A Singlecenter, single-arm, prospective study assessing efficacy and safety of Sirolimus granules twice daily administration in patients with intractuable vascular malformations ( Sirolimus granules twice daily administration for intractuable vascular malformations)

未提供0 个研究点目标入组 11 人开始时间: 2020年9月30日最近更新: 2025年5月9日

适应症

试验速览

阶段: 不适用
状态: 进行中（未招募）
入组人数: 11
主要终点: -

研究概览研究设计入排标准结局指标研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional
分配方式: Single Arm Study
干预模型: Single Assignment
主要目的: Treatment Purpose
盲法: Open(masking Not Used)

入排标准

年龄范围: 1month old over 至 No limit（—）
性别: All

入选标准

•one month and older
•Patients with intractable vascular anomaly diagnosed by the investigator/subinvestigator
•Have one or more measurable target lesions on MRI before treatment starts.
•Severe disability or refractory symptoms depending on target disease
•Have sufficient liver, kidney and heart functions at the time of registration
•Written consent to participate in this clinical trial has been given by the subject in person or by a legal guardian (when the subject is younger than 20 years at consent).

排除标准

•Patients who have received a targeted drug related to the mTOR pathway within 8 weeks before the start of study drug administration.
•Patients with infections that require systemic treatment.
•Patients with a Karnofsky PS score of 30 or less (10 years or older) or Lansky play-PS of 30 or less (under 10 years) due to permanent sequelae due to cerebral disorders.
•Patients with any of the following complications:
•Uncontrolled diabetes, Uncontrolled hypertension, Uncontrolled hyperlipidemia, Severe liver disease, Severe renal disease
•Patients receiving long-term immunosuppressive drugs (cyclosporine, tacrolimus, etc.) or steroids (4 weeks or more) at the time of registration.
•Patients who require administration of a drug that affects CYP3A4 activity one week before starting sirolimus administration.
•Patients with immunodeficiency such as HIV and primary immunodeficiency.
•Patients who are carriers of hepatitis B virus and / or carriers of hepatitis C virus.
•Patients who have undergone surgery (resection, sclerotherapy, endovascular treatment) for the target lesion within 2 weeks before obtaining consent.
•Patients who have received a therapeutic drug (propranolol, Eppikajutsuto, Tokikenchuto, interferon, octreotide, bisphosphonate, denosumab, etc.) for the target disease within 2 weeks before obtaining consent.
•Patients who received myelosuppressive chemotherapy, biologics, drugs not covered by insurance, etc. within 4 weeks before obtaining consent.
•Patients who received radiotherapy for the target lesion within 24 weeks before obtaining consent.
•Patients who meet any of the following.
•may be pregnant or pregnant, lactating, Disagree with contraception during this study
•If administering tablets, persons diagnosed with lymphangioma, lymphangiomatosis, or Gorham's disease.
•In addition, patients whose investigator / assigning doctor determines that participation in this study is inappropriate.

结局指标

主要结局

Target lesion response rate determined by Independent Review Facility after 24 weeks of treatments

次要结局

QOL improvement
ADL improvement
Adverse events and side effects
Clinical test values vital signs
Pharmacokinetics
Response rates of target lesions(12, 52 weeks after administration)
Improvement of lesions other than target lesions(12, 24, and 52 weeks after administration)
Respiratory function(24 and 52 weeks after administration)
Pleural effusion(12, 24 and 52 weeks after administration)
Ascites(12, 24, and 52 weeks after administration)
Anemia and blood coagulation parameters(12, 24, and 52 weeks after administration)
Bleeding(12, 24, and 52 weeks after administration)
Pain(12, 24 and 52 weeks after administration)

研究者

发起方

未提供

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