Overgrowth syndrome

This study tests alpelisib, a drug that targets a specific gene mutation (PIK3CA), in people with lymphatic malformations—abnormal growths of lymph vessels. The trial includes children and adults and compares alpelisib to a placebo to see if it can shrink the growths and ease sym…

This study tests a new drug called CYH33 in people with rare conditions that cause abnormal tissue growth and blood vessel malformations (PROS and PRVM). The trial has two phases: phase I finds the safest dose, and phase II checks if the drug shrinks growths. About 141 adults and…

This study tests a daily oral drug called alpelisib in people aged 2 and older with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. The goal is to see if the drug can shrink overgrown tissue and improve symptoms. About 104 participants …

This study tests whether custom 3D-printed titanium implants can safely and effectively rebuild facial bones in 30 adults with defects from injury, cancer surgery, or birth conditions. Each implant is designed from CT scans to match the patient's unique anatomy. Researchers will …

This Phase 2 study tests an oral drug called RLY-2608 in adults and children with overgrowth conditions (like CLOVES or Klippel-Trenaunay syndrome) caused by PIK3CA gene mutations. The drug is designed to block the faulty protein driving abnormal tissue growth. The trial will enr…

This early-stage study is testing a new drug called BGB-B2033, alone or with other drugs, in people with advanced liver, stomach, or lung cancers. The goal is to see if it is safe and how well it works. About 392 participants will take part.

This phase 2 trial is testing two targeted drugs—alpelisib and mirdametinib—in 50 people with slow-flow or fast-flow vascular malformations that haven't responded to standard treatments. The goal is to see if these drugs can improve each person's most bothersome symptom over 48 w…

This program provides access to the drug alpelisib (BYL719) for patients with PIK3CA-related overgrowth spectrum or breast cancer who have no other treatment options and cannot join a clinical trial. It is not a study but a way for doctors to request the drug for individual patie…

This study tests a new drug called AZD9793 in people with advanced liver cancer that has spread. The drug is an antibody designed to help the body's immune cells find and attack cancer cells. The main goals are to check if the drug is safe and if it can shrink tumors. About 304 a…

This study tests a new treatment for adults with advanced liver cancer that cannot be removed by surgery or has spread. The treatment uses specially engineered immune cells (CAR-NK cells) from a healthy donor, designed to target two proteins found on liver cancer cells. Participa…

This early-stage study tests a new drug called AST-201 in about 70 adults with advanced solid tumors that have a protein called GPC3. The main goal is to check the drug's safety and find the right dose. Researchers will also measure how the drug moves through the body and look fo…

This phase 2 trial tests alpelisib, a drug originally developed for cancer, in 20 people aged 2 to 40 with MCAP syndrome—a rare condition causing an enlarged brain, blood vessel issues, and developmental delays. Participants receive either alpelisib or a placebo for 6 months, the…

This early-stage study tests a drug called codrituzumab in children and young adults (ages 1 to 21) whose solid tumors have come back or not responded to treatment and carry a specific protein called GPC3. The main goal is to find a safe dose and see how the body handles the drug…

This early-phase trial is testing a new drug called MT-303, alone or with two standard cancer drugs, in people with advanced liver cancer that has spread or cannot be removed. The main goals are to check safety, find the right dose, and see early signs of effectiveness. About 70 …

This study tests a new radioactive tracer called 68Ga-GPC3 that targets a protein found on liver cancer cells. About 40 adults with suspected or confirmed hepatocellular carcinoma will receive both this new tracer and a standard tracer (18F-FDG) for PET/CT scans. Researchers will…

This study offers expanded access to a PET scan using a radioactive tracer called 18F-DOPA to locate small, insulin-producing lesions in the pancreas. It is for patients with congenital hyperinsulinism, Beckwith-Wiedemann syndrome, or insulinoma who have low blood sugar. The goal…

This study is testing a new type of PET scan that uses special probes to highlight digestive system cancers, such as stomach, liver, colon, and pancreatic cancers. Researchers will enroll 400 adults to see how well this imaging method finds tumors and tracks their response to tre…

This study aims to understand the genetic and biological causes of Proteus syndrome, a rare condition causing abnormal growth of bones, skin, and other tissues. Researchers will follow up to 1,500 participants over time, using medical exams, imaging, and blood tests to track how …

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study aims to find the genetic causes of Ollier disease and Maffucci syndrome, rare disorders that cause cartilage tumors and increase cancer risk. Researchers will study 100 people aged 2 and older using blood tests, scans, and genetic sequencing. Participants will stay at …

This study is creating a registry of up to 1,000 people with rare lymphatic system disorders. Researchers will collect information from medical records and yearly surveys to understand how these diseases progress, what treatments are used, and how they affect quality of life. The…

This observational study will follow 2000 children and adults with imprinting disorders—rare genetic conditions like Silver-Russell and Prader-Willi syndromes. Researchers aim to describe the natural history of these diseases and identify common metabolic profiles, risks for obes…

This study is for adults with advanced liver cancer who are starting a common immunotherapy combination (Atezolizumab and Bevacizumab). Researchers want to see if levels of a protein called Glypican-3 in the blood can help predict how well the treatment works. About 240 participa…

This study is building a registry of up to 400 people with Ollier disease or Maffucci syndrome. Researchers will collect medical history, genetic data, imaging, and quality-of-life information to better understand how these conditions progress and how they are best managed. The g…