This study is a single-arm exploratory trial conducted by Xuanwu Hospital, Capital Medical University, aiming to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations.
This study is a single-arm exploratory trial designed to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations. A total of 10 participants aged 18 to 65 years with a confirmed diagnosis of vascular malformation will be enrolled. Eligible patients must be deemed by the investigator to be unsuitable for effective surgical treatment. After screening, qualified participants will receive oral everolimus once daily in continuous cycles of 28 days. The daily dosage of everolimus for adult patients will be 10 mg. Treatment will continue until disease progression, intolerable toxicity, lack of clinical benefit as determined by the investigator, study termination, or any other pre-defined discontinuation criteria-whichever occurs first. The primary objective of this study is to assess the therapeutic efficacy of everolimus in patients with vascular malformations, primarily through evaluation of lesion volume using MRI sequences. All participants will undergo MRI-based target lesion volume analysis and assessments of microbleeding, iron deposition, and hemorrhage risk during the screening period, at the end of treatment cycles 3, 6, and 12, every 6 cycles thereafter, and at the end-of-treatment (EOT) visit. Clinical signs, symptom scores, and quality-of-life improvements will also be evaluated. The acceptable window for these assessments is ±7 days. If a participant has not undergone efficacy evaluation within 3 months, a final assessment should be conducted at the EOT visit.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
An oral mTOR inhibitor
the Department of Neurosurgery, China International Neuroscience Institute, Xuanwu Hospital, Capital Medical University
Beijing, China
RECRUITINGObjective Response Rate (ORR) assessed by MRI
Time frame: 1 year
Duration of Response (DOR) and 1-year DOR rate assessed by MRI
Time frame: 1 year
Mean Percentage Reduction in Target Lesion Volume at 3, 6, and 12 Months Assessed by MRI
Time frame: 1 year
Changes in Perilesional Microbleeds and Iron Deposition at 3, 6, and 12 Months Assessed by MRI QSM Sequence.
Time frame: 1 year
Changes in lesion hemorrhage risk during the follow-up period
Based on MRI, imaging evidence of hemorrhage was defined as changes meeting the following criteria: (1) an increase in lesion size or alteration in its shape; and (2) a change in signal intensity, primarily characterized by a shift from hypointensity to hyperintensity, especially on T1-weighted sequences. Hemorrhage rates were calculated at 3, 6, and 12 months during follow-up to comprehensively evaluate the temporal changes in hemorrhagic risk of the lesion.
Time frame: 1 year
The frequency and severity of intracranial hemorrhage events during the follow-up period were assessed through clinical records, imaging examinations (MRI), and the mRS scoring system.
Time frame: 1 year
Changes in patients' clinical signs and symptom scores from baseline.
The effectiveness of the medication and patient prognosis were comprehensively assessed by comparing the clinical symptoms and mRS scores of patients after treatment with baseline clinical symptoms and mRS scores. The mRS scale ranges from 0 to 5, with a decrease in score indicating a more favorable prognosis.
Time frame: 1 year
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Disease Control Rate (DCR) assessed by MRI
Time frame: 1 year
Progression-Free Survival (PFS) and 1-year PFS rate assessed by MRI
Time frame: 1 year
The frequency and types of seizures during the follow-up period were recorded and assessed through seizure event logs and classification of seizure types.
Time frame: 1 year