The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease (WD) is effective and safe. The study is enrolling children aged 8 years and older weighing at least 55 lb (25 kg) and adults with a recent diagnosis of WD. People recently diagnosed with WD, may be eligible for the study if they have either not started copper chelating treatment (such as DPA or trientine) or have been taking zinc salts for less than 28 days. Participants will be randomly allocated (like tossing a coin) to receive either DPA or TETA 4HCL for 48 weeks. During this time period participants will have up to 12 visits for health checks and assessments including blood and urine testing. In addition, at some visits participants may be asked to complete questionnaires on treatment satisfaction, and overall well-being.

开始日期2026-05-01

申办/合作机构

Orphalan SA

NCT07010575

/ Completed临床2期

Decentralized Study to Assess Patient Treatment Preference Comparing Their Current Standard-of-care Wilson's Disease (WD) Treatment to a New Trientine (TETA) 4HCl Formulation.

Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.

开始日期2025-07-15

申办/合作机构

Orphalan SA

CTR20244688

/ CompletedN/A

盐酸曲恩汀片在健康受试者中空腹生物等效性试验

主要目的：考察中国健康受试者在空腹状态下单次口服四川科瑞德制药股份有限公司生产的盐酸曲恩汀片与参比制剂体内药代动力学特征，评价两种制剂的生物等效性。次要目的：评价单次口服盐酸曲恩汀片受试制剂及参比制剂在中国健康受试者中的安全性。

开始日期2024-12-28

申办/合作机构

四川科瑞德制药股份有限公司

100 项与四盐酸曲恩汀相关的临床结果

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100 项与四盐酸曲恩汀相关的转化医学

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100 项与四盐酸曲恩汀相关的专利（医药）

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267

项与四盐酸曲恩汀相关的文献（医药）

2026-01-01·EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Further elucidation on the effect of food on the pharmacokinetics of trientine

Article

作者: Weiss, Karl Heinz ; Fu, ChauHwei ; Aliane, Verena ; Kruse, Carlot ; Scherpbier, Jan-Jaap ; van der Horst, Eric ; Abd-Elaziz, Khalid ; Dogterom, Peter ; Gerrits, Mireille

2025-12-01·Current Neurology and Neuroscience Reports

Pitfalls in the Diagnosis of Wilson Disease

Review

作者: Chakravarty, Ambar ; Roy, Debasish ; Mukherjee, Angshuman

PURPOSE OF REVIEW:

Wilson disease (WD), an uncommon autosomal recessive (AR) hereditary disorder characterized by abnormal accumulation of copper primarily in the liver and secondarily in other organs like the brain, is caused by a deficiency in the ATP7B transporter gene. The key to successful therapy is early diagnosis.

RECENT FINDINGS:

Mutant genes need to be inherited from both parents for phenotypic expression. The ATP7B gene located on chromosome 13q14.3 comprises 20 introns and 21 exons, encodes a protein of 165 amino acids, and this helps in incorporation of copper into ceruloplasmin, the copper binding protein. So far, more than 800 mutations have been reported, of which 380 have confirmed involvement in the pathogenesis of WD. The most common mutations are H1069Q and R778L in European and Asian populations respectively. Approximately 90%-98% of WD subjects are heterozygous, showing different mutations in each of the alleles encoding the ATP7B. Conversely, the phenotype and the penetrance of WD can be extremely variable. Even patients carrying two disease-causing mutations do not necessarily have a demonstrable alteration of copper metabolism. Considering the possibility of late-onset disease, asymptomatic cases, and phenotypic variability, it is crucial to evaluate previous and future generations of the index case. WD ranges from being asymptomatic in some patients to result in acute liver failure and/or a variety of neuropsychiatric disorders among others. Although WD may be present at any age, is more common between the ages of 5 and 35 years. However, it should be investigated in patients with liver failure of unknown cause and those with liver disease associated with neuropsychiatric symptomatology. Diagnosis requires a combination of clinical signs and symptoms, as well as relevant diagnostic tests such as measurement of serum ceruloplasmin, urinary excretion of copper, liver biopsy or genetic testing. Treatment is lifelong and includes chelating agents (penicillamine and trientine) and inhibitors of copper absorption (zinc salts). Liver transplant is an option for patients with end-stage liver disease. The key to successful therapy is early diagnosis.

2025-10-20·Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology

[Recommendations from the European Association for the Study of the Liver and the European Reference Network for Rare Liver Diseases Clinical Practice Guidelines for hepatolenticular degeneration].

Review

作者: Hou, W ; Zheng, S J ; Tang, S

Wilson disease (WD), also known as hepatolenticular degeneration, is an autosomal recessive inherited disorder of copper metabolism that primarily affects the liver, brain, and other organs. The diagnostic criteria include clinical features, biochemical tests (plasma ceruloplasmin, 24-hour urinary copper, liver copper content), and molecular genetic analysis. The Leipzig scoring system, supplemented by the use of exchangeable copper, is recommended for diagnosis. Pharmacotherapy mainly includes chelating agents (such as penicillamine and trientine) and zinc salts. Chelating agent therapy is recommended only for patients with severe liver disease. Patient monitoring is primarily based on clinical symptoms, liver biochemical indices, and copper metabolism parameters (such as 24-hour urinary copper and exchangeable copper) to identify poor adherence as well as over-treatment or under-treatment situations. The diagnosis and treatment of acute liver failure with WD is extremely challenging, as the diagnosis is difficult and medical treatment cannot save life. The role of liver transplantation has been clearly recognized in the treatment of acute liver failure with WD, and it may also be considered in cases with neurological involvement.

项与四盐酸曲恩汀相关的新闻（医药）

2026-02-13

·365医学网

年度盘点｜商洪才：肿瘤心脏病学：朝向多维度风险预测与精准干预

本文特邀：作者：商洪才单位：北京中医药大学东方医院随着肿瘤治疗迈入精准时代，化疗、免疫治疗等先进手段显著延长了众多肿瘤患者的生存期。然而，潜伏在抗肿瘤进程中的肿瘤治疗相关心血管毒性（CTR-CVT），正逐渐成为影响患者长期生存质量的关键问题。它不仅是肿瘤患者远期死亡的第二大诱因，在老年幸存者中，心血管疾病导致的死亡率甚至可能超过肿瘤本身，让抗肿瘤治疗的成效面临心血管功能受损的风险。传统诊疗的临床困境传统诊疗模式下应对 CTR-CVT 存在诸多局限：其一，早期临床表现隐匿，心肌肌钙蛋白（cTn）、脑钠肽（NP）等传统生物标志物对亚临床心肌损伤预测的敏感性仅 60%-70%，单一指标难以捕捉早期细微变化，导致漏诊率高；其二，传统心血管药物（如 ACEI、β 受体阻滞剂等）用于 CTR-CVT 预防的循证证据不充分，部分药物还与抗肿瘤治疗存在相互作用，且针对化疗、靶向治疗、免疫治疗等不同场景的特异性保护策略匮乏，难以覆盖多样化毒性机制；其三，传统模式缺乏规范化的多学科（MDT）协同机制，对儿童、孕产妇等特殊人群的心血管毒性管理数据有限，且随访体系不完善，未形成基于风险分层的全生命周期个体化监测方案，部分高危人群未能得到精准随访与干预。学术引领：领域专刊的系统探索为系统破解上述困境，学术探索持续深化。我们在权威期刊《Seminars in Cancer Biology》（中科院一区TOP，IF=15.7）上推出“Cancer therapy-related cardiovascular toxicity: Current progress and future perspective”专题专刊，系统梳理领域最新突破与临床实践经验。研究内容深入解析化疗、免疫治疗诱发心脏损伤的内在机制，剖析代谢、遗传及表观遗传层面的复杂调控因素，明确房颤等典型心血管并发症的早期识别方法与针对性干预策略，并探索契合临床实际的学科特色发展路径。该专刊的核心价值在于搭建基础研究与临床应用的桥梁，推动多学科交叉融合，助力精准防治策略落地，引领肿瘤心脏病学向个体化、精准化方向稳步发展，为全球科研与临床工作者提供系统前沿的学术参考。智能预测：从“被动检测”到“主动预警” 针对 CTR-CVT 早期预警的核心痛点，领域研究已形成共识：未来预警体系需突破“单一指标依赖”，转向多维度数据整合。通过联合 ST2、微小 RNA 等新兴生物标志物，结合超声心动图全球纵向应变（GLS）等影像学参数，再纳入肿瘤类型、治疗方案等临床基线信息，借助人工智能算法构建综合预测系统，实现从“被动检测”到“主动预警”的跨越，精准识别高风险人群，为早期干预争取时间，从源头降低心血管损伤风险。目前已有研究应用人诱导多能干细胞（hiPSCs）建模，精准复现不同肿瘤疗法诱导的心血管损伤表型，生成贴近人体真实病理状态的高质量数据。该模型可进一步捕捉药物毒性动态变化与个体敏感性差异，显著提升智能系统对肿瘤相关心血管风险的预判精度，为个体化风险预警与治疗方案优化奠定基础。药物治疗：传统局限与新型突破当前肿瘤心脏病传统治疗药物仍存在局限：部分药物存在疗效冲突（如右丙亚胺虽能减轻蒽环类药物心脏毒性，却可能加重化疗药物引起的骨髓抑制，同时影响抗肿瘤效果）；多数药物靶向性不足（如泛糖酵解抑制剂、广谱抗氧化剂缺乏组织特异性，易造成非选择性正常细胞损伤）；同时，传统心血管药物的治疗基于经典心血管疾病，未充分适配肿瘤治疗的特殊病理机制，导致其在肿瘤心脏病治疗中难以实现精准保护作用，且易受肿瘤患者的全身状态限制，无法满足临床多样化的防护需求。新型药物的精准突破：近年来，新型药物研发聚焦精准靶向与安全协同，取得多项关键进展：碳酸酐酶12（CA12）抑制剂indisulam，能精准阻断蒽环类药物诱导的心肌糖酵解异常，在动物模型中有效保护心脏且不干扰抗肿瘤疗效；铜螯合剂（四硫钼酸盐、曲恩汀）、铁螯合剂（去铁胺）等金属离子调控剂，分别针对铜死亡、铁死亡相关损伤，减少氧化应激与脂质过氧化；小檗碱、红景天苷等天然活性化合物，通过调节NRF2/GPX4通路、抑制铁死亡，展现出温和的心脏保护作用；跨界药物再利用也是重要方向：SGLT2抑制剂（恩格列净）、PDE5抑制剂（西地那非）不仅能保护心脏，还可能协同抑制肿瘤增殖。此外，氧化铈纳米酶、镁六氰合铁酸盐纳米催化剂等新型靶向制剂，通过靶向递送与精准抗氧化，进一步提升了心脏保护的安全性与有效性。这些新型药物正逐步破解传统治疗痛点，为肿瘤患者提供双重保障。中药探索：中西医结合的特色路径在中药治疗方面，依托国家科技重大专项（2023ZD0502700）资助，基于“先防后治、防治结合”的传统理念，针对蒽环类药物治疗的肿瘤患者，中药复方的大规模多中心随机对照临床试验持续推进。研究正初步验证其降低心脏损伤发生率、提升心脏损伤治疗缓解率的潜力，探索其既辅助保护心脏功能，又不干扰肿瘤治疗效果的核心优势及相关机制。这将为中西医结合防治CTR-CVT提供高级别临床证据支撑。中西医领域的并行探索与突破，有望为不同治疗场景下的CTR-CVT提供专属保护方案。肿瘤心脏病学正以多维度、跨学科的创新实践，为肿瘤患者搭建起稳固的心脏保护体系。该学科通过基础研究与临床应用的深度融合，借助AI、干细胞等前沿技术与中西医协同优势，让肿瘤治疗不再以牺牲心血管健康为代价。未来，随着更多精准靶点的发现、智能预警体系的完善与中西医结合证据的积累，肿瘤心脏病学将持续向个体化、全周期防治方向迈进，让每一位肿瘤患者既能成功战胜肿瘤，更能拥有健康的心脏与高质量的长期生存。学术关键词肿瘤心脏病学，多维度风险预测，精准干预，智能预警，多学科协同，中西医结合年度盘点学术特辑扫码查看更多内容参考文献（上下滑动阅览） 1. Li J, Chen L, Liu X, Zhang M, Liu X. Molecular mechanisms and therapies of immune checkpoint inhibitor associated myocarditis: Update on recent developments. Seminars in Cancer Biology 2025, 116: 108-120. 2. Liu C, Shen M, Liu Y, Manhas A, Zhao SR, Zhang M, et al. CRISPRi/a screens in human iPSC-cardiomyocytes identify glycolytic activation as a druggable target for doxorubicin-induced cardiotoxicity. Cell Stem Cell 2024, 31(12): 1760-1776.e1769. 3. Pinheiro EA, Fetterman KA, Burridge PW. hiPSCs in cardio-oncology: deciphering the genomics. Cardiovascular Research 2019, 115(5): 935-948. 4. Shang H, Cho W, Xing Y. Cancer therapy-related cardiovascular toxicity Current progress and future perspectives. Seminars in Cancer Biology 2026. 5. Stack JP, Moslehi J, Sayed N, Wu JC. Cancer therapy-induced cardiomyopathy: can human induced pluripotent stem cell modelling help prevent it? European Heart Journal 2019, 40(22): 1764-1770. 6. Yang F, Zhang G, An N, Dai Q, Cho W, Shang H, et al. Interplay of ferroptosis, cuproptosis, and PANoptosis in cancer treatment-induced cardiotoxicity: Mechanisms and therapeutic implications. Seminars in Cancer Biology 2024, 106-107: 106-122. 7. Yu C, Jiang L, Long L, Yu H. Atrial fibrillation in cancer patients: Epidemiology, identification and management. Seminars in Cancer Biology 2025, 111: 39-47. 8. Zeng C, Gao Y, Lan B, Wang J, Ma F. Metabolic reprogramming in cancer therapy-related cardiovascular toxicity: Mechanisms and intervention strategies. Seminars in Cancer Biology 2025, 113: 39-58. 9. Zhao Y, Jia H, Hua X, An T, Song J. Cardio-oncology: Shared Genetic, Metabolic, and Pharmacologic Mechanism. Current Cardiology Reports 2023, 25(8): 863-878. 声明：本文为学术进展综述，旨在传播医学信息，不构成任何具体的诊疗建议。如有疾病诊疗需求，请前往正规医疗机构就诊。专家简介商洪才 | 研究员/主任医师北京中医药大学东方医院（点击进入专家个人空间）教育部中医内科学重点实验室主任，北京中医药大学东方医院党委副书记、院长，国家杰出青年科学基金获得者。入选北京学者、岐黄学者、万人计划、教育部新世纪优秀人才计划、科技部创新人才推进计划，国家中医药管理局高水平中医药重点学科“中医药信息学”学科带头人，“十三五”“十四五”国家重点研发计划项目、科技创新2030重大项目负责人，国家科学技术进步奖二等奖第一完成人，全国创新争先奖获得者。致力于数智中医药及循证中医药研究，以方法创新突破中医药传承发展瓶颈。聚焦群体化循证规范与个体化中医辨治的融合，建立中医药循证研究“四证”方法学体系，覆盖高质量证据从产生到应用全链条，加速中医疗效优质证据的生产与转化。主持EXIT-SEP、DIRECTION、AUGUST-AHF等大规模多中心临床研究，明确了多项国际认可、中西医共识的高质量证据。创新性提出“数据筑基，智慧引航”的数智中医发展理念，主持建立广义望诊装备数智技术体系，探索中药循证毒理学数智融合研究新模式。获国家科技进步二等奖4项、省部级一等奖7项。授权发明专利17项，软著13项。在BMJ、Ann Intern Med、JAMA Intern Med、STTT等国内外期刊发表论文200余篇，H-index 46，连续六年（2020-2025）入选爱思唯尔“中国高被引学者”榜单，入选全球前2%顶尖科学家榜单（2023-2025）。主编《循证中医药学理论与实践》《智能中医学概论》、《单病例随机对照试验：理论与实践》、《循证中医药》等专著与教材10部。欢迎扫码入群交流长按识别二维码，即可进入沟通交流群专栏合集行业要闻｜创新研究文章｜课件｜视频｜音频高血压｜心脏重症｜心力衰竭｜冠心病｜瓣膜｜心律失常｜周围血管｜心电生理指南共识｜学术荟萃｜人物专访｜新心联播 ESC｜ACC｜ESC 2025 点击合集名称，查看更多内容👆👆👆 END 免责声明：365医学网所发表内容之知识产权为365医学网及主办方、原作者等相关权利人所有。未经许可，禁止进行转载、摘编、复制、裁切、录制等。经许可授权使用，亦须注明来源。欢迎转发、分享。如有侵权，请联系我们删除。投稿/会议发布，请联系010-63717246 editor@365yixue.com 点击下方卡片 / 获取更多内容点击“阅读原文”，开启学习之旅！

2025-12-12

·PRNewswire

Orphalan announces acquisition of Orphelia Pharma

PARIS, Dec. 12, 2025 /PRNewswire/ -- Orphalan, an international pharmaceutical company specializing in the development and commercialization of orphan drugs, has acquired Orphelia Pharma, a company committed to treating rare and severe pediatric diseases, notably in neurology and oncology. This strategic transaction strengthens Orphalan's position as a European leader with a global commercial footprint in rare diseases, expanding its therapeutic portfolio. Key highlights of the acquisition include: Expansion of our capabilities in rare neurological and oncological pediatric diseases Greater reach and improved access to medicines for children and adults with rare conditions A unique platform combining scientific excellence, agility, and global commercialization By bringing together complementary expertise, the companies are now positioned to accelerate innovation and improve access to transformative treatments for patients around the world. As they enter this new stage, their teams remain fully committed to ensuring continuity of care and supporting their partners and patients. Dr. Naseem Amin, Chief Executive Officer of Orphalan said: "The acquisition of Orphelia Pharma reinforces our mission: to provide innovative and accessible treatments to patients living with rare diseases, wherever they are. "Together, we are building a unique platform that brings together scientific excellence, agility, international development and commercial capabilities to advance care for patients of all ages suffering from orphan diseases." Dr. Hugues Bienaymé, Founder and Deputy Chief Executive Officer of Orphelia Pharma, added: "At Orphelia Pharma, we have dedicated ourselves to developing safe and effective pediatric medicines that address major unmet medical needs in rare diseases. "Joining Orphalan will allow us to increase the impact of our treatments and make them available more rapidly to the children who need them most." Gilles Alberici, Chief Executive Officer of Orphelia Pharma, commented: "Bringing together our two complementary companies marks an important milestone, both for our organizations and for the broader community of patients living with rare diseases." About Orphalan Founded in 2011, Orphalan SA is an international pharmaceutical company dedicated to the development and commercialization of orphan drugs. Originating from a collaboration with AGEPS and Hôpital Lariboisière, Orphalan developed Cuprior®, a treatment for Wilson's disease, now authorized in 38 countries and marketed in 28. Orphalan's ambition is to reduce diagnostic delays and make innovative orphan drugs accessible to as many patients as possible through a patient-centered approach and close collaboration with healthcare professionals. Orphalan's shareholders include Bpifrance, Advent, and the management team. About Orphelia Pharma Orphelia Pharma is a pharmaceutical company based in Paris and Lyon, specializing in the development and commercialization of medicines for rare and severe pediatric diseases. With one authorized medicine in the European Union ( Kigabeq®) and a second under regulatory review ( Kizfizo®), Orphelia Pharma has established regional distribution agreements across Europe and other regions and collaborates with academic and industrial partners to advance research in rare diseases. Further information: Bien Commun Advisory M. Benedict Brodrick +33.6.76.14.02.02 [email protected] SOURCE Orphalan 21% more press release views with Request a Demo

并购上市批准

2025-12-04

·百度百家

生物医药领域新动态：创新药物临床试验受理与进展

生物制药行业动态一览：创新药物临床试验申请受理情况。聚焦生物制药领域，我们为您追踪最新动态，了解企业资讯。聚焦生物制药领域，我们为您提供一个交流与分享的平台，让您随时了解生物企业的最新信息。通过分析6篇原创内容，深入剖析生物制药行业动态。 ❒ 创新药物临床试验受理聚焦生物制药领域，我们为您追踪行业动态，深入剖析热点话题，并提供一个交流与分享的平台，让您随时了解生物企业最新信息。 ❒ 盐酸曲恩汀片进展 Orphalan研发的盐酸曲恩汀片在2022年9月21日已顺利通过国家药品监督管理局药品审评中心（CDE）的上市许可申请受理环节。作为一种螯合剂，曲恩汀专为结合和清除体内铜而设计，是肝豆状核变性的有效治疗药物。自1982年获得FDA批准以来，该药已成为那些无法耐受青霉胺治疗患者的优选，其副作用也相对较轻。肝豆状核变性，又称威尔逊氏病，是一种常染色体隐性遗传病，由ATP7B基因突变引起体内铜离子转运障碍，导致铜在肝脏、神经系统等多处蓄积，引发一系列临床症状。发病者年龄跨度大，从婴儿到老年人都有报道。 ❒ OMTX705研究进展 OMTX705在临床前研究中显示出对多种癌症的显著疗效，包括肺癌、乳腺癌和肝癌等。其独特的作用机制和良好的安全性，使得OMTX705有望成为未来癌症治疗的新选择。 ❒ TQB2102药物特性 TQB2102注射用溶液，作为一款备受瞩目的候选药物，目前正处于临床研究的关键阶段。TQB2102是一种创新的氘代HER2双抗ADC，其独特之处在于能够特异性结合HER2表达细胞上的HER2的ECD4表位和ECD2表位。通过第一抗原结合片段（scFv）与第二抗原结合片段（Fab）的巧妙设计，TQB2102展现出了对HER2的高亲和力。此外，正大天晴药业集团通过采用氘代技术，成功规避了原研药的专利问题，为患者带来了更多的治疗选择。 ❒ LEO 152020临床试验据悉，LEO 152020是一款口服H4R拮抗剂，专为治疗特应性皮炎而设计。该药物的研究权在2018年8月由JW Pharmaceutical授权给LEO Pharma。多项研究显示，在健康成人中进行的单剂量和重复剂量研究均观察到良好的安全性和耐受性。目前，LEO 152020在美国和欧盟均处于II期临床阶段，正在进行一项随机、三盲、安慰剂对照、多中心的临床试验，旨在评估其治疗中度至重度AD成人的疗效。试验的主要终点是观察湿疹面积和严重程度指数（EASI）从基线到第16周的变化；同时，还将评估患者报告的症状，如瘙痒和失眠。 ❒ 南京征祥药物研究 ZX-4081是一种高选择性的PI3Kγ抑制剂，以其独特的抗肿瘤机理脱颖而出。它通过激活肿瘤微环境中的CD8+T细胞并促进巨噬细胞向M1（抗肿瘤）表型转化，从而有效抑制肿瘤的生长和转移。值得一提的是，PI3Kγ的抑制还能帮助克服免疫检查点抑制剂的耐药性。因此，当ZX-4081与PD-1/L1、CTLA4等免疫检查点抑制剂联合使用时，有望为那些对PD1和CTLA4类抑制剂产生耐药性的患者带来新的治疗希望。目前，ZX-4081在美国已进入临床1期，成为全球第二个进入此阶段的PI3Kγ抑制剂。 ❒ 重庆精准生物细胞制剂 C-13-60是一种旨在突破实体瘤微环境的CAR-T细胞制剂，其推测靶点为CEA。该制剂在官网数据中显示，其适应症广泛，涵盖结直肠癌、肾癌及食管癌。近期研究显示，CEA-CAR-T技术有望通过高压递送机制提升渗透性，进而增强抗肿瘤活性。 ❒ 标新生物GT919开发 GT919是一款靶向IKZF1/3的分子胶降解剂，专为恶性血液肿瘤治疗而设计。标新生物，这家成立于2020年2月的生物医药公司，专注于研发口服蛋白降解小分子药物。自2021年3月正式运营以来，公司已建立起包括人工智能虚拟筛选、体外药效筛选、药代动力学、蛋白质组学以及肿瘤动物药效模型在内的完备药物研发体系。其独特的分子胶降解剂（GLUE）和双机制降解剂（GLUETAC）开发平台，为公司带来了差异化的技术路线和发展战略。

100 项与四盐酸曲恩汀相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	A16AX	-

研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
肝豆状核变性	欧盟	Orphalan SA	2017-09-05
肝豆状核变性	冰岛	Orphalan SA	2017-09-05
肝豆状核变性	列支敦士登	Orphalan SA	2017-09-05
肝豆状核变性	挪威	Orphalan SA	2017-09-05

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06128954 (CTgov)	临床1期	肝豆状核变性	26	900mg TETA 4HCl Once Daily Formulation (Once Daily Dose Formulation (Treatment A))	壓衊範繭構築齋膚製簾(艱構鑰願獵醖願簾蓋構) = 廠獵觸獵襯繭簾窪鏇醖鬱憲襯製襯蓋築糧襯膚 (鑰襯糧選膚鑰壓顧醖窪, 衊繭鏇鏇餘築鹽遞築窪 ~ 範糧遞鑰齋窪壓餘淵艱) 更多	-	2025-08-15
				900mg TETA 4HCl Cuprior® (Cuprior® Comparator (Treatment B))	壓衊範繭構築齋膚製簾(艱構鑰願獵醖願簾蓋構) = 遞廠壓餘齋鹽簾衊醖糧鬱憲襯製襯蓋築糧襯膚 (鑰襯糧選膚鑰壓顧醖窪, 淵範鑰夢鬱襯膚鬱襯繭 ~ 觸艱憲觸獵網鹹鏇衊簾) 更多
NCT03539952 (CHELATE, EASL2022)	临床3期	维持 24-h urinary copper excretion \| alanine aminotransferase	76	D-penicillamine (DPA)	鬱選願憲壓醖鬱鬱醖繭(衊淵構製築鏇夢窪鑰鏇) = 範鹽衊壓鑰繭糧繭夢鏇觸窪顧鬱醖鬱願願衊積 (憲鑰範構遞艱蓋淵鏇餘 )	积极	2022-06-24
				trientine tetrahydrochloride	鬱選願憲壓醖鬱鬱醖繭(衊淵構製築鏇夢窪鑰鏇) = 壓餘膚糧夢獵製艱餘選觸窪顧鬱醖鬱願願衊積 (憲鑰範構遞艱蓋淵鏇餘 )
NCT03539952 (EASL2021)	临床3期	肝豆状核变性维持 non-ceruloplasmin copper;(NCC)	53	D-penicillamine	鹽鏇餘蓋襯鑰構窪蓋窪(鏇窪製願糧鏇繭構鹹壓) = 顧構廠鬱餘醖餘廠積襯顧壓範繭範壓衊醖選鹽 (廠餘繭鬱製積鏇齋鹽鑰 )	积极	2021-06-23
				TETA4	鹽鏇餘蓋襯鑰構窪蓋窪(鏇窪製願糧鏇繭構鹹壓) = 齋網衊憲窪顧簾簾衊膚顧壓範繭範壓衊醖選鹽 (廠餘繭鬱製積鏇齋鹽鑰 )