The overall aim of this project is to implement a non-invasive method of measuring quantitative regional cerebral blood flow (rCBF) on the UAB hybrid PET/MRI scanner to allow conducting such [O-15]water based scans with relative ease and safety in a large variety of important clinical and research applications. Participants will undergo imaging at baseline and after administration of a drug to increase cerebral blood flow to evaluate perfusion estimates during low and high flow states. The goal of this study is to generate data that will justify eliminating invasive arterial sampling in most [O-15]water-based PET protocols.

开始日期2025-02-01

申办/合作机构

The University of Alabama at Birmingham

CTRI/2024/09/074130 / Not yet recruiting临床2/3期IIT

A comparative clinical study of Punarnava Gokshur GhanVati and Acetazolamide Tablet in the management Of Vataj Adhimantha (Ocular Hypertension). - Nil

开始日期2024-10-01

申办/合作机构-

NCT06521476 / Not yet recruiting临床2期IIT

Impact of Acetazolamide on Central Sleep Apnea Patients Receiving Medication for Opioid Use Disorder

Patients with opioid use disorder treated with either methadone or buprenorphine are at risk of developing central sleep apnea (CSA) from these medications. Investigators will conduct a mechanistic trial using acetazolamide, a medicine known to improve CSA in other settings, to determine if acetazolamide can improve CSA due to medication for opioid use disorder and whether this leads to physiologic changes that might lead to reduced drug craving. Patients treated with medication for opioid use disorder and who have central sleep apnea will be randomized to treatment with acetazolamide or matching placebo for 7 days. At the end of the 7 days, they will undergo an overnight sleep study to assess the impact on breathing during sleep as well as sleep quality. In addition, measures of sympathetic tone, anxiety, arousal, cognition, and drug craving will be measured to determine if treatment of CSA with acetazolamide can produce physiologic changes that might contribute to improved health.

开始日期2024-10-01

申办/合作机构

University of Pittsburgh

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100 项与乙酰唑胺相关的临床结果

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100 项与乙酰唑胺相关的转化医学

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100 项与乙酰唑胺相关的专利（医药）

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项与乙酰唑胺相关的文献（医药）

2025-12-01·CHILDS NERVOUS SYSTEM

Cranial vault expansion in treatment of paediatric idiopathic intracranial hypertension

Article

作者: Cuthbert, Hadleigh ; Solanki, Guirish A ; Parida, Amitav ; Jagadeesan, Jagajeevan ; Rodrigues, Desiderio ; Afshari, Fardad T

INTRODUCTION:

Idiopathic intracranial hypertension (IIH) is a rare clinical entity in the paediatric population. Clinical presentation is mostly similar to adult counterpart and can include headaches, vomiting, papilloedema, deterioration in visual acuity or fields, and 6th cranial nerve palsy, leading to significant morbidity. Therapeutic lumbar puncture and medical treatment with acetazolamide are usually the first-line treatments. In a minority of refractory cases, particularly where visual function is threatened, CSF diversion has been traditionally used in clinical practice. CSF diversion in IIH patients can be associated with high rate of shunt malfunction and revision due to small ventricular size leading to repeated procedures. We describe our experience with cranial vault expansion as a method of treatment of paediatric IIH cases refractory to medical treatment as a CSF diversion sparing method.

METHODS:

Following review of IIH cases undergoing surgical treatment in our unit over 15 years (years 2009-2024), cases receiving cranial vault expansion as primary surgical treatment were selected and further analysed. Inclusion criteria were paediatric cases (age < 16) undergoing vault expansion as first surgical treatment for IIH refractory to pharmacological treatment for vision-threatening papilloedema. All cases were discussed within multidisciplinary meeting and selected following consideration of all management options.

RESULTS:

Over the studied period, we identified two cases of refractory IIH with severe papilloedema undergoing cranial vault expansion as primary shunt-sparing surgery. Both patients presented with symptoms of headaches, vomiting, and blurred vision with ophthalmological confirmation of severe papilloedema despite pharmacological therapy. Following multidisciplinary discussions, both underwent successful supratentorial vault expansion. Pharmacological treatment was discontinued in both cases following surgery. Evaluation at latest follow-up showed resolution of symptoms and papilloedema with no need for subsequent cerebrospinal fluid diversion at the latest follow-up.

CONCLUSION:

Cranial vault expansion is a viable and safe alternative surgical option in paediatric IIH cases refractory to medical treatment obviating the need for repeated shunt procedures. In cases with extremely small ventricles or where parental choice precludes CSF diversion, cranial vault expansion can be considered.

2025-03-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

Initial Results of the Paul Ahmed Comparison (PAC) Study in Refractory Childhood Glaucoma

Article

作者: Khaled, Omar M. ; Sayed, Mohamed S. ; Chauhan, Muhammad Z ; Chauhan, Muhammad Z. ; Elhusseiny, Abdelrahman M. ; Sayed, Mohamed S ; Shaarawy, Tarek ; Elhusseiny, Abdelrahman M ; Khaled, Omar M

PURPOSE:

To compare the effectiveness and safety of the Paul glaucoma implant (PGI) to the Ahmed glaucoma valve (AGV) in managing refractory childhood glaucoma.

DESIGN:

Randomized controlled trial.

SETTING:

Two clinical centers.

METHODS:

An ongoing randomized controlled trial including patients ≤ 18 years with refractory childhood glaucoma in whom glaucoma drainage implant surgery was planned. Patients were randomized to receive either PGI or AGV. The primary outcome was the intraocular pressure (IOP) reduction. Secondary outcomes included glaucoma medication reduction, success rate, and complications rate. The success rate was defined as achieving a postoperative IOP between 6-21 mmHg without or with glaucoma medications (up to three topical glaucoma medications), without the need for additional glaucoma surgeries, and without the occurrence of vision-threatening complications or evidence of disease progression. The use of oral glaucoma medications, such as acetazolamide, was considered a failure.

RESULTS:

The current report included 44 patients (44 eyes) who completed the one-year postoperative follow-up (25 in the PGI group versus 19 in the AGV group). The mean age at the time of surgery was 96.9 ± 59.1 months (90.5 ± 60.04 months in the PGI group and 105.4 ± 58.5 months in the AGV group). Preoperatively, the mean IOP was 32.6 ± 6.1 mmHg with a mean of 3.6 ± 0.6 glaucoma medications in the PGI group, compared with 29.8 ± 6.1 mmHg (P = .1) with a mean of 3.4 ± 0.7 glaucoma medications (P = .35) in the AGV group. At one year, there were no statistically significant differences in the mean IOP (14.9 ± 4.1 mmHg in the PGI group versus 15.5 ± 3.5 in the AGV group, P = .6) and number of glaucoma medications (1.1 ± 1 in the PGI group versus 1.6 ± 1.03 in the AGV group, P = .1). The success rate of PGI was 80% versus 73.6% in the AGV (P = .2). The postoperative complications rate was comparable in both groups (three eyes in each group).

CONCLUSION/RELEVANCE:

At one year postoperatively, the IOP reduction, reduction of glaucoma medications, success rates, and rate of complications were comparable between both groups.

2025-01-01·PLANT PHYSIOLOGY AND BIOCHEMISTRY

Multiple forms of carbonic anhydrase in Tetragonia tetragonioides leaves and the impact of heavy metal ions on enzyme activity

Article

作者: Onoiko, Olena ; Mykhaylenko, Natalia ; Topchiy, Nataliia ; Zolotareva, Elena

Tetragonia tetragonioides (Aizoaceae; Caryophyllales), an annual herbaceous plant, a halophyte native to the marine coasts of New Zealand is now cultivated worldwide as a minor salt-tolerant crop. The aim of this study was to evaluate the activity of carbonic anhydrase (CA, EC 4.2.1.1) and identify the CA isoforms in a fraction of total soluble proteins from T. tetragonioides leaves and to determine CA sensitivity to specific sulfonamide inhibitors and heavy metal (HM) ions. In higher plants, CA is one of the most abundant leaf enzymes catalyzing CO₂ and HCO₃^- interconversions that maintain the balance between dissolved forms of inorganic carbon. Catalytically active CA isoforms in the soluble protein fraction isolated from T. tetragonioides leaves were identified by a protonography method which involves the detection of CA activity in the gel following SDS-PAGE and subsequent removal of SDS (De Luca et al., 2015). This approach enabled the detection of active oligomeric forms of CA in the gel, allowing for the separate assessment of their activity. The protonogram revealed the presence of five sources of CA activity, which correspond to isoforms with approximate molecular masses of 26, 35, 41, 52, and 166 kDa. The total CA activity of T. tetragonioides leaf proteins was found to be inhibited by specific CA inhibitors, acetazolamide (AZ) and ethoxyzolamide (EZ), within the same concentration range as CAs from other plants. The half-maximal inhibition of hydratase activity was estimated to be 16 μM AZ and 2 μM EZ. The in vitro impact of selected heavy metal ions (Cu²⁺, Cd²⁺, Zn²⁺, Pb²⁺, Hg²⁺, and Ag⁺) on the hydratase activity of the soluble T. tetragonioides protein fraction was examined. High sensitivity of CA activity to inhibition by silver (I50-0.5 μM) and mercury ions (I₅₀ ∼ 2 μM) was shown. Copper and cadmium ions inhibited CA activity with I50 ∼ 40 and ∼12 μM, respectively. Zinc ions (I50 ∼ 200 μM) were weaker inhibitors, and lead ions in the concentration range of 50-500 μM insignificantly stimulated the hydratase activity of soluble proteins of T. tetragonioides. The high sensitivity of T. tetragonioides CA to Ag⁺ and Hg²⁺ ions, which are known to act as sulfhydryl poisons, can be attributed to the presence of SH-containing cysteines within the active site of the plant CAs.

项与乙酰唑胺相关的新闻（医药）

2024-12-05

·米内网

人福医药大爆发！18个新品获批，领跑5大百亿市场，20多款新药、4个首仿药在路上

精彩内容最近一周，人福医药喜讯“3连爆”：集团1款1类新药和2款首仿药新进2024版国家医保目录；12月3日，人福医药拿下18亿大品种熊去氧胆酸胶囊，今年集团已累计获批了18个新品；12月4日，备受市场瞩目的重组质粒-肝细胞生长因子注射液提交上市申请，集团将迎来首款生物药1类新药……近几年人福医药加快转型，在中国公立医疗机构终端已挺进5个百亿市场TOP20集团名单，目前有35个仿制药新品冲刺上市，20多款新药加速推进，未来可期。 3个重磅产品新进医保，今年已拿下18个新品 11月28日，万众瞩目的2024版国家医保目录正式公布，人福医药的1类新药注射用磷丙泊酚二钠，以及两个首仿药氯巴占片和盐酸氢吗啡酮缓释片顺利入保，集团成为了今年目录调整的大赢家之一。据悉，注射用磷丙泊酚二钠是全球唯一水溶性丙泊酚前体药物，对高脂血症患者具有优势，该产品是人福医药在2021年获批的1类新药。在中国城市公立医院、县级公立医院、城市社区中心及乡镇卫生院（简称中国公立医疗机构）终端，注射用磷丙泊酚二钠2023-2024上半年的销售额增长率分别为68.22%、398.91%。氯巴占片属于儿童罕见病用药，人福医药在2022年拿下国内首仿+首家过评。2023-2024上半年在中国公立医疗机构终端，人福医药的氯巴占片销售额增长率分别为1873.96%、18.32%，集团已成为该产品的领军企业。盐酸氢吗啡酮缓释片是国内唯一具有防滥用功能的24小时长效口服阿片类药物，人福医药在2023年拿下国内首仿+首家过评。本次顺利入保的3个重磅产品均为人福医药近几年获批的新品，纳入医保目录后将持续增加药品的市场可及性和销售潜力，并助力集团提升业绩。表1：人福医药2024年至今获批的新品（不含新适应症）来源：米内网中国申报进度（MED）数据库 12月3日，国家药监局发布最新药品获批信息，人福医药的熊去氧胆酸胶囊获批，该产品2023年在中国公立医疗机构终端的销售额超过18亿元。米内网数据显示，2024年至今人福医药已有18个新产品获批，包括了1个中成药和17个化学药。芍药甘草颗粒是中药3.1类新药，是人福医药首个按古代经典名方目录管理的中药复方制剂，同时集团也是该产品的首家获批企业，意义非凡。集团的酒石酸托特罗定缓释胶囊为国内首仿（同日获批），而恩扎卢胺软胶囊是集团首款获批的内分泌治疗用药，注射用福沙匹坦双葡甲胺是集团首款获批的止吐药和止恶心药。 2023年在中国公立医疗机构终端（化+生）市场，内分泌治疗用药的市场规模涨至190亿元以上，人福医药在2024年正式入局这个百亿市场，未来可期。领跑5大百亿市场，4个“光脚”产品抢攻第十批国采在中国公立医疗机构终端，人福医药在2023年成为了神经系统药物（化+生）的龙头集团，2024上半年继续保持领军优势，市场份额飙涨至9.46%，与TOP2集团拉开了超过2%的差距，一骑绝尘。图1：近几年人福医药的神经系统药物销售情况（单位：万元）来源：米内网中国公立医疗机构药品终端竞争格局人福医药在年报中提到，集团始终坚持“做医药细分市场领导者”的发展战略，目前集团的畅销产品分布在50多个亚类，2024上半年成功挤进了16个亚类市场的TOP20集团名单，成绩亮眼。 2023年中国公立医疗机构终端麻醉剂化药的销售规模超过200亿元，2024上半年人福医药以35亿元的销售额排在TOP1集团，市场份额升至34.84%。集团该类别超10亿产品有注射用盐酸瑞芬太尼、枸橼酸舒芬太尼注射液等，潜力产品注射用磷丙泊酚二钠2024上半年的销售额暴涨398.91%。 2023年中国公立医疗机构终端止痛药（化+生）的销售规模超过180亿元，2024上半年人福医药以7.8亿元的销售额排在TOP3集团，市场份额升至8.55%。集团该类别“亿级产品”有盐酸氢吗啡酮注射液、盐酸纳布啡注射液等，潜力产品盐酸羟考酮缓释片2024上半年销售额大涨95.42%。 2023年中国公立医疗机构终端精神安定药化药的销售规模超过150亿元，2024上半年人福医药以2.7亿元的销售额排在TOP7集团，市场份额升至3.58%。集团该类别“亿级产品”有咪达唑仑注射液、注射用苯磺酸瑞马唑仑等，潜力产品咪达唑仑口服溶液2024上半年销售额增长了55.44%。 2023年中国公立医疗机构终端中成药止咳祛痰平喘用药的销售规模超过160亿元，2024上半年人福医药排在TOP13集团，该类别畅销产品有寒喘祖帕颗粒、强力枇杷露等。 2023年中国公立医疗机构终端性激素及生殖系统调节剂（化+生）的销售规模超过100亿元，2024上半年人福医药排在TOP19集团，该类别畅销产品有米非司酮片、复方米非司酮片等。国家集中带量采购加速了市场洗牌，助力人福医药在细分市场提升竞争力。截至2024年12月4日，集团过评（含视同过评）的产品超过80个（按产品名统计），第二批至第九批国采已有14个产品成功中标。表2：人福医药国采中标产品情况来源：米内网中国上市药品（MID）数据库随着各批次国采陆续执行，人福医药多个中标产品的市场份额快速提升，2024上半年在中国公立医疗机构终端，人福医药已成为盐酸美金刚缓释胶囊的龙头企业，市场份额高达69.35%，集团也成为维生素B6片、布洛芬片、格列齐特缓释片的TOP2企业、成为盐酸克林霉素胶囊、米索前列醇片的TOP3企业。消息指，第十批国采将于本月12日开标，据米内网统计，人福医药将有“老产品”间苯三酚注射液以及近几年获批的新产品舒更葡糖钠注射液、枸橼酸托法替布缓释片、艾地骨化醇软胶囊、帕拉米韦注射液共5个产品迎战，“光脚”产品有望迎来新机遇。 20多款新药来势汹汹，35个仿制药加速冲刺 11月25日，人福医药再有中药新药获批临床，1.1类新药玉蚕颗粒功能主治为补脾益肾，活血利水，用于脾肾气虚、湿瘀阻络证糖尿病肾脏疾病蛋白尿的治疗。今年以来，人福医药已累计有5款新药获批临床，集团的新药矩阵越来越丰富。表3：人福医药今年以来获批临床的新药（不含新适应）来源：米内网中国申报进度（MED）数据库注射用RFUS-250是小分子阿片受体激动剂，临床拟用于治疗瘙痒和疼痛，HW071021片临床拟用于晚期实体瘤患者，HW211026软膏临床拟用于光化性角化病的治疗。司美格鲁肽注射液2023年在中国公立医疗机构终端的销售额超过40亿元，全球尚无化学合成的产品上市，潜力可期。表4：人福医药部分在研的重磅新药情况来源：米内网中国临床试验数据库 12月4日，CDE官网数据显示，武汉光谷人福生物医药提交了治疗用生物制品1类新药重组质粒-肝细胞生长因子注射液的上市申请，该新药拟用于治疗严重下肢缺血性疾病导致的肢体静息痛，有望成为人福医药首款获批的生物药1类新药。集团多款新药在2024年有了新进展：氨酚氢可酮缓释片在今年3月启动治疗中重度疼痛的III期临床，11月完成第一例入组。右美托咪定透皮贴剂、注射用RF16001、舒芬太尼透皮贴剂分别在今年5月、7月、8月完成II期临床。复方薏薢颗粒、RFUS-144注射液分别在今年2月、4月启动II期临床。HZ-J001乳膏、HW060015胶囊、注射用RFUS-250、司美格鲁肽注射液分别在今年3月、5月、9月、11月启动I期临床。表5：2022年至今人福医药报产在审的仿制药新品（不含新适应症）来源：米内网中国申报进度（MED）数据库 2022年至今，人福医药共有34个化药仿制药和1个中药仿制药报产在审，涉及12个大类（22个亚类），其中神经系统药物占了12个，包括了布洛氢可酮片、奥卡西平口服混悬液、利斯的明透皮贴剂、琥珀酸地文拉法辛缓释片、依托咪酯中/长链脂肪乳注射液、盐酸他喷他多片、马来酸咪达唑仑片、多巴丝肼片、盐酸羟考酮片、利丙双卡因乳膏、枸橼酸芬太尼口腔贴片、卡马西平缓释片(Ⅱ)，千亿市场龙头集团正持续发力，巩固领先地位。目前市场上暂无布洛氢可酮片、盐酸他喷他多片、萘普生钠软胶囊、枸橼酸芬太尼口腔贴片的国产仿制药获批，人福医药将争夺这4个产品的国内首仿。值得注意的是，早前人福医药暂无性功能障碍类药物上市，盐酸达泊西汀片或将为集团打开新市场大门。结语人福医药通过“聚焦、创新、国际化”三大战略路径不断增强可持续发展能力，目前集团的国际化进程稳步推进，今年以来顺利拿下了非诺贝特片、双氟泼尼酯眼用乳液、乙酰唑胺片、熊去氧胆酸片4个产品的ANDA文号，集团拥有的ANDA文号数量逐步向300个靠近，继续稳守制剂出海第一梯队。资料来源：米内网数据库、国家药监局官网、国家医保局官网、公司公告等注：米内网《中国公立医疗机构药品终端竞争格局》，统计范围是：中国城市公立医院、县级公立医院、城市社区中心以及乡镇卫生院，不含民营医院、私人诊所、村卫生室；上述销售额以产品在终端的平均零售价计算。数据统计截至12月4日，如有疏漏，欢迎指正。本文为原创稿件，转载请注明来源和作者，否则将追究侵权责任。投稿及报料请发邮件到872470254@qq.com稿件要求详询米内微信首页菜单栏商务及内容合作可联系QQ：412539092 【分享、点赞、在看】点一点不失联哦

上市批准一致性评价医药出海

2024-11-16

·丁香园

自发性低颅压诊疗专家共识（2024）发布

自发性低颅压（SIH）由脊髓脑脊液漏引起，以其特有的直立性头痛为主要表现，严重影响患者的生活质量，且误诊率高、检查和治疗手段开展不足。近日，中国医师协会神经内科医师分会和中国研究型医院学会头痛与感觉障碍专委会联合发布了《自发性低颅压诊疗专家共识（2024）》，为临床医师提供最新的诊疗指南。共识结合了最新的临床研究成果和临床实践情况，重点介绍了 SIH 的临床识别、检查流程、诊断标准和鉴别诊断、干预性治疗和并发症的处理，共计制订了 30 条推荐意见：滑动可查看完整内容 ↓ 推荐意见 1：当临床表现为头痛，且坐立位 2h 内头痛出现或加重，平躺后 1h 内头痛消失或减轻时，应当考虑直立性头痛。（2D）推荐意见 2：当临床表现为直立性头痛，或病程中头痛症状曾出现过典型的直立性特点时，应当考虑 SIH 的可能性。（1B）推荐意见 3：当直立性头痛伴随颈部僵硬、前庭耳蜗症状、恶心呕吐等症状时，更支持 SIH 的诊断。（2C）推荐意见 4：咳嗽或做 Valsalva 动作时头痛加重，应考虑存在 SIH 的可能性。（2C）推荐意见 5：当缺乏典型直立性头痛时，应综合考虑，不能完全排除 SIH 的诊断。（1C）推荐意见 6：推荐至少使用一种量表评估 SIH 疼痛症状严重程度和对生活质量的影响。（1D）推荐意见 7：急诊就诊时，推荐进行头颅 CT 检查排除蛛网膜下腔出血等其他疾病，并对硬膜下血肿进行评估。（1D）推荐意见 8：推荐头颅 MRI 平扫和增强扫描作为 SIH 的一线检查。（1B）推荐意见 9：重点评估硬脑膜强化、静脉窦扩张、鞍上池变窄、硬膜下积液/血肿、桥前池变窄和乳头体脑桥间距变窄 6 个指标，推荐使用伯尔尼评分预测CSF漏。（1B）推荐意见 10：当临床症状符合 SIH，但一次头颅 MRI 扫描缺乏 SIH 相关征象时，不能除外 SIH，建议动态复查。（2C）推荐意见 11：推荐脊髓 MRI 扫描作为 SIH 的一线检查，扫描序列应包含 T2 加权序列（横轴位和矢状位）、T2 加权压脂序列［横轴位和（或）矢状位］和三维高分辨率重 T2加权脂肪抑制脊髓成像；重点评估有无 SLEC。（1B）推荐意见 12：推荐优先采用动态脊髓造影定位 CSF 漏口，包括动态 CTM、DSM 或 CDM。（1C）推荐意见 13：CSF 压力正常不能排除SIH的诊断，不推荐常规进行腰椎穿刺测 CSF 压力诊断 SIH；但为排除其他疾病而行腰椎穿刺时，应测量 CSF 压力。（1B）推荐意见 14：推荐采用以下诊断标准进行诊断（表 2 ）。（1D）推荐意见 15：新发病的 SIH 患者建议首先采用保守治疗2周，包括卧床、口服或静脉补液、避免 Valsalva 动作。（1D）推荐意见 16：不推荐常规使用糖皮质激素。（2D）推荐意见 17：保守治疗 2 周症状仍然无法缓解，排除禁忌证后，推荐行EBP治疗；有条件的医疗机构优先行靶向EBP治疗。（1D）推荐意见 18：如初次 EBP 治疗无效或效果不持续，可考虑于 2～4 周后重复 EBP 治疗。（2D）推荐意见 19：CVF 可考虑 CT 引导下纤维蛋白胶注射治疗。（2C）推荐意见 20：当经过保守治疗和多次靶向 EBP 治疗后，仍然有严重的头痛或严重的颅神经症状或具有高危的硬膜下血肿等并发症时，可以考虑外科手术治疗。（1C）推荐意见 21：CVF 患者保守治疗无效时，可直接考虑外科手术治疗。（1C）推荐意见 22：具体外科手术的方式应结合 CSF 漏的类型和位置、操作的可行性和风险性，以及患者的偏好而决定。（1D）推荐意见 23：SIH 患者在术前应充分告知术后RIH的可能；若术后发生 RIH 可予以严密观察，暂不给予特殊药物处理；必要时复查头和脊柱 CT，排查自体血蛛网膜下腔内弥散。（1D）推荐意见 24：当 RIH 明显并且持续时间较长时，可考虑尝试乙酰唑胺、托吡酯和利尿剂治疗，必要时需进一步检查排除其他头痛病因。（2D）推荐意见 25：当 SIH 出现体积小且无症状的硬膜下血肿时，建议保守治疗硬膜下血肿，同时靶向治疗 CSF 漏。（1B）推荐意见 26：当 SIH 出现无症状的最大厚度超过 1cm的硬膜下血肿时，建议积极靶向治疗 CSF 漏，并严密观察病情；如出现意识障碍，则急诊行血肿清除术，并联合靶向治疗 CSF 漏。（1C）推荐意见 27：当 SIH 出现局灶性神经功能障碍、癫痫发作或头痛模式发生改变时，推荐行头颅 CT 或磁共振静脉血管成像排查 CVT。（2C）推荐意见 28：当 SIH 并发 CVT 时，应积极给予靶向 EBP 治疗；是否启动抗凝，应当谨慎评估出血风险。（2C）推荐意见 29：推荐与患者充分沟通慢性 CSF漏潜在的长期风险，并充分结合患者意愿选择保守观察、EBP 或手术治疗 CSF 漏；如果患者选择保守观察，建议每 1～2 年复查一次头 MRI 和脊髓 MRI（具体所含序列参考前文）。（2C）推荐意见 30：经过治疗后临床症状明显缓解的患者，建议随访 3～6 个月；临床症状无明显缓解或在随访期复发的患者，建议及时复查和治疗。（1D）最新推荐诊疗流程插图、内容来源：自发性低颅压诊疗专家共识（2024）策划：云也｜监制：z_popeye 👇👇👇 点击「阅读原文」查看共识原文

临床研究

2024-09-03

·Business Wire

Obsidian Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for OBX-115 for the Treatment of Advanced Melanoma

CAMBRIDGE, Mass.--( BUSINESS WIRE )-- Obsidian Therapeutics, Inc. , a clinical-stage biotechnology company pioneering engineered cell and gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OBX-115 for the treatment of patients with unresectable or metastatic melanoma that is resistant to immune checkpoint inhibitor (ICI) therapy. OBX-115 is a novel engineered tumor-derived autologous T cell immunotherapy (tumor-infiltrating lymphocyte [TIL] cell therapy) armored with membrane-bound IL15 (mbIL15) that is pharmacologically regulated using FDA-approved small molecule drug acetazolamide. This second FDA designation follows a July 2024 announcement of Fast Track designation for OBX-115. “RMAT designation for OBX-115 highlights the unmet need in ICI-resistant melanoma and OBX-115’s potential to provide patient-centric, transformative care in this setting,” said Parameswaran Hari, M.D., Chief Development Officer of Obsidian. “In an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, initial Phase 1 data showed that OBX-115 exhibited a favorable safety profile relative to what has been observed historically from IL2-dependent non-engineered TIL cell therapy, encouraging efficacy in patients with heavily pre-treated disease, and compatibility with non-surgical core needle biopsy tumor tissue procurement . We look forward to continued collaborative interaction with the FDA as we advance OBX-115 clinical development in both melanoma and non-small cell lung cancer (NSCLC).” Established under the 21st Century Cures Act, RMAT designation is a dedicated program that includes the benefits of Fast Track and Breakthrough Therapy designations and is designed to expedite the development and review processes for certain promising drug candidates, including cell therapies. An investigational cell therapy is eligible for RMAT designation if it meets the definition of regenerative medicine therapy, is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for that disease. RMAT designation provides early and frequent interactions with the FDA, including intensive guidance on efficient drug development. Candidates receiving RMAT designation may also be eligible for Accelerated Approval and Priority Review. About OBX-115 Obsidian’s lead investigational cytoTIL15™ program, OBX-115, is a novel engineered tumor-derived autologous T cell immunotherapy (tumor-infiltrating lymphocyte [TIL] cell therapy) armored with pharmacologically regulatable membrane-bound IL15 (mbIL15). OBX-115 has the potential to become a meaningful therapeutic option for patients with advanced or metastatic melanoma and other solid tumors by leveraging the expected benefits of mbIL15 and Obsidian’s proprietary, differentiated manufacturing process to enhance persistence, antitumor activity, and clinical safety of TIL cell therapy. Obsidian is investigating OBX-115 in a multicenter trial in patients with advanced melanoma or relapsed / refractory NSCLC ( NCT06060613 ). About Obsidian Therapeutics Obsidian Therapeutics, Inc. is a clinical-stage biotechnology company pioneering engineered cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian’s proprietary cytoDRiVE® technology is designed to precisely regulate the timing and level of protein function by using FDA-approved small-molecule drugs. Obsidian is headquartered in Cambridge, MA. The Company has collaborations with Bristol Myers Squibb and Vertex Pharmaceuticals. For more information, please visit www.obsidiantx.com and follow us on LinkedIn.

细胞疗法免疫疗法快速通道临床1期ASCO会议

100 项与乙酰唑胺相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D00218	乙酰唑胺	S01EC01	DB00819

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
先兆子痫	日本	Sanwa Kagaku Kenkyusho Co., Ltd.	1994-06-02
睡眠呼吸暂停综合征	日本	Sanwa Kagaku Kenkyusho Co., Ltd.	1988-03-29
高眼压症	中国	天津力生制药股份有限公司	1983-01-01
呼吸性酸中毒	日本	Sanwa Kagaku Kenkyusho Co., Ltd.	1955-03-29
青光眼	日本	Sanwa Kagaku Kenkyusho Co., Ltd.	1955-03-29
梅尼埃病	日本	Sanwa Kagaku Kenkyusho Co., Ltd.	1955-03-29
经前综合征	日本	Sanwa Kagaku Kenkyusho Co., Ltd.	1955-03-29
内分泌与代谢疾病	美国	Teva Women's Health, Inc.	1954-06-25
急性闭角型青光眼	美国	Teva Branded Pharmaceutical Products R&D, Inc.	1953-07-27
高原反应	美国	Teva Branded Pharmaceutical Products R&D, Inc.	1953-07-27
水肿	美国	Teva Branded Pharmaceutical Products R&D, Inc.	1953-07-27
癫痫	美国	Teva Branded Pharmaceutical Products R&D, Inc.	1953-07-27
开角型青光眼	美国	Teva Branded Pharmaceutical Products R&D, Inc.	1953-07-27
继发性青光眼	美国	Teva Branded Pharmaceutical Products R&D, Inc.	1953-07-27

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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


NCT04639193 (RESCUE-Combo, Pubmed \| Ann Am Thorac Soc)	临床2期	阻塞性睡眠呼吸暂停综合征	20	Acetazolamide+eszopiclone	顧齋艱糧窪衊齋憲觸簾(積淵築衊壓積憲繭鏇選) = 廠觸鹽選積網繭鑰願鑰積憲壓鬱繭襯淵繭廠鑰 (艱淵繭窪鹽壓顧遞膚鏇, -24.1 ~ -5.2)	积极	2024-11-08
NCT03505788 (ADVOR Trial \| Advor, CTgov)	临床4期	音量过载 \| 代谢性疾病 \| 急性失代偿性心力衰竭	519	placebo (Placebo)	艱願築觸蓋鏇築築網憲(憲鹽獵齋鑰鏇簾糧網糧) = 膚憲鹹糧鏇艱繭願壓糧淵餘觸鏇願淵窪積壓壓 (蓋獵衊蓋艱製壓餘構壓, 鏇鬱鹽願齋廠鹹艱淵製 ~ 艱蓋襯壓齋構簾築壓夢) 更多	-	2024-10-28
				Acetazolamide (Acetazolamide)	艱願築觸蓋鏇築築網憲(憲鹽獵齋鑰鏇簾糧網糧) = 艱醖窪願醖選廠鑰遞鬱淵餘觸鏇願淵窪積壓壓 (蓋獵衊蓋艱製壓餘構壓, 艱鬱積蓋鹹餘築膚觸餘 ~ 繭顧積廠鹹鹹鑰衊鹽築) 更多
NCT05473364 (Acetazolamide in Persons with Type 1 Diabetes, Pubmed \| J Am Soc Nephrol)	临床1期	1型糖尿病	12	Acetazolamide 62.5 mg	衊積網築鏇淵廠淵積憲(選淵憲繭願製淵鹽襯鑰) = 夢窪壓廠簾製夢網膚範構夢廠繭簾遞齋鹹鏇夢 (壓獵構簾積壓構製願範 ) 更多	积极	2024-10-08
				Acetazolamide 125 mg	衊積網築鏇淵廠淵積憲(選淵憲繭願製淵鹽襯鑰) = 鏇網簾襯網獵繭壓壓鹽構夢廠繭簾遞齋鹹鏇夢 (壓獵構簾積壓構製願範 ) 更多
NCT03505788 (ADVOR Trial, Pubmed \| Circ Heart Fail)	临床4期	音量过载 \| 心脏衰竭追加 serum chloride	519	Acetazolamide	廠鹹窪醖鬱齋繭鏇壓窪(顧範鹽鏇襯餘簾鑰網網) = Hypochloremia is associated with diuretic resistance and worse clinical outcomes 鏇繭積積齋範襯網築齋 (壓範繭壓廠襯鹹構顧醖 )	积极	2024-10-01
				Placebo
EURETINA2024 人工标引	N/A	黄斑水肿	7	acetazolamidement (acetazolamide and localized anti-inflammatory agents)	積遞壓顧簾簾憲選衊廠(淵夢鏇鹹襯獵憲鑰製艱) = 廠膚遞蓋醖醖鹹蓋廠醖鬱鏇選觸窪窪衊窪膚構 (憲簾鹽鑰獵衊廠淵顧製 )	积极	2024-09-19
				Anti-vascular endothelial growth factor (anti-VEGF) injections	積遞壓顧簾簾憲選衊廠(淵夢鏇鹹襯獵憲鑰製艱) = 憲繭獵糧構齋鹽簾壓簾鬱鏇選觸窪窪衊窪膚構 (憲簾鹽鑰獵衊廠淵顧製 )
ESC2024	N/A	急性失代偿性心力衰竭	-	Acetazolamide plus standard of care (SOC)	繭顧壓範醖觸鹹鏇壓築(簾願築遞簾鹹網鹹餘範): RR = 1.36 (95% CI, 1.1 ~ 1.68), P-Value = 0.005	-	2024-09-02
				Standard of care (SOC) alone
ESC2024	N/A	急性失代偿性心力衰竭	-	Acetazolamide-use	衊蓋齋願範衊繭憲築積(膚淵醖鹹遞膚獵醖願餘) = 壓範簾醖鬱醖積壓觸繭艱選鹹遞製夢顧獵製鏇 (齋膚醖糧淵願襯遞鑰淵 ) 更多	-	2024-08-31
				acetazolamide (No-use)	衊蓋齋願範衊繭憲築積(膚淵醖鹹遞膚獵醖願餘) = 膚憲膚淵鹹顧獵艱鬱齋艱選鹹遞製夢顧獵製鏇 (齋膚醖糧淵願襯遞鑰淵 ) 更多
ESC2024	N/A	急性充血性心力衰竭	-	Acetazolamide adjunctive therapy	憲鬱壓糧醖觸壓餘選鹹(鏇範選襯衊構憲觸積積) = 窪膚製夢構築選醖網鬱簾顧淵艱餘觸遞繭鏇簾 (鬱膚鹹觸構築製選鹽艱, 45.10 ~ 115.51) 更多	-	2024-08-31
ADVOR (ESC2024)	N/A	音量过载 \| 心脏衰竭追加 serum chloride	519	Acetazolamide	願艱餘糧窪淵構憲鹹糧(淵鏇蓋膚鹹遞鑰醖網顧) = 鬱襯窪淵遞鬱願廠艱築獵襯餘顧糧製齋範憲壓 (觸蓋觸窪齋選選願構積 ) 更多	积极	2024-05-14
				Placebo	願艱餘糧窪淵構憲鹹糧(淵鏇蓋膚鹹遞鑰醖網顧) = 衊窪鹽鏇選襯鑰獵淵淵獵襯餘顧糧製齋範憲壓 (觸蓋觸窪齋選選願構積 ) 更多
NCT04639193 (RESCUE-Combo, CTgov)	临床2期	阻塞性睡眠呼吸暂停综合征	20	Acetazolamide+Eszopiclone (Dual-Therapy)	鏇構衊鬱遞廠觸夢壓遞(鹹顧鹽選憲齋積膚鑰製) = 齋膚鏇獵淵壓願廠簾簾繭鹹鑰網憲淵構築憲壓 (鑰蓋鹹夢鑰餘積夢襯鏇, 鑰淵繭鏇願窪選選製遞 ~ 鏇糧鬱觸壓鑰鬱憲鏇鏇) 更多	-	2024-05-02
				Placebo+Acetazolamide+Eszopiclone (Placebo)	鏇構衊鬱遞廠觸夢壓遞(鹹顧鹽選憲齋積膚鑰製) = 衊餘膚觸網糧範鏇鹹鹹繭鹹鑰網憲淵構築憲壓 (鑰蓋鹹夢鑰餘積夢襯鏇, 糧遞襯繭構繭積簾選壓 ~ 鹽蓋鏇顧構衊構繭簾糧) 更多