PIK3CA and CCM mutations fuel cavernomas through a cancer-like mechanism.
Aileen A Ren, Daniel A Snellings, Yourong S Su...
https://pubmed.ncbi.nlm.nih.gov/33910229Actively Recruiting
Phase 2
Age: 18Years - 65Years
All Genders
ID06091332
Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: a Randomized, Placebo-controlled Pilot Trial
Led by Huashan Hospital · Updated on 2024-12-20
75
Participants Needed
2
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating sirolimus, a drug approved by the FDA and used for certain malformations, to see if it is safe and tolerable in reducing brain hemorrhages in people with brainstem cavernous malformations (BSCMs). This pilot phase 2 clinical trial compares sirolimus to a placebo to inform a future larger study. The focus is on preventing intracerebral hemorrhage (ICH) during high-risk periods for rebleeding in adults aged 18 to 65. Participants are randomly assigned to one of three groups: high-dose sirolimus targeting blood levels of 9-15 ng/ml, low-dose sirolimus targeting 3-7 ng/ml, or a placebo made of starch. Treatments are taken orally and continued continuously for 12 months. The study is blinded so that neither participants nor researchers know the assigned group. During the 24-month follow-up, researchers will monitor safety and tolerability of sirolimus, track recurrent brain hemorrhages, assess brain scans using MRI, and evaluate participants' quality of life. Participants will undergo regular assessments including imaging and clinical evaluations to measure these outcomes. The study aims to gather important data on sirolimus treatment effects and safety over two years.
CONDITIONS
Brief Title
Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial
Who Can Participate
Age: 18Years - 65Years
All Genders
Eligibility Criteria
You may qualify if you...
- Age 18 to 65 years, any gender
- First symptomatic brainstem cavernous malformation intracerebral hemorrhage within 6 months before randomization
- Diagnosed with solitary brainstem cavernous malformation confirmed by T2, GRE/T2*, or SWI MRI
- Intracerebral hemorrhage within or around the lesion confirmed by CT or MRI
- Able to sign informed consent with guardian support and understanding
You will not qualify if you...
- History of cancer
- Pregnancy or lactation
- Allergy to sirolimus or starch
- Modified Rankin Scale score of 5, respiratory failure, or severe bleeding requiring life support
- Abnormal liver or kidney function or abnormal white blood cell or platelet counts
- Previous immunosuppressive therapy
- Prior surgery for cavernous malformation
- Prior cranial radiation therapy
- Familial or multiple cavernous malformations
- Current active severe infections
- Uncontrolled diabetes mellitus
- Participation in another clinical trial
- Unable or unwilling to undergo MRI
- Use of drugs affecting CYP3A4 enzymes (e.g., ketoconazole, voriconazole)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 12 months
Participants receive oral sirolimus or placebo continuously as assigned to manage brainstem cavernous malformations.
Regular visits for monitoring during treatment
Follow-up
Duration - 12 months
Participants are monitored for safety, tolerability, and efficacy outcomes after treatment.
Periodic visits up to 24 months from randomization
Trial Site Locations
Total: 2 locations
1
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, China, 200040
Active, Not Recruiting
2
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, China, 200040
Actively Recruiting
Loading map...
Research Team
Z
Zongze Li, Doctor
W
Wei Zhu, Doctor
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
Population-Based Prevalence of Cerebral Cavernous Malformations in Older Adults: Mayo Clinic Study of Aging.
Kelly D Flemming, Jonathan Graff-Radford, Jeremiah Aakre...
https://pubmed.ncbi.nlm.nih.gov/28492932Somatic variants of MAP3K3 are sufficient to cause cerebral and spinal cord cavernous malformations.
Jian Ren, Yazi Huang, Yeqing Ren...
https://pubmed.ncbi.nlm.nih.gov/36995941Metformin limits cerebral cavernous malformation development by targeting KLF4-mediated mitochondrial damage.
Cong Yan, Yongqing Ye, Nan Liu...
https://pubmed.ncbi.nlm.nih.gov/40570636