| Item type |
文献 / Documents(1) |
| 公開日 |
2024-02-01 |
| アクセス権 |
|
|
アクセス権 |
open access |
| 資源タイプ |
|
|
資源タイプ識別子 |
http://purl.org/coar/resource_type/c_6501 |
|
資源タイプ |
journal article |
| 出版社版DOI |
|
|
|
識別子タイプ |
DOI |
|
|
関連識別子 |
https://doi.org/10.2196/42032 |
|
|
言語 |
ja |
|
|
関連名称 |
10.2196/42032 |
| 出版タイプ |
|
|
出版タイプ |
VoR |
|
出版タイプResource |
http://purl.org/coar/version/c_970fb48d4fbd8a85 |
| タイトル |
|
|
タイトル |
An Exploratory Trial of EPI-589 in Amyotrophic Lateral Sclerosis (EPIC-ALS) : Protocol for a Multicenter, Open-Labeled, 24-Week, Single-Group Study |
|
言語 |
en |
| 著者 |
土師, 正太郎
藤田, 浩司
オキ, リョウスケ
大崎, 裕亮
宮本, 亮介
森野, 豊之
ナガノ, セイイチ
アツタ, ナオキ
金澤, 裕樹
松元, 友暉
アリサワ, アツコ
カワイ, ヒサシ
サトウ, ヤスタカ
坂口, 暁
八木, 健太
ハマタニ, タツト
カギムラ, タツオ
楊河, 宏章
モチヅキ, ヒデキ
ドウユウ, マナブ
ソブエ, ゲン
原田, 雅史
和泉, 唯信
|
| 抄録 |
|
|
内容記述タイプ |
Abstract |
|
内容記述 |
Background: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder, with its currently approved drugs, including riluzole and edaravone, showing limited therapeutic effects. Therefore, safe and effective drugs are urgently necessary. EPI-589 is an orally available, small-molecule, novel redox-active agent characterized by highly potent protective effects against oxidative stress with high blood-brain barrier permeability. Given the apparent oxidative stress and mitochondrial dysfunction involvement in the pathogenesis of ALS, EPI-589 may hold promise as a therapeutic agent.
Objective: This protocol aims to describe the design and rationale for the EPI-589 Early Phase 2 Investigator-Initiated Clinical Trial for ALS (EPIC-ALS).
Methods: EPIC-ALS is an explorative, open-labeled, single-arm trial that evaluates the safety and tolerability of EPI-589 in patients with ALS. This trial consists of 12-week run-in, 24-week treatment, and 4-week follow-up periods. Patients will receive 500 mg of EPI-589 3 times daily over the 24-week treatment period. Clinical assessments include the mean monthly change of Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised total score. The biomarkers are selected to analyze the effect on oxidative stress and neuronal damage. The plasma biomarkers are 8-hydroxy-2′-deoxyguanosine (8-OHdG), 3-nitrotyrosine (3-NT), neurofilament light chain (NfL), phosphorylated neurofilament heavy chain (pNfH), homocysteine, and creatinine. The cerebrospinal fluid biomarkers are 8-OHdG, 3-NT, NfL, pNfH, and ornithine. The magnetic resonance biomarkers are fractional anisotropy in the corticospinal tract and N-acetylaspartate in the primary motor area.
Results: This trial began data collection in September 2021 and is expected to be completed in October 2023.
Conclusions: This study can provide useful data to understand the characteristics of EPI-589. |
|
言語 |
en |
| キーワード |
|
|
言語 |
en |
|
主題Scheme |
Other |
|
主題 |
amyotrophic lateral sclerosis |
| キーワード |
|
|
言語 |
en |
|
主題Scheme |
Other |
|
主題 |
biomarker |
| キーワード |
|
|
言語 |
en |
|
主題Scheme |
Other |
|
主題 |
clinical trial |
| キーワード |
|
|
言語 |
en |
|
主題Scheme |
Other |
|
主題 |
magnetic resonance imaging |
| キーワード |
|
|
言語 |
en |
|
主題Scheme |
Other |
|
主題 |
oxidative stress |
| 書誌情報 |
en : JMIR Research Protocols
巻 12,
p. e42032,
発行日 2023-01-30
|
| 収録物ID |
|
|
収録物識別子タイプ |
ISSN |
|
収録物識別子 |
19290748 |
| 出版者 |
|
|
出版者 |
JMIR Publications |
|
言語 |
en |
| 権利情報 |
|
|
言語 |
en |
|
権利情報 |
This is an open-access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work, first published in JMIR Research Protocols, is properly cited. The complete bibliographic information, a link to the original publication on https://www.researchprotocols.org, as well as this copyright and license information must be included. |
| EID |
|
|
識別子 |
397652 |
|
識別子タイプ |
URI |
| 言語 |
|
|
言語 |
eng |
JMIRRP_12_e42032.pdf (281 KB)
.png)
