Tests sirolimus to prevent brainstem bleeding from cavernous malformations
Tests sirolimus against placebo in adults with brainstem cavernous malformations at risk of bleeding.
Key facts
- Who runs it
- Huashan Hospital
- Where
- Shanghai, ChinaSee the site
- Trial code
- Registry title
- Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial
- Status
- Recruiting
- Phase
- Phase 2
- People taking part
- 75 people, planned
- Started
- 5 Jan 2024
- Expected to end
- 31 Dec 2026
- Type of study
- Interventional, 3 groupsOne group gets a placebo (a dummy treatment), for comparison.
- Treatments tested
- Sirolimus
- Starch flake
- Record updated
- 20 December 2024
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults aged 18-65 who had a first bleed in the brain from a single cavernous malformation in the brainstem within the last six months. It tests sirolimus against placebo.
- Age
- 18 to 65 years
- Sex
- All sexes can join.
- Disease stage
- First symptomatic bleed (ICH) from a single brainstem cavernous malformation (BSCM) within the six months before randomisation. The bleed must be confirmed by CT or MR scan.
- Earlier treatment
- People cannot join if they had immunosuppressive treatment, surgery for cavernous malformation, or radiation to the head before.
- Biomarker or test result
- A single brainstem cavernous malformation seen on MR imaging (T2, GRE/T2*, or SWI).
Main reasons for not taking part
- History of cancer
- Pregnancy or breastfeeding
- Allergy to sirolimus or starch
- Familial cavernous malformation or multiple malformations
- Cannot or will not have an MRI
The registry criteria, word for word
Show all 19 criteriaShow less
Who can take part
- 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
Who cannot take part
- History of previous immunosuppressive therapy
- History of prior surgical intervention for CCM
- History of prior cranial radiation therapy
- Patients with concurrent acute active infections (e.g., severe bacterial, viral, or fungal infections)
- Co-administration of drugs affecting CYP3A4 enzymes (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin)
- Cancer history
- Sirolimus/starch allergy
- Modified Rankin Scale (mRS) score 5, respiratory failure or currently severe bleeding requiring life support treatment
- Uncontrolled diabetes mellitus
- 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 above 9.5 x 109/L or exceeding normal values, platelet count below 100 or above 300)
- Pregnancy or lactation
- Familial CCM or people with multiple CCM
- Currently participating in another clinical trial
- Patient unwilling/unable to undergo MRI
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Where in China
- Huashan Hospital, Fudan UniversityShanghai, ChinaContact: Zongze Li, DoctorNo longer taking part
About the trial
What the trial wants to find out
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.
The 3 groups in the trial
- High-dose sirolimus groupTreatment being tested
Participants will receive oral sirolimus with a target blood concentration of 9-15ng/ml continuously for 12 months.
Treatment: Sirolimus
- Low-dose sirolimus groupTreatment being tested
Participants will receive oral sirolimus with a target blood concentration of 3-7ng/ml continuously for 12 months
Treatment: Sirolimus
- Placebo control groupPlacebo, for comparison
Participants will receive oral placebo(starch formulation) for 12 months.
Treatment: Starch flake
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: a Randomized, Placebo-controlled Pilot Trial
- Plain-language title
- Tests sirolimus to prevent brainstem bleeding from cavernous malformationsGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT06091332, who can take part
- Record updated
- 20 December 2024