NCT06091332

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: Huashan Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The aim of this pilot phase trial is to assess the safety and tolerability, and estimate the efficacy of sirolimus in reducing the incidence of ICH during high-risk periods for rebleeding, compared to placebo. This pilot trial will inform the design of a future definitive clinical trial on sirolimus treatment for CCM.

Conditions

• Cavernous Malformations
• Intracerebral Hemorrhage
• Brainstem Stroke

Interventions

Type	Name	Description
DRUG	Sirolimus	Sirolimus is an mTORC1 inhibitor that has received approval from the U.S. Food and Drug Administration (FDA) and has recently been successfully used to treat lymphatic malformations and venous/lymphatic malformations associated with the same PIK3CA GOF mutations.
DRUG	Starch flake	The placebo is composed of starch material and is formulated at 0.5 grams per tablet.

Timeline

• Start date: 2024-01-05
• Primary completion: 2025-12-31
• Completion: 2026-12-31
• First posted: 2023-10-19
• Last updated: 2024-12-20

Locations

2 sites across 1 country: China