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.
Patients aged ≥18 and ≤65 years;
No gender restrictions;
Diagnosed with vascular malformation by MRI;
No major surgery within the past 3 months;
Diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT), Arteriovenous Malformation (AVM), or PTEN Hamartoma Tumor Syndrome (PHTS);
Patients who have previously received any of the following treatments after birth: participation in other interventional clinical trials targeting cerebral cavernous malformations (CM);
Presence of malignant tumors currently or within the past three years, except for curatively treated non-melanoma skin basal cell carcinoma, ductal carcinoma in situ of the breast, or cervical carcinoma in situ;
Unable to undergo MRI scans and/or have contraindications for MRI (e.g., interference from prosthetics, orthodontic devices, etc., affecting target lesion volume analysis on MRI);
Single group
Everolimus 10mg daily
DrugAn oral mTOR inhibitor
Objective Response Rate (ORR) assessed by MRI
Duration of Response (DOR) and 1-year DOR rate assessed by MRI
Mean Percentage Reduction in Target Lesion Volume at 3, 6, and 12 Months Assessed by MRI
Changes in Perilesional Microbleeds and Iron Deposition at 3, 6, and 12 Months Assessed by MRI QSM Sequence.
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.
Incidence of Treatment-Emergent Adverse Events
The type and frequency of treatment-emergent adverse events (TEAEs) occurring during the treatment period, evaluated according to NCI-CTCAE version 5.0;Treatment-related TEAEs;Serious adverse events (SAEs);TEAEs leading to permanent discontinuation of treatment;Incidence and frequency of death occurring within 30 days after the last dose of treatment;Laboratory abnormalities assessed according to NCI-CTCAE version 5.0.
Changes in patients' FACIT scores during the follow-up period
The change in the patient's quality of life compared to baseline was primarily assessed using the Functional Assessment of Chronic Illness Therapy (FACIT) system. The score ranges from 0 to 108, and the scale is divided into four parts: Physical Well-Being (PWB), Social and Family Well-Being (SWB), Emotional Well-Being (EWB), and Functional Well-Being (FWB). Among these, lower scores in Physical Well-Being and Emotional Well-Being indicate better quality of life and prognosis, while higher scores in Social and Family Well-Being and Functional Well-Being indicate better quality of life and prognosis.
Pharmacokinetic (PK) profile/parameters
In this study, we will evaluate the pharmacokinetic (PK) profile of the drug by measuring "peak plasma concentration (Cmax)" over the one-year treatment period to assess the drug's metabolic behavior.
Pharmacokinetic (PK) profile/parameters
In this study, we will assess the pharmacokinetic (PK) profile of the drug by measuring the area under the plasma concentration versus time curve (AUC) over the one-year treatment period to evaluate the drug's metabolic characteristics.
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Xuanwu Hospital, Beijing
Lead sponsor
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Collaborator
