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

• Status: Not yet recruiting
• Trial end date: 2025-12-31
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Huashan Hospital
• Treatments: Sirolimus

Criteria

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.