A Phase 2 interventional study of Sirolimus and Starch flake in Cavernous Malformations, Intracerebral Hemorrhage and Brainstem Stroke, sponsored by Huashan Hospital. Recruiting at 2 sites in China. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2024-12-20.
Sponsored by Huashan Hospital · Phase 2, Interventional, and Treatment
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.
457 studies on the registry are indexed under Cerebral Hemorrhage; 172 are open to participants now.
This study's planned enrollment of 75 is below the median of 100 across 269 interventional studies indexed under Cerebral Hemorrhage.
Browse Cerebral Hemorrhage studies →Huashan Hospital is the lead sponsor of 247 studies on the registry; 104 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive oral sirolimus with a target blood concentration of 9-15ng/ml continuously for 12 months.
Drug: Sirolimus
Participants will receive oral sirolimus with a target blood concentration of 3-7ng/ml continuously for 12 months
Drug: Sirolimus
Participants will receive oral placebo(starch formulation) for 12 months.
Drug: Starch flake
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.
Also known as: Rapamycin
The placebo is composed of starch material and is formulated at 0.5 grams per tablet.
The primary outcome is to explore the safety of sirolimus in the management of BSCMs.
A serious adverse event is an SAE occurring during any study phase that fullfils one or more of the following criteria: i. Results in death, ii. Is life-threatening, iii. Requires inpatient hospitalization or prolongation of existing hospitalization, iv. Results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, v. Is an important medical event that may jeopardize the patient or may require medical intervention to prevent one of the outcomes listed above. SAE will be assessed at each follow-up visit (15 days, 2 months, 6 months, 12 months, 18 months, 24 months). During the trial, participants can promptly report potential SAEs to the research team via the telephone. We will establish continuous, real-time communication with patients via the telephone, facilitating round-the-clock reporting of potential endpoints or adverse reactions to the clinical trial investigators.
Time frame: 24 months
We will measure the tolerability of sirolimus in the management of BSCMs.
Based on prior clinical experience and reports from sirolimus trials, adverse events (AE) have included oral ulcers, upper respiratory tract infections, headaches, respiratory illnesses, stomatitis, seizures, and fever. Other commonly reported AEs are nausea, vomiting, fatigue, dizziness, diarrhea, and mild allergic reactions such as rash or itching. Minor infections, mild gastrointestinal disturbances, and fluctuations in blood pressure or heart rate have also been observed. Unanticipated Adverse Device Effect (UADE): Any serious adverse effect on health or safety or any life-threatening problem or death caused by or associated with sirolimus, if that effect, problem or death was not previously identified in nature, severity or degree of incidence in the investigational plan, or any other unanticipated serious problem associated with sirolimus that related to the rights, safety or welfare of subjects.
Time frame: 24 months
We will measure the occurrence of recurrent ICH from the BSCM within 24 months.
a. The primary efficacy outcome will be the occurrence of recurrent ICH from the BSCM within 24 months. ICH is defined as the presence of new focal neurological dysfunction or new headache symptoms, and confirmed by head CT/MR imaging examinations indicating new cerebral haemorrhage attributed to BSCM. If the primary efficacy outcome occurs, surgery will be considered for participants with a second ICH during follow-up. Radiosurgery or conservative treatment may also be considered for patients with a high risk of surgical intervention.
Time frame: 24 months
We will measure the efficacy outcomes in MR
A secondary efficacy outcome will be change in magnetic susceptibility using quantitative susceptibility mapping (QSM) MRI34 at 24 months after randomisation. Recent studies have described the feasibility, reliability, and validity of QSM as a biomarker for the effect of drugs on CCM.
Time frame: 24 months
We will measure several the quality of life
Quality of Life (mRS score and EuroQOL five dimensions questionnaire (EQ-5D) scale) within 24 months after enrollment
Time frame: 24 months
Not enrolling at this site.
Plan to share: No
Started Jan 2024 (actual). Primary completion is estimated for Dec 2025, 23 months after the start.
Of 24 phase 2 interventional studies under Cerebral Hemorrhage, half reached primary completion within 38 months of starting (the median), counting those still running; a quarter within 28 and three quarters within 62. This study's planned 23 months is below that.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Sirolimus