Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial

Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: a Randomized, Placebo-controlled Pilot Trial

Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial (NCT06091332) is a Phase 2 interventional studying Cavernous Malformations and Intracerebral Hemorrhage, sponsored by Huashan Hospital. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Cavernous Malformations and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 75 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Cavernous Malformations subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Age 18-65 years, any gender; 2. Patients who have experienced their first symptomatic BSCM ICH within the six months before randomisation; 3. Diagnosed with solitary BSCM through T2, GRE/T2\*, or SWI MR imaging; 4. ICH within or around the BSCM confirmed by CT /MR; 5. Capable of signing an willing to sign a consent form form with the accompaniment and understanding of a guardian. Who Should NOT Join This Trial: 1. Cancer history; 2. Pregnancy or lactation; 3. Sirolimus/starch allergy; 4. Modified Rankin Scale (mRS) score 5, respiratory failure or currently severe bleeding requiring life support treatment; 5. 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); 6. History of previous immunosuppressive therapy; 7. History of prior surgical intervention for CCM ; 8. History of prior cranial radiation therapy ; 9. Familial CCM or people with multiple CCM; 10. Patients with concurrent acute active infections (e.g., severe bacterial, viral, or fungal infections); 11. Uncontrolled diabetes mellitus; 12. Currently participating in another clinical trial; 13. Patient unwilling/unable to undergo MRI. 14. Co-administration of drugs affecting CYP3A4 enzymes (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin). Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: 1. Age 18-65 years, any gender; 2. Patients who have experienced their first symptomatic BSCM ICH within the six months before randomisation; 3. Diagnosed with solitary BSCM through T2, GRE/T2\*, or SWI MR imaging; 4. ICH within or around the BSCM confirmed by CT /MR; 5. Capable of signing an informed consent form with the accompaniment and understanding of a guardian. Exclusion Criteria: 1. Cancer history; 2. Pregnancy or lactation; 3. Sirolimus/starch allergy; 4. Modified Rankin Scale (mRS) score 5, respiratory failure or currently severe bleeding requiring life support treatment; 5. 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); 6. History of previous immunosuppressive therapy; 7. History of prior surgical intervention for CCM ; 8. History of prior cranial radiation therapy ; 9. Familial CCM or people with multiple CCM; 10. Patients with concurrent acute active infections (e.g., severe bacterial, viral, or fungal infections); 11. Uncontrolled diabetes mellitus; 12. Currently participating in another clinical trial; 13. Patient unwilling/unable to undergo MRI. 14. Co-administration of drugs affecting CYP3A4 enzymes (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin).

Treatments Being Tested

DRUG

Sirolimus

Sirolimus is an mTORC1 inhibitor that has received approval from the U.S. Food and Drug Administration (FDA) and has recently been successfully used to treat lymphatic malformations and venous/lymphatic malformations associated with the same PIK3CA GOF mutations.

DRUG

Starch flake

The placebo is composed of starch material and is formulated at 0.5 grams per tablet.

Locations (2)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Huashan Hospital, Fudan University

Shanghai, Shanghai Municipality, China

Huashan Hospital, Fudan University

Shanghai, Shanghai Municipality, China

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06091332), the sponsor (Huashan Hospital), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06091332 clinical trial studying?

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 full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06091332?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06091332?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06091332. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06091332. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.