Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: a Randomized, Placebo-controlled Pilot Trial
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• Age 18-65 years, any gender;
• Patients who have experienced their first symptomatic BSCM ICH within the six months before randomisation;
• Diagnosed with solitary BSCM through T2, GRE/T2\*, or SWI MR imaging;
• ICH within or around the BSCM confirmed by CT /MR;
• Capable of signing an informed consent form with the accompaniment and understanding of a guardian.
Locations
Other Locations
China
Huashan Hospital, Fudan University
ACTIVE_NOT_RECRUITING
Shanghai
Huashan Hospital, Fudan University
RECRUITING
Shanghai
Contact Information
Primary
Zongze Li, Doctor
lizongze666@aliyun.com
+8613121226581
Backup
Wei Zhu, Doctor
drzhuwei@fudan.edu.cn
+8613121226581
Time Frame
Start Date: 2024-01-05
Estimated Completion Date: 2026-12-31
Participants
Target number of participants: 75
Treatments
Experimental: High-dose sirolimus group
Participants will receive oral sirolimus with a target blood concentration of 9-15ng/ml continuously for 12 months.
Experimental: Low-dose sirolimus group
Participants will receive oral sirolimus with a target blood concentration of 3-7ng/ml continuously for 12 months
Placebo_comparator: Placebo control group
Participants will receive oral placebo(starch formulation) for 12 months.
Related Therapeutic Areas
Sponsors
Leads: Huashan Hospital