喷他脒依西酸盐(Pentamidine Isethionate) - 在研适应症：肺孢子菌肺炎，心肌梗塞_专利_临床

概要

基本信息

药物类型

小分子化药

别名

1,5-bis(4-amidinophenoxy)pentane、4,4'-(1,5-pentanediylbis(oxy))bis-benzenecarboximidamide、4,4'-(pentamethylenedioxy)dibenzamidine

+ [19]

靶点-

作用方式-

作用机制-

治疗领域

感染呼吸系统疾病其他疾病+ [1]

在研适应症

肺孢子菌肺炎心肌梗塞

非在研适应症

晚期胰腺腺癌肝细胞癌结肠转移性腺癌+ [2]

原研机构

Fresenius Kabi AG

在研机构

Medical College of Wisconsin

Fresenius Kabi USA LLCSanofi KK

非在研机构

Verlyx Pharma, Inc.

权益机构-

最高研发阶段批准上市

首次获批日期

美国 (1984-10-16),

肺孢子菌肺炎

最高研发阶段(中国)-

特殊审评孤儿药 (美国)

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结构/序列

分子式C23H36N4O10S2

InChIKeyYBVNFKZSMZGRAD-UHFFFAOYSA-N

CAS号140-64-7

关联

40

项与喷他脒依西酸盐相关的临床试验

NCT03730363

/ Completed临床1期IIT

A Phase I Study of Pentamidine in Combination With Salvage Chemotherapy for Relapsed/Refractory Classical Hodgkin Lymphoma

Primary Objective:
To evaluate dose limiting toxicity and to determine the recommended phase 2 dose (RP2D) of pentamidine in combination with salvage chemotherapy with ifosfamide, carboplatin and etoposide (ICE) on a 3-weeks schedule in relapsed/refractory classical Hodgkin lymphoma (cHL).
Secondary Objective:
* To estimate the overall best treatment response at 5- and 16-weeks from study enrollment. Although the clinical benefit of these drugs in combination has not been established, offering this treatment may provide a therapeutic benefit. The patients will be carefully monitored for tumor response and symptom relief, in addition to safety and tolerability.
* To estimate the duration of response to the proposed combined therapy.
* To measure the protein of regenerating liver-3 (PRL-3) level of expression in patients at time of relapse.
* To measure circulating biomarkers of response (soluble CD30 (sCD30), and thymus and activation-related chemokine (TARC)) in serum samples collected throughout treatment and inhibition of (pSTAT, pAKT) in peripheral blood mononucleated cells (PBMC).
Exploratory Objective:
* To measure cell-free messenger RNA (cfmRNA) in peripheral blood.
* To measure cell-free DNA in peripheral blood

开始日期2018-12-19

申办/合作机构

University of Kentucky

NCT03403621

/ Terminated临床1/2期IIT

Double Blind, Single-Center, Randomized, Within Subject Placebo, Phase I Study Evaluating the Effects of Novel Topical Gel in Prevention of Hypertrophic Scar Formation

Researchers are trying to find out more about the side effects of topical (applied to the skin) Pentamidine, to determine if it is safe for use in people. They also want to find out if topical use of Pentamidine can help treat hypertrophic scars.
Pentamidine is a medicine that is currently used to treat certain kinds of infection. It is most often given by intravenous (into a vein) or inhalation (through a breathing device). This medication is approved by the U.S. Food and Drug Administration (FDA) for use in these forms.
Everyone in this study will receive topical Pentamidine (TP) in a silicone based gel (PCCA Pracasil Plus). Topical treatment of Pentamidine is still experimental and has not been formally tested for safety or effectiveness in a randomized control trial within the United States. The FDA has allowed the use of topical Pentamidine in this research study.

开始日期2018-03-05

申办/合作机构

Mayo Clinic

NCT03201159

/ Withdrawn临床1期IIT

A Pilot, Exploratory Dose Escalating Study on the Safety, Pharmacodynamics and Preliminary Efficacy of VLX103 in the Treatment of Moderate Alcoholic Steatohepatitis

The study drug (VLX103) is being developed for the treatment of Alcoholic Steatohepatitis and other liver diseases. Alcoholic Steatohepatitis is an inflammatory (associated with irritation, swelling and cell damage) disease that affects the liver. It is associated with heavy and chronic intake of alcohol and presence of fat in the liver. Signs and symptoms often include fever, yellowing of the skin, nausea and impairment of liver function.
The main objective of this study is to evaluate the safety, pharmacodynamics (what the drug does to the body) and pharmacokinetics (how the drug is handled by the human body, like absorption and elimination) of increasing doses of VLX103 in subjects with moderate Alcoholic Steatohepatitis. In other words, we will evaluate how your body tolerates VLX103 at a specific dose and the effects that this VLX103 dose has on your liver and your body in general. The secondary objectives of this study are to evaluate if VLX103 has the potential to treat Alcoholic Steatohepatitis patients, to determine the maximum dose that can be tolerated, and to measure the levels of VLX103 in your blood at different time points during the study.
VLX103 is an experimental drug. Experimental means that the drug has not been approved by the Food and Drug Administration (FDA) for the treatment of Alcoholic Steatohepatitis. The active ingredient in VLX103, pentamidine, is approved for treating parasitic (microorganisms) infections. Pentamidine is currently approved and marketed in about 20 countries, including the United States, for use by injection (administered by a syringe) and by inhalation (administered by a nebulizer) for other health conditions. However, VLX103 is the first oral form of pentamidine being developed, and is administered by mouth as an oral tablet.

开始日期2017-06-25

申办/合作机构

National Institute on Alcohol Abuse & Alcoholism

[+3]

100 项与喷他脒依西酸盐相关的临床结果

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100 项与喷他脒依西酸盐相关的转化医学

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100 项与喷他脒依西酸盐相关的专利（医药）

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2,970

项与喷他脒依西酸盐相关的文献（医药）

2025-12-10·ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

A CRISPR-based diagnostic tool to survey drug resistance in human African trypanosomiasis

Article

作者: Pérez Antón, Elena ; Rotureau, Brice ; Dujeancourt-Henry, Annick ; Glover, Lucy

ABSTRACT:

The World Health Organization aims to eliminate human African trypanosomiasis caused by Trypanosoma brucei gambiense (gHAT) by 2030. With the decline of reported cases, maintaining active surveillance is essential, including for the potential emergence of drug-resistant parasites. We have developed new highly specific diagnostic tools, using the Cas13a-based Specific High-Sensitivity Reporter Enzymatic UnLOCKing (SHERLOCK) technology, for the detection of drug-resistant genotypes that (i) are already circulating, such as the AQP2/3 (814) chimera providing resistance to pentamidine and melarsoprol or (ii) could emerge, such as the Tb CPSF3 (N 232 H) mutation, associated with acoziborole resistance under laboratory conditions. The AQP2/3 (814) SHERLOCK assay detected RNA from both cultured parasites and field strains isolated from gHAT patients who relapsed following melarsoprol or pentamidine treatment. The CPSF3 (SNV) SHERLOCK assay discriminated between wild-type CPSF3 RNA and CPSF3 bearing a single A-C mutation that confers resistance to acoziborole in vitro . These SHERLOCK assays are amenable for use as a high-throughput screening method to monitor for drug-resistant-associated mutations in Trypanosoma brucei , providing a new molecular tool for epidemiological surveillance during the gHAT elimination phase.

2025-12-01·International Journal for Parasitology-Drugs and Drug Resistance

Biological and molecular characterisation of in vitro selected miltefosine-resistant Leishmania amazonensis lines

Article

作者: Escobar, Patricia ; Neira, Laura Fernanda ; Ribeiro, Juliana Martins ; Murta, Silvane Maria Fonseca ; Costa-Silva, Héllida Marina

Miltefosine (MTF) is currently the only available oral treatment for leishmaniasis. However, increasing reports of therapeutic failure have raised concerns about emerging resistance. This study aimed to investigate the effects of reduced MTF susceptibility loss in the protozoan parasite Leishmania (Leishmania) amazonensis, with a particular focus on its impact on key biological and molecular parameters. Two distinct Leishmania lines (LaR-40 Line 1 and Line 2) were generated through stepwise in vitro selection with increasing concentrations of MTF, reaching up to 40 μM MTF. They were compared to their wild-type counterpart (LaWT). After 12 weeks of selection, LaR-40 promastigotes exhibited IC₅₀ values that were 4- to 8-fold higher than those of LaWT, with resistance remaining stable even after three months without drug pressure and following passage through BALB/c mice. No cross-resistance was detected against pentamidine, ketoconazole, or amphotericin B. MTF-resistant parasites exhibited reduced reactive oxygen species production, reduced lipid droplets (LD) abundance (in LaR-40 Line 1), delayed lesion onset, and smaller cutaneous lesions in mice, while maintaining normal infectivity in THP-1 macrophages. Quantitative RT-PCR analysis revealed consistent downregulation of the miltefosine transporter (mt) gene in both MTF-resistant lines, indicating that reduced drug uptake is the main mechanism underlying resistance. Line-specific changes, such as the upregulation of the serine palmitoyltransferase (spt) gene or the downregulation of the trypanothione reductase (tryr) gene, suggest that distinct metabolic pathways may act in a compensatory manner to reinforce resistance once transporter function is impaired. These findings indicate that MTF resistance in L. amazonensis is polygenic, stable, and adaptable. Routine monitoring of mt gene expression, combined with therapeutic strategies that target lipid or redox metabolism alongside drug uptake pathways, may help preserve the efficacy of current treatment regimens against leishmaniasis.

2025-12-01·INFECTION

Use of fexinidazole in gambiense human African trypanosomiasis: a retrospective analysis of cases treated in Lui Hospital, South Sudan (2018–2024)

Article

作者: Rusconi, Stefano ; Gelormino, Elena ; Paggi, Riccardo ; Putoto, Giovanni ; Benson, Grant Sebit ; Scanagatta, Chiara ; Zammarchi, Lorenzo ; Amodu, Abiodun ; Mariotti, Francesca ; Pettenuzzo, Sofia ; Dacquino, Stefano ; Basilico, Matteo

Abstract:

Purpose:

Fexinidazole, an oral molecule, replaced pentamidine and combined treatment with nifurtimox and eflornithine (NECT) therapy for stage 1 and non-severe stage 2 gambiense human African Trypanosomiasis ( g -HAT), respectively. The study aims to evidence differences of outcome at discharge and adverse drug reactions (ADRs) between fexinidazole and pentamidine/NECT regimens in patients with stage 1 and non-severe stage 2 g -HAT admitted to Lui Hospital (Western Equatoria, South Sudan), a historical g -HAT focus.

Methods:

Data of patients ( n = 86) admitted to Lui Hospital from July 2018 to June 2024 with g- HAT diagnosis were included. Among them, we considered for the analysis patients eligible for both fexinidazole and pentamidine/NECT regimens (i.e. patients without symptoms/signs compatible with severe stage 2 g -HAT).

Results:

In the study population 17% of patients were registered with an unfavourable outcome (signs or symptoms of g -HAT at discharge or death attributable to g -HAT or g -HAT treatment occurred during hospitalization). No significant differences between fexinidazole and pentamidine/NECT in terms of outcome at discharge (23% vs. 6%, p = 0.230) and ADRs frequency (70% vs. 50%, p = 0.181) were reported. Although fexinidazole cohort experienced more gastro-intestinal ADRs than pentamidine/NECT cohort (63% vs. 19%, p = 0.005), discontinuation of oral treatment has not been recorded.

Conclusion:

Patients treated with fexinidazole and pentamidine/NECT showed similar results in terms of outcome at discharge and ADRs, in line with current data available in literature. However, few real-life studies on efficacy of fexinidazole treatment were published: to our knowledge, this is the first one conducted in South Sudan.

1

项与喷他脒依西酸盐相关的新闻（医药）

2024-03-07

·Business Wire

Montdorex Announces Acceptance of Its Abstract for Presentation at the AACR Annual Meeting 2024

MONTREAL--( BUSINESS WIRE )--Montdorex Inc. (“Montdorex” or “the Company”), a privately-owned precision medicine company, today announced that Terry Chow, Ph.D., Founder and CEO will present insights on the relevance of inhibiting the endo-exonuclease (EE) enzyme in a poster presentation at the American Association for Cancer Research (AACR) Annual Meeting, to be held in San Diego, CA, April 5-10, 2024. The endo-exonuclease enzyme plays a key role in DNA damage repair mechanisms, but it is also overexpressed in cancer tumors. In a proof-of-concept study, Montdorex showed that tumors with increased EE expression showed the best response to pentamidine, an endo-exonuclease inhibitor. The Company has also identified mono- and di-amidine analogs of pentamidine that are more effective than the parent drug in both in vitro and in vivo studies. “Our lead candidate, MTDX203, showed an increased anti-cancer activity with a higher safety index than pentamidine in preclinical animal studies,” said Terry Chow of Montdorex. “I look forward to presenting our detailed findings to the AACR scientific and investor community at the upcoming annual meeting.” Details of the AACR poster presentation are as follows: Poster Presentation (#5604): Modulating DNA repair through endo-exonuclease inhibition: a new therapeutic paradigm in Oncology [ link to the abstract ] Session Category: Molecular/Cellular Biology and Genetics Session Title: DNA Damage and Repair 2 Session Date and Time: Tuesday, April 9, 2024 1:30 PM – 5:00 PM (PT) Location: Poster Section 14, Poster Board Number 2 About Montdorex Based in Montreal, Canada, Montdorex Inc. is a privately-owned, early-stage precision medicine company, targeting the endo-exonuclease (EE) enzyme involved in cancer tumor growth and inflammatory processes. The company is developing proprietary mono-amidine and di-amidine EE inhibitors to modulate DNA repair in oncology and non cytotoxic di-amidine analogs for anti-inflammatory applications, such as gastrointestinal conditions and liver diseases. For more information, please visit montdorex.com .

AACR会议临床结果

100 项与喷他脒依西酸盐相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D00834	喷他脒依西酸盐	P01CX01	DB00738

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
肺孢子菌肺炎	美国	Fresenius Kabi USA LLC	1984-10-16

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10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
转移性非小细胞肺癌	临床2期	波兰	Verlyx Pharma, Inc.	2012-09-01
转移性结直肠癌	临床2期	加拿大	Verlyx Pharma, Inc.	2011-03-01
晚期胰腺腺癌	临床2期	加拿大	Verlyx Pharma, Inc.	2009-01-01
结肠转移性腺癌	临床2期	加拿大	Verlyx Pharma, Inc.	2008-03-01
肝细胞癌	临床1期	加拿大	Verlyx Pharma, Inc.	2014-08-01
心肌梗塞	临床前	美国	Medical College of Wisconsin	2025-04-09

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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


NCT03403621 (CTgov)	临床1/2期	皮肤增生性瘢痕	6	Pentamidine Isethionate (Topical Pentamidine Isethionate)	淵顧餘襯鹽蓋範觸鹽願 = 齋獵鬱夢齋繭窪餘膚鏇獵夢製簾蓋壓襯網繭窪 (衊膚顧憲艱鏇積糧製廠, 膚淵範鏇憲衊顧簾選膚 ~ 築餘鹽繭醖積糧窪獵顧) 更多	-	2023-11-22
NCT03403621 (CTgov)	临床1/2期	皮肤增生性瘢痕	6	Placebo (Placebo Control)	淵顧餘襯鹽蓋範觸鹽願 = 淵簾獵觸鑰築構範觸憲獵夢製簾蓋壓襯網繭窪 (衊膚顧憲艱鏇積糧製廠, 簾醖廠憲範蓋鬱顧廠膚 ~ 淵鏇憲顧願築製淵衊網) 更多	-	2023-11-22
NCT01360762 (CTgov)	临床3期	获得性免疫缺陷综合征 \| 内脏利什曼病	74	Pentamidine	鹹壓襯製願膚齋衊糧觸 = 鹹構齋醖選構鬱衊鬱鏇壓艱鑰觸憲糧齋遞憲艱 (醖餘積鑰鹽簾醖蓋蓋壓, 網簾繭鏇糧顧艱顧鏇醖 ~ 衊糧遞窪簾構夢壓範構) 更多	-	2019-02-15
NCT02669706 (Pubmed \| Bone Marrow Transplant)	N/A	造血干细胞移植	50	Intravenous pentamidine	壓淵窪鑰壓願願製齋網(窪鑰繭餘簾簾蓋壓壓壓) = 選鑰築選鏇壓糧餘醖鏇觸願選製製壓範廠糧窪 (選選餘積繭鏇繭艱願鑰 )	-	2018-03-01
NCT01360762 (Pubmed \| Clin Infect Dis)	临床3期	HIV感染 \| 内脏利什曼病	74	Pentamidine secondary prophylaxis	構鏇鏇淵憲襯積糧製繭(網壓糧廠獵鹽蓋鹹簾膚) = 淵範鑰艱衊餘憲願廠艱膚衊餘窪選構鏇鏇積願 (醖鹹繭鑰醖膚製廠範壓 ) 更多	-	2018-01-18
Tandem Meetings ASTCT and CIBMTR2017	N/A	血液肿瘤 \| 肺孢子菌肺炎	-	IV Pentamidine	鑰築糧壓齋觸鏇鹽窪鬱(齋壓鹽築鏇鑰選衊壓製) = 觸網選遞齋廠繭艱願餘鏇鑰構顧夢艱鏇選糧鹽 (鏇獵廠選鑰簾膚鬱鬱餘 ) 更多	-	2017-03-01
NCT00729807 (CTgov)	临床2期	难治性黑色素瘤 \| 皮肤黑色素瘤	6	pentamidine	鏇選壓鏇遞繭廠顧憲鏇 = 醖範壓糧顧鑰繭築製餘鏇艱願繭選醖壓鑰選積 (鹹窪簾夢製鑰範網獵範, 繭鹽選鬱齋鹽鹽餘築襯 ~ 夢遞艱鏇顧觸願獵夢襯) 更多	-	2016-10-21
Tandem Meetings ASTCT and CIBMTR2016	N/A	肺孢子菌肺炎 \| 造血干细胞移植	58	Intravenous Pentamidine	衊積夢構積鹽鏇淵憲鏇(獵餘淵齋蓋鬱範繭蓋製) = 襯積壓繭鹽鬱膚獵鹽觸選網糧鹽廠壓鏇選鑰範 (選觸憲選憲顧壓蓋夢鏇 )	积极	2016-03-01
ARVO2013	N/A	棘阿米巴角膜炎	24	Pentamidine isethionate	夢築顧獵願艱製鏇淵膚(願艱鑰範顧餘蓋齋齋壓) = 願繭繭簾夢製鹽蓋簾襯窪艱壓鹽積糧選淵憲願 (製糧蓋顧襯鏇遞顧鏇顧, 21.7) 更多	-	2013-06-01
NCT00014911 (CTgov)	临床2期	1型糖尿病	36	Islet Transplantation+Ganciclovir+Sulfamethoxazole+Tacrolimus+Sirolimus+Daclizumab+Pentamidine+Trimethoprim	蓋願餘鹽襯廠艱窪願遞 = 淵夢膚蓋構憲襯壓艱遞鏇壓窪觸積觸製遞顧繭 (襯顧齋遞積鏇網膚鑰築, 遞獵鹽製製繭膚餘憲餘 ~ 壓範積範選鑰築淵網顧) 更多	-	2012-10-17
NCT00078559 (CTgov)	临床1/2期	肾脏疾病 \| 肾移植排斥反应	10	Kidney transplant+Acyclovir+Acetaminophen+Bactrim+Nystatin+Sirolimus+Clotrimazole+Trimethoprim+Diphenhydramine+TMP+Alemtuzumab+Valgancyclovir+Sulfa+Methylprednisolone+Tacrolimus+Pentamidine	窪壓鹽鑰鬱鏇簾蓋簾窪 = 鑰廠夢獵願襯衊顧觸鹹構襯艱齋繭壓夢襯廠齋 (鬱積顧夢觸醖壓糧遞鑰, 觸鑰憲鏇淵艱窪鹹製夢 ~ 壓蓋顧壓糧鬱衊齋範廠) 更多	-	2012-07-09