Prospective Observational Study Evaluating the Prevalence of Adenosine Deaminase (ADA) Enzyme Deficiency Disease in Patients With Lymphopenia and/or Immunoglobulin E Elevation in Adult Immunology and Hematology Clinics

This study was designed as an observational, prospective, epidemiological screening study. Patients who have been admitted to the center and whose lymphopenia and/or Immunoglobulin E elevation has been detected in at least one examination in their medical history will be included.
In accordance with the relevant legislation, patients are required to accept and sign the Informed Consent Form regarding their participation in the study.
Current data that the physician has already questioned in his daily practice will be collected from patients who have agreed to participate in the study, and a blood sample will be taken from patients on Guthrie paper. This sample will be prepared by taking it from the patient as the physician deems appropriate, dripping it into a special area designated on Guthrie paper and drying it.
The test result will be sent to the researcher by e-mail. In case of formation of new information for each patient, consultation will be provided by the responsible researcher.
Thus, the prevalence of ADA enzyme deficiency disease in patients with lymphopenia will be evaluated. In addition, with this study, it will be scientifically demonstrated whether lymphopenia is a parameter that facilitates early diagnosis of ADA patients.

开始日期2021-12-03

申办/合作机构

TRPHARM Ilaç Sanayi Tic. AS

NCT05300360 / Unknown statusN/A

Prospective Observational Study Evaluating the Prevalence of Adenosine Deaminase (ADA) Enzyme Deficiency Disease in Adult Patients With Pulmonary Alveolar Proteinosis in Pulmonology Clinics

This observational study was designed as a prospective epidemiological screening study. Patients who have applied to the centers participating in the study and who have previously been clinically or pathologically diagnosed with PAP (Pulmonary alveolar proteinosis) will be included in the study. Up-to-date data will be collected from patients who have agreed to participate in the study, and a blood sample with DBS will be taken from patients. The blood taken will be subjected to analysis for ADA metabolites. For patients with a high metabolic test, the responsible researcher will advise on clarifying the diagnosis with a genetic test other than the study. In case of formation of new information for each patient, consultation will be provided by the responsible researcher. Thus, the prevalence of ADA enzyme deficiency disease will be evaluated in patients diagnosed with PAP.

开始日期2021-08-16

申办/合作机构

TRPHARM Ilaç Sanayi Tic. AS

NCT05300347 / Unknown statusN/A

Prospective Observational Study Evaluating the Prevalence of Adenosine Deaminase (ADA) Enzyme Deficiency Disease in Adult Patients With Bronchiectasis, Lymphopenia and/or Immunoglobulin E Elevation in Pulmonology Clinics

This observational study was designed as a prospective epidemiological screening study. Patients who applied to the centers participating in the study, bronchiectasis was detected on at least one computed tomography of the lungs; Immunoglobulin E height and/or were found to be lymphopenic on at least one examination will be included in the study. Up-to-date data will be collected from patients who have agreed to participate in the study, and a blood sample with DBS will be taken from patients. The blood taken will be subjected to analysis for ADA metabolites. For patients with a high metabolic test, the responsible researcher will advise on clarifying the diagnosis with a genetic test other than the study. In case of formation of new information for each patient, consultation will be provided by the responsible researcher. Thus, the prevalence of ADA enzyme deficiency disease in patients with bronchiectasis, Immunoglobulin E elevation and/or lymphopenia will be evaluated. In addition, with this study, it will be scientifically demonstrated whether lymphopenia and/or Immunoglobulin E height is a parameter that facilitates the early diagnosis of patients with late-onset ADA enzyme deficiency.

开始日期2021-06-17

申办/合作机构

TRPHARM Ilaç Sanayi Tic. AS

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项与 TRPHARM Ilaç Sanayi Tic. AS 相关的文献（医药）

2024-06-01·Journal of Clinical Oncology

Effect of sodium thiosulfate anhydrous on cisplatin-induced ototoxicity in pediatric cancers: Turkish compassionate use treatment protocol—Updated results.

作者: Unal, Emel Cabi ; celik Ates, Dilvin ; Canpolat, Cengiz ; Sevinir, Betul ; Sivrice, Ayse Cigdem ; Tuncel, Tolga ; Tacyildiz, Nurdan ; Ataseven, Eda

e15075 Background: Cisplatin induced ototoxicity (CIO), with permanent hearing loss is observed in >60% pediatric patients in Turkey. PEDMARK (Sodium Thiosulfate anhydrous) reduced the risk of CIO cases with non-metastatic solid tumors observed in Phase 3 studies (SIOPEL 6 & COG ACCL0431). We aimed to evaluate the investigational product PEDMARK provided for pediatric cases via a National Compassionate Use Treatment Protocol (CUP) in Turkey. Methods: Patients (1 month to <18 years) with a localized, nonmetastatic solid tumor, standard-risk hepatoblastoma (HBB), nasopharyngeal carcinoma (NPC) and medulloblastoma MB whose treatment plans were including cisplatin were eligible. PEDMARK was administered intravenously 6 hours after each cisplatin dose infusion. Adverse events, demography, pre and post- hearing were recorded from 21OCT2019-21SEPT2022. Results: The CUP enrolled 12 patients, 8 HBB, 3 NPC and 1 MB in 3 years, from 6 academic institutions. HBB patients' median age was 1year (3mos-3 yrs). All pts had newly diagnosed HBB PRETEXT 3 (n = 5) with a median baseline AFP OF 54.000. 3 NPC age 13-17 yrs, MB 6yrs of age. All patients had Brock Grade 0 on hearing tests prior to treatment Patients received a median 0f 6 cycles (range 4-6) of cisplatin and PEDMARK. Patients with hearing tests available post-treatment (n=11), all maintained Brock Grade 0. At a median of 0.8mos post-treatment, CR were observed in all patients except one HBB with progressive disease. All CRs wer sustained 18.8 mos. AEs in 2 patients were Grade 1-2 nausea/vomiting, no Grade >3 AEs were reported. Conclusions: PEDMARK was well tolerated, provided via a Turkish CUP to reduce the CIO in pediatric patients with nonmetastatic solid tumors. No hearing loss was observed/ detected post-treatment and the tumor outcomes were consistent with the Phase 3 studies. This is very encouraging.

2017-05-04·Human Vaccines & Immunotherapeutics3区 · 医学

Vaccinating healthcare workers: Level of implementation, barriers and proposal for evidence-based policies in Turkey

3区 · 医学

Article

作者: Tanriover, Mine Durusu ; Ozisik, Lale ; Unal, Serhat ; Altınel, Serdar

The role of healthcare workers in life-long vaccination is very important in the means of 2 sided infection, rising patient awareness and being a role model for the patients. Numerous organizations publish guidelines for vaccination of HCWs, while healthcare facilities develop vaccination policies according to the accreditation standards. Nevertheless, vaccination rates among HCWs are far below targets. The obstacles to getting vaccinated or recommending vaccination may include rather universal factors such as the vaccine paradox, however in the case of HCWs, probably a different set of factors are included. The aims of this article are to gain an overview of vaccination strategies for HCWs, to assess the coverage rates of HCWs and make in-depth analyses of the potential barriers to vaccination and potential factors to motivate HCWs for vaccination in Turkey and to compare them with the global picture to improve implementation of policies concerning vaccination of HCWs.

项与 TRPHARM Ilaç Sanayi Tic. AS 相关的新闻（医药）

2022-07-05

·医药魔方Info

盘点：2022上半年license-out交易

根据医药魔方全球新药库——医药交易模块，2022年共达成license-out（出海）项目共28起，包括20项创新药/新技术交易项目，4个生物类似药项目及4个仿制药/改良型新药项目。在此，对达成的创新药相关项目做一简单分析。20个license-out创新药合作/授权项目交易总金额累计达56.7亿美元，其中首付款合计2.08亿美元，里程碑付款合计54.6亿美元。注：1.此表仅统计了披露数据的情况；2.“/”表示金额未披露；列表下载请点击↓↓↓2022上半年license-out交易20款license-out创新药合作/授权项目中，有80%（16/20）属于肿瘤领域，其他领域项目仅占20%，其中包括济民可信与Orion达成的非阿片类镇痛新药项目，逸达生物与TR-Pharm达成的罕见病项目（口服变构ALDH2激活剂），以及礼来与和誉医药达成的治疗代谢性疾病的新型小分子项目。从开发阶段来看，这20款新药/新技术项目除了亿一生物的艾贝格司亭α正在申报上市外，均处于I/II期以及临床前研究阶段。从转让方和受让方来看，转让方为小型企业或者biotech/biopharma，尚无传统大药企的身影。而受让方既有像罗氏、默沙东、礼来、强生等大型跨国药企，也有一些国外小企业。从所授权的项目技术类型来看，既有新型小分子、单抗类药物，也有双抗、ADC（抗体-药物偶联物）、CAR-T细胞疗法等新技术。不过，总体来看，生物药交易占比更高（15:5）。接下来，对交易总金额TOP 5的项目做一简单介绍：NO.1 SAFEbody平台掩蔽安全抗体3月2日，天演药业宣布，其和赛诺菲达成合作，双方就天演药业独有的安全抗体SAFEbody技术授权开展多靶点合作，开发新一代掩蔽抗体候选药物，全部合作交易金额达25亿美元，这一消息一时被刷屏。早在此次合作之前，赛诺菲就已积极布局掩蔽技术。今年年初，其耗资超10亿美元收购了美国一家专注于开发经掩蔽的蛋白酶激活的肿瘤免疫治疗剂公司——Amunix。天演药业SAFEbody还吸引了众多国际合作伙伴。其与美国Exelixis、瑞士ADC Therapeutics以及日本田边研究实验室，在单抗、双抗、ADC等不同药物模式上达成合作。这不仅验证了该技术在不同药物类型上均具有优异性能，更表明了这一技术已经受到广泛的国际认可。掩蔽抗体的优势在于抗体在肿瘤微环境中被特定酶有条件激活后，才能与表达特异性受体的癌细胞结合。从而大大提高了安全性，并扩大治疗窗口。NO.2 生物大分子抗癌药5月13日，科伦博泰与默沙东签订协议，将具有自主知识产权的生物大分子肿瘤项目A的大中华区以外权益以4700万美元首付款（以及不超过13.63亿美元的里程碑付款和产品上市后的净销售额提成）许可给默沙东。据科伦药业最新公告，科伦博泰已于2022年6月24日收到MSD支付的3000万美元付款。这项交易的首付款排今年上半年国产新药出海交易金额首位，总金额排第2位。虽然未披露具体是哪一款产品，经业内推测大概率为靶向TROP2的抗体偶联药物SKB26。目前全球仅有一款TROP2-ADC获批上市，为Immunomedics（已被吉利德收购）的戈沙妥珠单抗（Trodelvy）。2019年4月，云顶新耀从Immunomedics引进Trodelvy。2020年4月，该药首次在美国获批上市，用于三线治疗转移性TNBC，2021年4月，Trodelvy新适应症获批，治疗转移性尿路上皮癌。随着该类药物市场前景逐渐明朗，加上默沙东面临重磅产品Keytruda在2028年跌落专利悬崖将带来的收入空缺的窘境。重金获得该款产品也在情理之中。目前SKB26的开发进展处于第一梯队，单药三线治疗晚期或转移性TNBC的III期注册临床试验正在进行中，有望成为全球第二款获批的TROP2 ADC。NO.3 CLDN18.2/CD3双抗4月7日，和铂医药与阿斯利康就CLDN18.2xCD3双特异性抗体（HBM7022）的开发与商业化达成全球对外授权协议。HBM7022是基于和铂医药HCAb的免疫细胞衔接器平台HBICE开发的一种新型双特异性抗体。和铂医药将获得2500万美元的预付款和最高达3.25亿美元的里程碑付款，以及基于未来HBM7022销售额的特许权使用费。这是国内双抗药物首个出海授权，对于和铂医药而言也具有里程碑意义，意味着MNC对和铂医药核心技术以及自主研发实力的认可。这笔交易也使得和铂医药股价开盘大涨60%。NO.4 TIGIT抗体1月10日，君实生物与Coherus BioSciences共同宣布，双方将扩大于2021年达成的肿瘤免疫（特瑞普利单抗）领域合作，Coherus已启动行使JS006（君实生物自主研发的抗TIGIT单抗）在美国和加拿大的许可选择权的程序。Coherus将向君实生物支付3500万美元首付款，最高达2.55亿美元的开发、申报和销售里程碑付款，以及产品销售净额18%的销售分成。TIGIT单抗是近几年肿瘤免疫治疗热门靶点。根医药魔方新药数据库，目前在研的靶向药多达60款。罗氏tiragolumab作为第一梯队药物，接连在两项关键III期研究中遭遇失败也使这一靶点的前途被蒙上阴影。相关阅读：TIGIT靶点遇挫，制药巨头们60亿美元赌注蒙上阴影两家公司合作的特瑞普利单抗一线和二线治疗鼻咽癌的BLA申请在今年上半年遭到了FDA延期，FDA要求君实生物进行一项质控流程变更，君实生物表示将于今年仲夏重新递交BLA申请，受新冠疫情影响，具体现场核查时间也将另行通知。NO.5 新型小分子1月18日，和誉医药宣布其已与礼来订立一份全球合作及独家许可协议，以进一步针对心脏代谢疾病等未被满足医疗需求的重大疾病中的未公开靶点，在新型小分子药物的发现、开发及潜在商业化领域开展合作。和誉医药将负责利用其专有的药物研发平台进一步开展小分子药物的发现和开发，这些小分子药物将作用于新型且具有挑战性的药物靶点。和誉医药有权收取最多2.58亿美元的潜在费用，以及因该协议产生的销售分级特许权使用费。在过去的五年中，和誉医药专有技术平台已成功研发出10款候选药物分子，包括4种已经进入临床试验阶段的药物。和誉医药管线中FGFR为其布局重点，在研药物均具有迭代可能性。肿瘤免疫领域，不同于其他药企开发抗体为主，和誉医药针对CSF-1R、PD-L1、CD73、CXCR4和RORγt等靶点均开发小分子抑制剂。迭代小分子靶向药物和小分子肿瘤免疫疗法，是和誉医药快速发展的两架强大引擎。License out交易越发频繁，并且涉及到许多创新技术平台和早期临床项目。这一方面说明中国创新药出海寻求发展是企业生存的必然之路，另一方面也反映出中国企业创新能力的崛起并逐渐得到了广泛认可。期待越来越多的中国原创新药能够走出国门，造福全球患者。‍

抗体合作抗体药物偶联物引进/卖出小分子药物

2022-06-28

·BioSpace

Blood Cancer Segment Will Expand The Fastest, Expanding At A 10% CAGR During The Projected Period. “Oncology clinical research has gained traction in recent years, and this trend has resulted in the discovery of various unique medications. Medical experts have paid close attention to leukemia research in particular. Given the aforementioned considerations, the worldwide oncology biosimilars market will gain traction in coming years,” says a Fact.MR Analyst. Fact.MR – A Market Research and Competitive Intelligence Provider: The global oncology biosimilars market is expected to surge nearly 3x across the 2022-2032 forecast period, reaching US$ 12 Billion. As of 2022, the market is expected to garner a valuation of US$ 4.2 Billion. Advancements in cancer research to develop breakthrough therapeutics and diagnostics is expected to impel market growth in the future. From 2015 to 2021, sales of oncology biosimilar drugs flourished at a CAGR of 9%, closing at a value of US$ 4 Billion. The increased incidences of cancer, such as lung cancer and breast cancer, a susceptible ageing population, and an increase in research and development efforts done by numerous pharmaceutical firms have all led to the expansion of the oncology biosimilars market. Additionally, increased patient awareness, advancements in therapeutic approaches, and the high cost of biologics medications are all factors influencing the demand for oncology biosimilars. Conversely, product recalls, allied with restrictive regulations, are factors inhibiting the growth of this sector. Because of the increasing frequency of cancer and the increase in the number of FDA-approved medications, North America has seen a good expansion in the oncology biosimilars market throughout the anticipated period. For more insights into the Market, Get A Sample of this Report! Key Takeaways from the Market Study Demand for oncology biosimilars is expected to grow at an 11% CAGR between 2022 and 2032. By 2032, the oncology biosimilars market in the United States will reach US$ 5.6 Billion by 2020. China is expected to register a 7.7% CAGR with regard to the Oncology Biosimilars industry Japan is expected to document a CAGR worth 7.5% in the Oncology Biosimilars market The neutropenia segment is expected to grow at a rapid 7% CAGR over the assessment period. Competitive Landscape The worldwide market for oncology biosimilars is a highly consolidated market. The top five market participants jointly account for more than 70% of the market share. To expand their market footprints, these companies rely heavily on active collaborations to boost their product distribution networks. In 2018, Biocon, for example, established an exclusive global agreement with Sandoz on next-generation biosimilars. Similarly, Dr. Reddy's cooperated with TR Pharm in 2016 to offer three biosimilars in Turkey. Pfizer, Inc., Celltrion, Inc., Sandoz International GmBH, STADA Arzneimittel AG, and Apotex, Inc. are some of the other significant industry participants in the global oncology biosimilars market. Atos has announced recently in April 2022 that it intends to forge a 5-year contractual agreement to deliver managed services for digital operations, applications and cybersecurity for Independent Health (IH). Atos’ operational solutions will leverage infrastructure and digital platforms to optimize operating costs and generate efficiencies across the business landscape. For Comprehensive Insights Ask An Analyst Here Key Segments Covered in the Oncology Biosimilars Industry Report Oncology Biosimilars by Drug G-CSF Monoclonal Antibody Hematopoietic Agents Oncology Biosimilars by Disease Indication Breast Cancer Non-Small Cell Lung Cancer Colorectal Cancer Neutropenia Blood Cancer Leukemia Myeloid Leukemia Chronic Lymphocytic Leukemia (CLL) Non-Hodgkin Lymphoma Others Oncology Biosimilars by Distribution Channel Hospital Pharmacy Retail Pharmacy Online Pharmacy Oncology Biosimilars by Region North America Europe Asia Pacific Rest of the World Get Customization on this Report for Specific Research Solutions Fact.MR’s Domain Knowledge in Healthcare Our healthcare consulting team guides organizations at each step of their business strategy by helping you understand how the latest influencers account for operational and strategic transformation in the healthcare sector. Our expertise in recognizing the challenges and trends impacting the global healthcare industry provides indispensable insights and support - encasing a strategic perspective that helps you identify critical issues and devise appropriate solutions. Check out more studies related to Healthcare Industry, conducted by Fact.MR: Aesthetic Medicine Market - Aesthetic Medicine Market Analysis by Procedure Type (Invasive Procedures, Non-Invasive Procedures), by Region - Global Forecast 2022-2032 Regulatory Affairs Outsourcing Market - Regulatory Affairs Outsourcing Market by Services (Regulatory Consulting Outsourcing, Legal Representation Outsourcing), by Category (Drugs, Biologics), by Indication (Oncology, Neurology, Radiology), by Stage, by Company Size, by End Use & Regional Forecast 2022-2032 mHealth Apps Market - mHealth Apps Market Analysis by Product Type (Medical Apps [Women’s Health Apps, Personal, Health Record Apps, Medication Management Apps, Diagnostic Apps], Fitness Apps [Diet & Nutrition, Exercise & Fitness, Lifestyle & Stress]), by Region - Global Forecast 2022-2032 Biotechnology & Pharmaceutical Services Outsourcing Market - Biotechnology & Pharmaceutical Services Outsourcing Market Analysis by Service (Product Testing & Validation, Training & Education, Product Design & Development, Product Maintenance, Regulatory Affairs, Consulting), by End Use & Region - Global Forecast 2022-2032 Electronic Health Records Market - Electronic Health Records Market Analysis by Product (Client-server-based HER, Web-based HER), by Type (Acute, Ambulatory, Post-acute Electronic Health Records), by Business Model, by End Use, by Region - Global Forecast 2022-2032 Home Infusion Therapy Market - Home Infusion Therapy Market Analysis By Product (Infusion Pumps, Intravenous Sets, IV Cannulas, Needless Connectors), By Application (Anti-infective, Endocrinology, Hydration Therapy, Chemotherapy, Parenteral Nutrition), By Region - Global Insights 2022 to 2032 Skincare Devices Market - Skincare Devices Market Analysis by Product (Diagnostic Devices [Dermatoscopes, Microscopes, Biopsy Devices], Treatment Devices [Light Therapy Devices, Electrosurgical Equipment, Liposuction Devices, Cryotherapy Devices]), by Application, by End Use & Regional Forecast 2022-2032 Clear Aligners Market - Clear Aligners Market Analysis by Age Outlook (Adults, Teens), by End-use Outlook (Hospitals, Standalone Practices, Group Practices), by Region - Global Forecast 2022-2032 Dental Laboratories Market - Dental Laboratories Market Analysis By Type (Restorative, Orthodontic, Endodontic, Oral care, Implant), By Application (Dental Radiology Equipment, Dental Lasers, Systems & Parts), By Region - Global Insights 2022-2032 Biomarkers Market - Biomarkers Market Analysis by Disease Type (Cardiovascular Diseases, Cancer, Neurological Diseases, Immunological Diseases), by Type (Safety, Efficacy, Validation Biomarkers), by Application & by Region - Global Forecast 2022-2032 About Us: Market research and consulting agency with a difference! 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