Iltamiocel (Cook MyoSite)(Iltamiocel (Cook MyoSite)) - 在研适应症：大便失禁，压力性尿失禁，吞咽障碍_专利_临床

概要

基本信息

药物类型

干细胞疗法

别名

AMDC - Cook MyoSite、AMDC-USR、Autologous muscle derived cells - Cook MyoSite

+ [1]

靶点-

作用方式-

作用机制

细胞替代物

治疗领域

消化系统疾病泌尿生殖系统疾病肿瘤+ [3]

在研适应症

大便失禁压力性尿失禁吞咽障碍+ [1]

非在研适应症-

原研机构

Cook MyoSite, Inc.

在研机构

Cook MyoSite, Inc.

非在研机构-

最高研发阶段临床3期

首次获批日期-

最高研发阶段(中国)-

特殊审评-

登录后查看时间轴

关联

12

项与 Iltamiocel (Cook MyoSite) 相关的临床试验

NCT03997318

/ Withdrawn临床3期

JPN1: A Confirmatory, Bridging, Double-Blind, Randomized, Controlled Trial in Japan Comparing the Safety and Efficacy of AMDC-USR With Placebo in Female Subjects With Stress Urinary Incontinence

This is a confirmatory/bridging study to evaluate the efficacy and safety of Autologous Muscle Derived Cells for Urinary Sphincter Repair (AMDC-USR) compared with placebo in the treatment of stress urinary incontinence (SUI) in adult Japanese female subjects. Half of the participants will receive AMDC-USR (injections with cells) and the other half will receive placebo.

开始日期2025-06-01

申办/合作机构

Cook MyoSite, Inc.

NCT06743620

/ Not yet recruitingN/A

Prospective Observational Long-Term Safety and Effectiveness Registry of Patients Who Have Received Iltamiocel as Part of Cook Myosite, Inc. Sponsored CELLEBRATE Clinical Study in Patients With Persistent or Recurrent Stress Urinary Incontinence (SUI) Following Surgical Treatment

The main purpose of this registry is to collect observational, long-term safety and effectiveness data in subjects who have received at least one dose of iltamiocel as part of the CLBT clinical study.

开始日期2025-03-01

申办/合作机构

Cook MyoSite, Inc.

NCT05776277

/ Recruiting临床3期

A Two-stage, Randomized, Controlled Trial Comparing the Safety and Efficacy of Iltamiocel With Placebo in the Treatment of Female Participants With Chronic Fecal Incontinence and a History of Obstetric Anal Sphincter Injury (DigniFI)

This study evaluates the efficacy and safety of a single injection of iltamiocel (300 x 10^6 cells) compared to a placebo in the reduction of fecal incontinence episode frequency in adult female participants with chronic fecal incontinence and a history of obstetric anal sphincter injury. Half of the participants will receive iltamiocel (injections with cells) and the other half will receive placebo.

开始日期2024-03-21

申办/合作机构

Cook MyoSite, Inc.

100 项与 Iltamiocel (Cook MyoSite) 相关的临床结果

登录后查看更多信息

100 项与 Iltamiocel (Cook MyoSite) 相关的转化医学

登录后查看更多信息

100 项与 Iltamiocel (Cook MyoSite) 相关的专利（医药）

登录后查看更多信息

15

项与 Iltamiocel (Cook MyoSite) 相关的文献（医药）

2024-11-01·Neurourology and Urodynamics

Iltamiocel Autologous Cell Therapy for the Treatment of Female Stress Urinary Incontinence: A Double‐Blind, Randomized, Stratified, Placebo‐Controlled Trial

Article

作者: Antosh, Danielle ; Peters, Kenneth M. ; Marguet, Charles G. ; Lee, Una J. ; Tarnay, Christopher M. ; Kaufman, Melissa R. ; Snyder, Scott ; Heit, Michael H. ; Goldman, Howard B. ; Dmochowski, Roger ; Benson, Kevin D. ; Rehme, Christian ; Sokol, Eric R. ; Kennelly, Michael J. ; Canestrari, Emanuele ; Quiroz, Lieschen H. ; Chancellor, Michael B. ; Wolter, Christopher E. ; Jankowski, Ron J. ; Karram, Mickey ; Bennett, Jason B. ; Chermansky, Christopher J. ; Katz, Daniel M. ; Thomas, Sherry

ABSTRACTAimsThis study aimed to determine the efficacy and safety of iltamiocel investigational autologous muscle cell therapy in females with stress urinary incontinence (SUI).MethodsAdult females were randomized 2:1 to iltamiocel (150 × 106 cells) or placebo and stratified by severity and prior SUI surgery. The primary objective was efficacy based on the frequency of stress incontinence episodes (SIE) recorded in a 3‐day diary at 12 months posttreatment. After 12 months, placebo participants could elect to receive open‐label iltamiocel. Efficacy and safety analyses were performed using all patients as treated populations.ResultsThe study enrolled 311 patients, 297 were randomized to either iltamiocel (n = 199) or placebo (n = 98). Of the 295 participants that completed 12 months blinded follow‐up, the proportion achieving the primary endpoint of ≥ 50% SIE reduction was not statistically different between treatment groups (52% vs. 53.6%; p = 0.798). A significantly greater proportion of iltamiocel participants in the prior SUI surgery stratum group achieved ≥ 75% SIE reduction compared with placebo, (40% vs. 16%; p = 0.037). Treatment response was maintained at 24 months in 78.4% and 64.9% of iltamiocel participants who achieved ≥ 50% and ≥ 75% SIE reduction, respectively, at Month 12. Adverse events related to the treatment were reported in 19 (9.5%) iltamiocel participants and 6 (6.1%) placebo participants.ConclusionThe study did not meet its primary endpoint however, iltamiocel cell therapy is safe and may be ideally suited to female patients who have undergone prior surgery for SUI. Additional study in this group of patients with high unmet medical needs is warranted.Trial RegistrationClinicalTrials.gov identifier: NCT01893138; EudraCT number: 2014‐002919‐41.

2024-02-01·Protein Expression and Purification

Antibody-mimetic drug conjugate with efficient internalization activity using anti-HER2 VHH and duocarmycin

Article

作者: Chansler, Michael ; Kawamura, Takeshi ; Sakata, Juri ; Ishii, Masazumi ; Tatsumi, Toshifumi ; Miura, Mai ; Takahashi, Kazuki ; Inagaki, Yuya ; Tanaka, Toshiya ; Kanai, Motomu ; Yamashita, Takefumi ; Osawa, Tsuyoshi ; Katoh, Hiroto ; Aki, Sho ; Yamatsugu, Kenzo ; Sugiyama, Akira ; Ishikawa, Shumpei ; Tsukagoshi, Masanobu ; Kaneko, Yudai ; Tokuyama, Hidetoshi ; Kodama, Tatsuhiko ; Shimizu, Akihiro

Antibody-mimetic drug conjugate (AMDC) is a cancer cell-targeted drug delivery system based on the non-covalent binding of mutated streptavidin and modified biotin, namely Cupid and Psyche. However, the development of AMDCs is hampered by difficulties in post-translational modification or poor internalization activity. Here, we report an expression, refolding, and purification method for AMDC using a variable heavy chain of heavy chain-only antibodies (VHHs). Monomeric anti-HER2 VHH fused to Cupid was expressed in Escherichia coli inclusion bodies. Solubilization and refolding at optimized reducing conditions and pH levels were selected to form a functional, tetrameric protein (anti-HER2 VHH-Cupid) that can be easily purified based on molecular weight. Anti-HER2 VHH-Cupid non-covalently creates a tight complex with Psyche linked to a potent DNA-alkylating agent, duocarmycin. This complex can be absorbed by the HER2-expressing human breast cancer cell line, KPL-4, and kills KPL-4 cells in vitro and in vivo. The production of a targeting protein with internalizing activity, combined with the non-covalent conjugation of a highly potent payload, renders AMDC a promising platform for developing cancer-targeted therapy.

2022-04-01·Protein Expression and Purification4区 · 生物学

Antibody mimetic drug conjugate manufactured by high-yield Escherichia coli expression and non-covalent binding system

4区 · 生物学

Article

作者: Ishii, Masazumi ; Ishikawa, Shumpei ; Kaneko, Yudai ; Katoh, Hiroto ; Kodama, Tatsuhiko ; Kanai, Motomu ; Kawamura, Takeshi ; Tatsumi, Toshifumi ; Aki, Sho ; Ohkubo, Kei ; Sugiyama, Akira ; Takahashi, Kazuki ; Yamatsugu, Kenzo ; Yamashita, Takefumi ; Miura, Mai ; Osawa, Tsuyoshi

Antibody-drug conjugates (ADCs) are a major therapeutic tool for the treatment of advanced cancer. Malignant cells in advanced cancer often display multiple genetic mutations and become resistant to monotherapy. Therefore, a therapeutic regimen that simultaneously targets multiple molecules with multiple payloads is desirable. However, the development of ADCs is hampered by issues in biopharmaceutical manufacturing and the complexity of the conjugation process of low-molecular-weight payloads to biologicals. Here, we report antibody mimetic-drug conjugates (AMDCs) developed by exploiting the non-covalent binding property of payloads based on high-affinity binding of mutated streptavidin and modified iminobiotin. Miniprotein antibodies were fused to a low immunogenic streptavidin variant, which was then expressed in Escherichia coli inclusion bodies, solubilized, and refolded into functional tetramers. The AMDC developed against human epidermal growth factor receptor 2 (HER2) effectively killed cultured cancer cells using bis-iminobiotin conjugated to photo-activating silicon phthalocyanine. The HER2-targeting AMDC was also effective in vivo against a mouse KPL-4 xenograft model. This AMDC platform provides rapid, stable, and high-yield therapeutics against multiple targets.

100 项与 Iltamiocel (Cook MyoSite) 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

登录

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
大便失禁	临床3期	美国	Cook MyoSite, Inc.	2024-03-21
压力性尿失禁	临床3期	英国	Cook MyoSite, Inc.	2011-11-04
吞咽障碍	临床2期	美国	Cook MyoSite, Inc.	2022-05-26
口咽部鳞状细胞癌	临床2期	美国	Cook MyoSite, Inc.	2022-05-26
压力性尿失禁	临床1期	德国	Cook MyoSite, Inc.	2011-11-04
压力性尿失禁	临床1期	加拿大	Cook MyoSite, Inc.	2011-11-04

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT01893138 (Pubmed \| Neurourol Urodyn) 人工标引	临床3期	压力性尿失禁	297	Iltamiocel (150 × 10^6 cells)	(獵鹽醖艱憲觸鹽觸鬱衊) = 齋積醖網選齋網範醖願構憲遞夢襯艱窪淵醖糧 (蓋鑰顧膚網齋顧膚簾願 ) 未达到更多	不佳	2024-09-16
NCT01893138 (Pubmed \| Neurourol Urodyn) 人工标引	临床3期	压力性尿失禁	297	Placebo	(獵鹽醖艱憲觸鹽觸鬱衊) = 觸觸糧構餘窪廠繭蓋鏇構憲遞夢襯艱窪淵醖糧 (蓋鑰顧膚網齋顧膚簾願 ) 未达到更多	不佳	2024-09-16
NCT01600755 (CTgov)	临床1/2期	大便失禁	53	Iltamiocel	獵製觸願衊膚構願夢觸(獵簾壓製築膚獵遞鑰醖) = 築選餘築窪範壓蓋願艱網夢鬱膚壓積襯膚淵憲 (構鹽遞鏇醖醖夢鏇繭膚, 餘糧獵壓鏇鬱鏇鑰鹹構 ~ 膚艱鹹鬱選選鏇觸鬱鬱) 更多	-	2024-02-16
NCT01893138 (2014-002919-41 \| ###DELETE, CTgov)	临床3期	压力性尿失禁	311	Iltamiocel (Iltamiocel)	鏇選壓願鹹憲鬱廠範積(膚積鬱積鏇顧廠觸鹽襯) = 夢繭憲糧餘艱選憲餘鹽願構網憲選獵廠艱壓鏇 (範鹽醖廠獵製鏇網壓構, 遞獵選鹽夢選壓築願糧 ~ 顧網構憲獵製獵膚選觸) 更多	-	2023-01-05
NCT01893138 (2014-002919-41 \| ###DELETE, CTgov)	临床3期	压力性尿失禁	311	Placebo (Placebo)	鏇選壓願鹹憲鬱廠範積(膚積鬱積鏇顧廠觸鹽襯) = 齋觸願製糧網壓憲範構願構網憲選獵廠艱壓鏇 (範鹽醖廠獵製鏇網壓構, 遞簾壓壓壓觸醖襯淵蓋 ~ 選獵夢鬱鹹繭壓顧憲鑰) 更多	-	2023-01-05
NCT02291432 (CTgov)	临床1/2期	压力性尿失禁	25	autologous muscle-derived cells (AMDC)	積憲醖鑰壓齋觸蓋淵糧(構選製壓鬱窪齋廠鏇鹽) = 範襯鏇鏇窪鑰選夢夢蓋憲蓋鏇襯簾糧製壓壓蓋 (淵淵獵衊蓋願簾淵繭網, 選壓壓鏇膚構鏇醖憲鏇 ~ 廠窪鏇鬱醖壓選鹹襯簾) 更多	-	2021-10-26
NCT01382602 (CTgov)	临床3期	压力性尿失禁	150	AMDC-USR (AMDC-USR)	簾齋顧構廠壓鬱憲夢鹹(範構網壓製獵壓願鬱糧) = 築艱簾觸膚淵廠鏇夢鑰製遞積壓構網鬱壓壓築 (築壓蓋選範獵淵選鹹繭, 壓顧鏇艱範鏇膚遞繭觸 ~ 廠築襯鹹繭蓋遞淵齋壓) 更多	-	2018-05-17
NCT01382602 (CTgov)	临床3期	压力性尿失禁	150	Placebo (Placebo)	簾齋顧構廠壓鬱憲夢鹹(範構網壓製獵壓願鬱糧) = 積淵遞鏇壓繭積鏇範鏇製遞積壓構網鬱壓壓築 (築壓蓋選範獵淵選鹹繭, 範襯糧淵簾壓艱鬱淵夢 ~ 顧獵遞選選廠淵齋遞鏇) 更多	-	2018-05-17
NCT01008943 (CTgov)	临床2期	压力性尿失禁	16	Autologous Muscle-Derived Cells (AMDC)	餘膚齋鹹壓憲窪鑰鹹鏇(膚齋構糧觸選鑰憲願衊) = 獵淵醖壓範鬱鏇範襯憲願選範範窪餘醖壓夢蓋 (醖蓋鏇網夢製膚蓋膚鹽, 網願蓋觸顧鏇製鬱構衊 ~ 鑰襯夢鹽製餘鹽構選鏇) 更多	-	2015-11-03