Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: a Single Centre, Randomised, Placebo-controlled Trial

2023-816

This study employs a single-center, prospective, randomized controlled, double-blind exploratory research design. To investigate whether Sirolimus can reduce the rebleeding rate of brainstem cavernous malformations within 24 months after the first symptomatic bleeding event.

2023-12-01

2025-12-31

2025-12-31

Not yet recruiting

Early phase I

60

Inclusion Criteria: 1. Age between 18 and 65 years, any gender. 2. Patients who experienced their first symptomatic bleeding caused by brainstem cavernous malformation within six months. 3. Diagnosed with brainstem cavernous malformation through SWI and MR T2 imaging. 4. Confirmed intracranial or perilesional bleeding by CT scan. 5. Capable of signing an informed consent form with the understanding and accompaniment of a guardian. Exclusion Criteria: 1. History of cancer. 2. Pregnancy or lactation. 3. Hypersensitivity to rapamycin or placebo. 4. Respiratory failure or severe bleeding requiring life support treatment. 5. Abnormal liver or kidney function (transaminases greater than 50, creatinine greater than 110), white blood cell/platelet abnormalities (white blood cell count below 3.5 or above 9.5 x 10\^9/L, platelet count below 100 or above 300). 6. History of previous immunosuppressive therapy. 7. History of bleeding more than 6 months ago. 8. History of surgical treatment for cavernous malformation. 9. History of radiation therapy for cerebral cavernous malformation. 10. History of previous statin medication treatment. 11. History of previous propranolol treatment. 12. Presence of intracranial cavernous malformation in a location other than the brainstem. 13. Patients with concurrent acute active infections (such as severe bacterial, viral, or fungal infections). 14. Uncontrolled diabetes. 15. Participation in other clinical trials.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'According to the study protocol, patients were randomly divided into a normal-dose group, low-dose group, and control group at a ratio of 1:1:1. Additionally, to minimize potential bias, this study employed a double-blind approach.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'TRIPLE', 'maskingDescription': 'A double-blind design means that throughout the entire study, neither the participating patients nor the investigators are aware of which treatment group the patients are assigned to, in order to minimize potential biases. The blinding level is double-blind, which means that both the patients in the treatment group and those in the control group, as well as the investigators, are unaware of the treatment the patients receive, ensuring objectivity and reliability of the study results.', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 60, 'type': 'ESTIMATED'}}

2023-11-22