This Study Aims to Improve the Stratification of Patients With Acute Myeloid Leukemia to Enable More Personalized and Effective Treatments, and to Identify and Functionally Validate, at the Single-cell Level, Biomarkers That Can Predict Response to Venetoclax.
报名条件（概要）
- 年龄：18 Years – 不限
- 性别：不限
- 健康志愿者：不接受
完整入排标准请以官方页面为准。
开展国家/地区
Italy
药品与企业信息
This intervention enrolls \~420 patients to investigate AML through multi-level genomic and transcriptomic analysis. Bone marrow samples are collected at diagnosis, relapse, and day 28 of treatment. All patients undergo bulk NGS genotyping using a 69-gene panel. Forty patients receiving venetoclax are selected for detailed single-cell RNAseq analysis (\~5000 cells) to dissect cellular heterogeneity, identify blast populations, assess microenvironment composition, and predict cell states via RNA velocity. Four patients undergo spatial transcriptomic analysis (Xenium) on bone marrow biopsies at diagnosis and day 28 to evaluate RNA expression within tissue architecture. This comprehensive approach enables identification of genomic/transcriptomic features associated with treatment response, chemoresistance mechanisms, and personalized targeted treatment markers in heterogeneous AML populations.
研发企业
试验官方登记的主要申办方；研究机构申办时会显示相应机构名称。
生产企业
公开药品批准资料或正式说明书暂未提供明确的生产企业信息。
合作机构
- ASST Grande Ospedale Metropolitano Niguarda研究机构/其他
- AOU S. Orsola MalpighiUNKNOWN
- Humanitas Clinical and Research Center研究机构/其他
- A.O. Ospedale Papa Giovanni XXIII研究机构/其他
- Azienda Ospedaliero-Universitaria di Parma研究机构/其他
- Ospedale Policlinico San Martino研究机构/其他
研发企业与生产企业可能不是同一家。研究阶段药品可能尚未公布生产企业；生产企业仅取自美国上市原研药说明书所列企业（不含仿制药），不作推测；仅在其他国家上市的药品暂无法显示。
研究简介（原文）
The current classification of acute myeloid leukemias (AML) includes more than 20 different diseases, grouped based on their distinctive genomic characteristics, which reflect substantial heterogeneity. The rationale underlying this project is the idea that an effective diagnostic process and functional dissection of transcriptional heterogeneity at the single-cell level will lead to the identification of cell-intrinsic or cell-extrinsic biomarkers of response to novel therapies in order to improve patient care. In particular, with the study presented here, the investigators intend to obtain data relating to the sub-classification of patients into genomic subgroups with biological and prognostic relevance. Genomic subgroups are categories into which patients affected by the same disease can be divided based on the genetic characteristics found in each patient. The study foresees that part of the biological sample, collected through bone marrow blood sampling, already provided for by clinical practice will be subjected to further analyses. Specifically, DNA and RNA sequencing analyses will be performed on the cells that will be isolated from the bone marrow blood. The objective is to search for molecular markers that may allow better biological and prognostic classification of patients. Molecular markers are particular DNA sequences that, in our study, would enable the classification of patients into subgroups.
适应症
主动联系研究团队
这项试验目前在中国大陆没有研究中心
这种情况下，直接发"入组咨询"往往会被回复"您所在地区没有中心"。更有效的做法是申请同情用药／扩展准入（由主治医生一同提出），或询问能否到最近的境外中心参加。下面已为您切换到对应的信件版本。
查看完整申请路径与成功率说明 →研究中心联系方式
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Haematology UnitMilan，Italy
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英文版（发给研究团队）
Dear Study Team, I am a patient in China and I am interested in participating in your clinical trial: Study Title: This Study Aims to Improve the Stratification of Patients With Acute Myeloid Leukemia to Enable More Personalized and Effective Treatments, and to Identify and Functionally Validate, at the Single-cell Level, Biomarkers That Can Predict Response to Venetoclax. ClinicalTrials.gov Identifier: NCT07874100 Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico Investigational product: Bulk genotyping of AML samples at diagnosis and relapse, scRNA analysis at diagnosis, scRNAseq and genotyping analysis at day 28 after therapy, spatial transcriptomic analysis at day 0 and 28 About me: - Age: (please fill in) - Sex: (please fill in) - Confirmed diagnosis and stage: (please fill in) - Biomarkers / genetic testing: not available - Prior and current treatments: (please fill in) - Performance status (ECOG): not assessed - Current location: China - Travel ability: (please fill in) Could you please let me know: 1. Whether I might be eligible for this study; 2. Which site would be closest and most practical for me, and whether remote pre-screening is possible; 3. What documents (medical records, pathology or imaging reports, recent labs) I should prepare for pre-screening. I can provide English translations of my medical records. Thank you very much for your time and help. Kind regards, (your name) (your email / phone with country code)
中文对照（供您核对）
尊敬的研究团队： 我是一位来自中国的患者，希望咨询参加以下临床试验的可能： 研究名称：This Study Aims to Improve the Stratification of Patients With Acute Myeloid Leukemia to Enable More Personalized and Effective Treatments, and to Identify and Functionally Validate, at the Single-cell Level, Biomarkers That Can Predict Response to Venetoclax. 试验编号：NCT07874100 申办方：Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico 研究药物：Bulk genotyping of AML samples at diagnosis and relapse, scRNA analysis at diagnosis, scRNAseq and genotyping analysis at day 28 after therapy, spatial transcriptomic analysis at day 0 and 28 我的基本情况： - 年龄：（请填写） - 性别：（请填写） - 确诊疾病与分期：（请填写） - 基因／标志物检测：暂无 - 既往及当前治疗：（请填写） - 体能状态（ECOG）：未评估 - 目前所在城市：中国 - 可前往范围：（请填写） 想请教三个问题： 1. 我是否可能符合本研究的入组标准？ 2. 哪个中心对我最方便可行？能否远程预筛？ 3. 预筛需要准备哪些资料（病历、病理、影像、近期化验）？ 我可以提供英文翻译版病历。感谢您的时间与帮助。 顺祝安康 （您的姓名） （您的邮箱／带国际区号的电话）
提示：请与主治医生一起发信——由医生署名的申请，回复率远高于患者单独发信。首封邮件不要附身份证号等敏感信息。研究团队通常在 3–10 个工作日内回复，两周无回音可礼貌追问一次。
下一步怎么做？
- 把本页信息带给您的主治医生，评估是否适合参加。
- 点击下方按钮打开官方注册页，查看研究中心联系方式。
- 直接联系研究团队咨询报名事宜（通常有中文同声翻译服务可协助沟通）。