Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Ra
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.
| Condition(s) | Cavernous Malformations, Intracerebral Hemorrhage, Brainstem Stroke |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| 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. |
| Who can participate | Inclusion Criteria: 1. Age 18-65 years, any gender; 2. Patients who have experienced their first symptomatic BSCM ICH within the six months before randomisation; 3. Diagnosed with solitary BSCM through T2, GRE/T2\*, or SWI MR imaging; 4. ICH within or around the BSCM confirmed by CT /MR; 5. Capable of signing an informed consent form with the accompaniment and understanding of a guardian. Exclusion Criteria: 1. Cancer history; 2. Pregnancy or lactation; 3. Sirolimus/starch allergy; 4. Modified Rankin Scale (mRS) score 5, respiratory failure or currently severe bleeding requiring life support treatment; 5. Abnormal liver and/or kidney function (transaminase levels greater than 50, creatinine greater than 110), abnormal white blood cell/platelet counts (white blood cell count below 3.5 or ab |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Lead sponsor | Huashan Hospital |
| Locations | Shanghai, Shanghai Municipality, China; Shanghai, Shanghai Municipality, China |
| Start date | 2024-01-05 |
| NCT ID | NCT06091332 |
| Official listing | https://clinicaltrials.gov/study/NCT06091332 |