更新于：2024-11-01

TTR x 淀粉样蛋白轻链

更新于：2024-11-01

基本信息

关联

项与 TTR x 淀粉样蛋白轻链相关的药物

NPT-189

靶点

淀粉样蛋白轻链 x TTR

作用机制

TTR抑制剂 [+1]

在研机构

Proclara Biosciences, Inc.

原研机构

Proclara Biosciences, Inc.

在研适应症

转甲状腺素运载蛋白淀粉样变性 [+1]

非在研适应症-

最高研发阶段临床1期

首次获批国家/地区-

首次获批日期1800-01-20

项与 TTR x 淀粉样蛋白轻链相关的临床试验

NCT03879278 / Terminated临床1期

A Phase 1 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of NPT189 in Healthy Subjects

This will be a Phase 1, randomized, double-blind, single center, placebo-controlled, multiple ascending dose (MAD) study in a maximum of 3 cohorts of 8 healthy male and female subjects each. Subjects in Cohorts 1, 2 and 3 will receive ascending multiple IV doses of NPT189 or matching placebo.

开始日期2019-03-06

申办/合作机构

Proclara Biosciences, Inc.

NL-OMON48151 / CompletedN/A

A phase I randomized, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, tolerability and pharmacokinetics of NPT189 in healthy subjects - Study to evaluate the safety, tolerability and PK of NPT189

开始日期2019-02-12

申办/合作机构

Proclara Biosciences, Inc.

NCT03610035 / Completed临床1期

A Phase 1 Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of NPT189 in Healthy Subjects

The purpose of the study is to evaluate the safety and tolerability of single dose of NPT189 in healthy volunteers. The study will also evaluate the pharmacokinetic characteristics of NPT189.

开始日期2018-07-24

申办/合作机构

Proclara Biosciences, Inc.

100 项与 TTR x 淀粉样蛋白轻链相关的临床结果

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100 项与 TTR x 淀粉样蛋白轻链相关的转化医学

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0 项与 TTR x 淀粉样蛋白轻链相关的专利（医药）

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项与 TTR x 淀粉样蛋白轻链相关的文献（医药）

2024-04-01·Human Pathology

Morphological findings in different subtypes of hepatic amyloid

Article

作者: Yasir, Saba ; Zhang, Lizhi ; Chen, Zongming Eric ; Torbenson, Michael ; Hartley, Chris

The classic findings have been well described for light-chain amyloid involving the liver. In addition to light chain, however, many additional proteins are now known to be amyloidogenic and can involve the liver. A total of 58 surgical pathology specimens with amyloid deposits were analyzed for patterns of amyloid deposition, including amyloid from light chain lambda (N = 17), light chain kappa (N = 15), transthyretin (N = 15), serum amyloid A (N = 4), apolipoprotein A1 (N = 4), fibrinogen alpha (N = 2), LECT2 (N = 1). Amyloid deposits predominately targeted the liver vasculature, including the walls of the hepatic arteries, portal veins, and sinusoids. While there was overlap, light chain amyloid predominately involved the sinusoids, while transthyretin amyloid predominately targeted the hepatic arteries, especially the larger ones in the hilum and larger portal tracts. Serum amyloid A formed nodular deposits that started in the portal vasculature but then extended into the portal tract stroma, leading to large, bulbous, portal-based amyloid deposits. Apolipoprotein A amyloid also formed large portal-based nodules. Fibrinogen was mild and subtle on H&E and predominately affected portal veins. Amyloid deposits in hilar nerves were prominent with amyloid light chain, transthyretin, and apolipoprotein A1. In conclusion, the histology of hepatic amyloid is diverse and shows several distinct clusters of findings that can aide in recognition in surgical pathology specimens.

2023-06-01·Journal of nuclear medicine technology

Clinical Application of ^99mTc-Pyrophosphate Scintigraphy for Diagnosis of Cardiac Amyloidosis: A Case Series.

Article

作者: Shoura, Sami ; Malhotra, Saurabh

Cardiac amyloidosis (CA) is an infiltrative cardiomyopathy resulting from deposition of insoluble amyloid protein in the myocardial interstitium. The accumulation of amyloid protein causes the myocardium to thicken and stiffen, leading to diastolic dysfunction and, eventually, heart failure. Two primary types of amyloidosis-transthyretin and immunoglobulin light chain-account for nearly 95% of all CA diagnoses. Three case studies are presented. The first demonstrates a patient positive for transthyretin amyloidosis, the second demonstrates a patient positive for light-chain CA, and the third demonstrates a patient showing blood-pool uptake on the [^99mTc]Tc-pyrophosphate scan but negative for CA.

2023-01-01·Mayo Clinic Proceedings

Real-Life Evaluation of an Algorithm for the Diagnosis of Cardiac Amyloidosis

Article

作者: Deux, Jean-François ; Zaroui, Amira ; Funalot, Benoit ; Kharoubi, Mounira ; Bézard, Mélanie ; Le Bras, Fabien ; Teiger, Emmanuel ; Molinier-Frenkel, Valérie ; Damy, Thibaud ; Chalard, Coraline ; Lemonnier, François ; Moktefi, Anissa ; Itti, Emmanuel ; Guendouz, Soulef ; Oghina, Silvia ; Fanen, Pascale ; Galat, Arnault ; Hittinger, Luc ; Abulizi, Mukedaisi ; Poullot, Elsa

OBJECTIVETo evaluate the real-life use of a modified Gillmore algorithm with a "one-stop-shop" approach, bone scintigraphy (BS), a monoclonal gammopathy test (GT), a salivary gland biopsy (SGB), and genetic testing performed at the same time for the diagnosis of cardiac amyloidosis at the French National Reference Centre for Cardiac Amyloidosis (Henri Mondor Hospital, Créteil, France).METHODSThis retrospective cohort study included a total of 1222 patients with suspected amyloidosis who underwent BS and GT between June 2008 and May 2019.RESULTSOf 1222 patients, 349 had no cardiac uptake on BS and negative GT (BS-/GT-), 276 were BS-/GT positive (GT+), 420 patients were BS+/GT-, and 177 were BS+/GT+. Our one-stop-shop check-up enabled us to diagnose 892 (72.9%) patients; 330 (27.0%) patients required additional examinations, such as mass spectrometry and/or a cardiac biopsy. This subset notably included 112 patients with amyloid light chain amyloidosis. More than 64% of the patients with transthyretin amyloidosis or another type of amyloidosis were diagnosed during the one-stop shop visit. Sensitivity and specificity of BS for transthyretin amyloidosis diagnosis was 99% and 96%, respectively. For amyloid light chain diagnosis, sensitivity and specificity were 100% and 76%, respectively, for GT and 54% and 100%, respectively, for SGB. Of 910 transthyretin genetic tests, 205 (17%) detected mutations.CONCLUSIONThe results of our real-life cohort study confirmed the ability of a one-stop-shop approach with a modified Gillmore algorithm to diagnose cardiac amyloidosis and the interest of simultaneous testing for earlier diagnosis. The SGB has diagnostic value because it is easy, quick, and less invasive than a cardiac biopsy.