Adrenal cortex disorder

This early-phase trial tested a new therapeutic vaccine (EO2401) combined with the immunotherapy drug nivolumab in 70 people with advanced adrenocortical carcinoma or malignant pheochromocytoma/paraganglioma. The goal was to see if the combination is safe and can shrink tumors or…

This early-stage study tested a combination of two drugs, niraparib and dostarlimab, in children and teens (ages 6 months to 17 years) with solid tumors that had come back or were not responding to treatment. The goal was to find a safe dose and see if the drugs could shrink tumo…

This early-phase trial tested a radioactive drug designed to seek out and destroy cancer cells in people with advanced solid tumours that had stopped responding to other treatments. The drug targets a protein called IGF-1R found on many cancer cells. The study aimed to find a saf…

This study tested an experimental drug called tildacerfont in 67 children aged 2 to 17 with congenital adrenal hyperplasia (CAH), a genetic condition that disrupts hormone production. The goal was to see if adding this once-daily pill to standard steroid treatment could improve d…

This early-phase study tested a drug called abiraterone acetate (normally used for prostate cancer) in 4 children with congenital adrenal hyperplasia (CAH). The goal was to see if it could lower high male hormone levels and reduce the need for strong steroid medications that can …

This early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and e…

This study tested a daily tablet called Tildacerfont in 100 adults with classic congenital adrenal hyperplasia, a condition where the body can't make certain hormones properly. The goal was to see if the drug could safely reduce the high doses of steroids patients need to take. T…

This study followed 65 adult men with a rare inherited nerve disease called AMN (a form of spastic paraplegia) to understand how their symptoms change over time. Researchers collected data on walking ability and quality of life. The goal was to fill gaps in knowledge about the di…