to find a safe dose and check for side effects. About 320 participants will be enrolled across several groups, inc…

This study tests a 16-week inclusive exercise program called PACE for adults with intellectual disability. Participants will attend fitness classes, meet with coaches, and use a web dashboard to set goals. The trial includes 376 people and will measure daily steps and moderate-to…

This study tests a new drug called OM-RCA-01, an antibody that blocks a protein called FGFR1 found on some cancer cells. About 58 adults with metastatic kidney, lung, prostate, breast, or head and neck cancers that express FGFR1 will receive the drug by IV every two weeks. The go…

This study tests a new breast reconstruction method for women who have had breast cancer or have Poland syndrome. The approach uses a special tissue expander to stretch the skin, followed by fat grafting from the patient's own body, and finally a permanent breast implant. The goa…

This study tests an experimental drug called E7090 in people with advanced or returning solid tumors that have a specific change in the FGFR gene. About 75 participants will receive the drug to see if it can shrink tumors and control the disease. The goal is to offer a new option…

This trial tests a wearable robot called ENMS-BSF that combines soft muscles, electrical stimulation, and balance feedback to correct foot drop and foot inversion after a stroke. Sixty people who had a stroke at least six months ago and have mild-to-moderate walking problems will…

This pilot study tests whether a custom splint called a Relative Motion Orthosis (RMO) can help people regain finger motion after an injury that causes stiffness. Sixty-four adults with a stiff middle finger joint will be randomly assigned to receive either standard therapy (stre…

This study looks at whether adding a foot abduction brace to the French method (a stretching and taping technique) helps babies with clubfoot more than the French method alone. Researchers will enroll 18 infants under 12 months old who have not had prior treatment. They will meas…

This study tests if a less invasive freezing technique (cryoneurotomy) works as well as surgery to reduce foot stiffness in people who had a stroke. About 114 adults with chronic foot spasticity will be randomly assigned to one of the two treatments. The goal is to see if the fre…

This study tests whether applying Mastisol, a liquid adhesive, during clubfoot casting helps babies with clubfeet get better faster and with fewer problems. Researchers will compare outcomes like how many weeks of casting are needed and how often complications like sores or swell…

This study tests whether a custom 3D-printed mouth splint can help burn patients who have developed microstomia (a tight, small mouth opening). Ten participants will use the splint and be measured for changes in mouth size and function using a special survey. The goal is to ease …

This study tests a behavioral therapy for children with Cornelia de Lange syndrome (CdLS) who have problem behaviors like aggression or self-injury. Researchers will identify what triggers these behaviors and then train parents to use a tailored treatment. The goal is to reduce p…

This study looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This pilot study looks at how botulinum toxin (Botox) injections affect the muscles, tendons, and nerves in the feet of people who have had a stroke and developed a tight, twisted foot (equinovarus). Researchers will use special ultrasound and movement tests to track these change…

This study looks at DNA methylation patterns (chemical tags on DNA) in fetuses with rare genetic diseases. Researchers will analyze DNA from amniotic fluid and tissue samples to create reference signatures. The goal is to improve diagnosis of these conditions before birth. The st…

This study aims to find genes that cause eye misalignment (strabismus) and related conditions involving the cranial nerves and brainstem. Researchers will analyze DNA from up to 20,000 participants with these disorders. The goal is to better understand the genetic causes, which c…

This study is building a global database of 1000 people with homozygous familial hypercholesterolemia (HoFH), a rare inherited condition causing extremely high cholesterol from birth. Researchers are gathering de-identified medical data from clinics around the world to better und…

This study looks at how using a 3D-printed prosthetic hand changes brain activity in children born with missing fingers, hands, or arms. Researchers will use a brain imaging technique called fNIRS to measure brain responses while children perform tasks with the prosthesis. The go…

This study is testing whether using rapid genetic sequencing can help doctors diagnose and treat birth defects in newborns in intensive care. Researchers will enroll 2,000 babies and compare death rates, disability rates, and genetic findings. The goal is to see if personalized t…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…