What this Trial Is About

Researchers are conducting a Phase 2 pilot trial to evaluate the safety and tolerability of sirolimus in treating patients with hemorrhagic brainstem cavernous malformations (BSCMs). The study aims to estimate how well sirolimus reduces the risk of intracerebral hemorrhage (ICH) during periods when rebleeding risk is high, comparing sirolimus treatment to a placebo. This trial will help design a larger future study to assess sirolimus for cavernous malformations management. Participants will be randomly assigned to receive either sirolimus, an FDA-approved mTORC1 inhibitor used in related vascular malformations, or a placebo made of starch tablets. The treatment period lasts 24 months, during which the safety and effects of sirolimus on preventing brainstem hemorrhages will be closely monitored. Throughout the study, participants will undergo medical evaluations including MRI scans to confirm diagnosis and monitor disease progression. Researchers will assess safety by tracking adverse effects and laboratory tests. The primary outcome is to explore the safety of sirolimus over 24 months. Participants must be between 18 and 65 years old and able to consent with guardian support if needed.

Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial