Pilot Study of Sirolimus to Treat Hemorrhagic Brainstem Cavernous Malformations Randomized, Placebo-Controlled Phase 2 Trial in Adults Aged 18 to 65

What this Trial Is About

Researchers are evaluating sirolimus, a drug approved by the FDA and used for certain malformations, to see if it is safe and tolerable in reducing brain hemorrhages in people with brainstem cavernous malformations BSCMs. This pilot phase 2 clinical trial compares sirolimus to a placebo to inform a future larger study. The focus is on preventing intracerebral hemorrhage ICH during high-risk periods for rebleeding in adults aged 18 to 65. Participants are randomly assigned to one of three groups high-dose sirolimus targeting blood levels of 9-15 ngml, low-dose sirolimus targeting 3-7 ngml, or a placebo made of starch. Treatments are taken orally and continued continuously for 12 months. The study is blinded so that neither participants nor researchers know the assigned group. During the 24-month follow-up, researchers will monitor safety and tolerability of sirolimus, track recurrent brain hemorrhages, assess brain scans using MRI, and evaluate participants quality of life. Participants will undergo regular assessments including imaging and clinical evaluations to measure these outcomes. The study aims to gather important data on sirolimus treatment effects and safety over two years.

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