帕利夫明(Palifermin) - 药物靶点：FGFR1 x FGFR2_在研适应症：口腔炎_专利_临床

概要

基本信息

药物类型

生长因子

别名

24-163 fibroblast growth factor 7 (human)、KGF、Keratinocyte growth factor

+ [9]

靶点

FGFR1 x FGFR2

作用机制

FGFR1刺激剂(成纤维细胞生长因子受体-1刺激剂)、FGFR2刺激剂(成纤维细胞生长因子受体-2刺激剂)

治疗领域

口颌疾病

在研适应症

口腔炎

非在研适应症

急性白血病晚期头颈癌费城染色体阳性慢性粒细胞白血病+ [16]

原研机构

Sobi Analytics Ltd.

在研机构

Swedish Orphan Biovitrum AB

非在研机构

Amgen, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2004-12-15),

口腔炎

最高研发阶段(中国)-

特殊审评-

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序列信息

Sequence Code 31652

来源: *****

关联

51

项与帕利夫明相关的临床试验

NCT06325709 / Recruiting临床1/2期IIT

Phase 1/2 Trial of Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-linked Chronic Granulomatous Disease

Background:
Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly. This places them at risk of developing infections that may be life-threatening. Stem cell transplant can cure CGD but transplanting stem cells donated by other people can have serious complications. In addition, not everyone has a matched donor. Another approach is a type of gene therapy that involves base-editing to correct the mutation in a person s own stem cells. Researchers want to know if the base-edited stem cells can improve the white cells' functioning and result in fewer CGD-related infections.
Objective:
To learn if base-edited stem cells will improve white blood cells' ability to fight against infections in people with CGD.
Eligibility:
Males aged 18 years and older with X-linked CGD.
Design:
This is a non-randomized study. Participants with the specific mutation under study will be screened during the initial phase.
During the development phase, participants will undergo apheresis to collect stem cells for base-editing correction of the mutation.
During the treatment phase, participants will receive the base-edited cells after chemotherapy with busulfan. Participants will remain in the hospital until their immunity recovers.
Follow-up visits will continue for 15 years.

开始日期2024-04-17

申办/合作机构

National Institute of Allergy & Infectious Diseases

NCT04177004 / Recruiting临床1期IIT

A Randomized Pilot Study of Human Lysozyme Goat Milk in Recipients of Standard Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation

This phase I trial studies the side effects of human lysozyme goat milk in preventing graft versus host disease in patients with blood cancer undergoing a donor stem cell transplant. Sometimes the transplanted cells from a donor can cause an immune response against the body's own normal cells (call graft versus host disease). The goat milk in the study is from goats that have been genetically engineered to produce human lysozyme in the milk. Human lysozyme is a natural enzyme found in human milk and acts as an antimicrobial. Lysozyme is key to the digestive health of breast-fed human infants, since it helps the growth of beneficial gut bacteria and reduces the growth of bacteria that causes diarrhea and intestinal disease. Giving human lysozyme goat milk may reduce the rate of graft versus host disease in blood cancer patients undergoing a donor stem cell transplant.

开始日期2021-04-30

申办/合作机构

City of Hope National Medical Center

[+1]

NCT03100877 / Withdrawn临床1/2期IIT

Phase I-II Single Cycle Melphalan/Total Marrow Irradiation (TMI) and Autologous Stem Cell Transplantation (ASCT) Followed by Maintenance in Patients With High-Risk Myeloma and/or Poor Response to Induction Therapy Within 12 Months of Diagnosis

This phase I/II trial studies the side effects and best dose of melphalan and total marrow irradiation and how well they work with autologous stem cell transplantation in treating patients with high-risk multiple myeloma. Drugs used in chemotherapy, such as melphalan, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Total marrow irradiation is a type of radiation therapy and a form of total body irradiation that may deliver focused radiation to the major marrow sites where cancer cells reside. Giving chemotherapy and total-body irradiation before a peripheral autologous blood stem cell transplant helps kill any cancer cells that are in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. After treatment, stem cells are collected from the patient's blood and stored. More chemotherapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

开始日期2018-01-01

申办/合作机构

City of Hope National Medical Center

[+1]

100 项与帕利夫明相关的临床结果

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100 项与帕利夫明相关的转化医学

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100 项与帕利夫明相关的专利（医药）

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771

项与帕利夫明相关的文献（医药）

2024-05-01·Journal of Biotechnology

Enhanced prokaryotic expression, purification, and biological activities of human keratinocyte growth factor

Article

作者: Choe, Han ; Vu, Thi Luong ; Song, Jung-A ; Chong, Seonha ; Nguyen, Thi Kieu Oanh

Keratinocyte growth factor (KGF), also known as fibroblast growth factor 7 (FGF7), plays a critical role in embryonic development, cell proliferation, and differentiation. However, efficient production of recombinant KGF remains a challenge due to its low expression levels and high tendency for aggregation in Escherichia coli. This study aimed to enhance the expression and solubility of KGF by employing different protein tags-PDIb'a', MBP, and His-fused to the N-terminus of KGF. Among these, H-PDIb'a'-KGF demonstrated superior stability and was selected for large-scale production and purification. The purified KGF was confirmed through liquid chromatography with tandem mass spectrometry analysis, which showed an 81% fragment mass identification coverage. Biological activity assessments using human breast cancer MCF-7 cells indicated that purified KGF significantly increased cell proliferation, with an EC₅₀ of 6.4 ± 0.5 pM. Interestingly, PDIb'a' alone also exhibited a stimulatory effect on MCF-7 cells. Furthermore, the purified KGF enhanced the wound healing of HaCaT keratinocytes in a dose-dependent manner. These findings provide valuable insights into the efficient production and functional characterization of recombinant KGF for potential applications in therapeutic interventions.

2023-12-01·Journal of Genetic Engineering and Biotechnology

The potential role of the KFG and KITLG proteins in preventing granulosa cell apoptosis in Bubalus bubalis

Article

作者: Rawal, Leena ; Panwar, Deepak ; Ali, Sher

AbstractBackgroundThe dynamics of mammalian follicular development and atresia is an intricate process involving the cell-cell communication mediated by secreted ovarian factors. These interactions are critical for oocyte development and regulation of follicular atresia which in part are mediated by keratinocyte growth factor (KGF) and kit ligand (KITLG), but their roles in the regulation of apoptosis in buffalo granulosa cells have not yet been defined. During mammalian follicular development, granulosa cell apoptosis triggers the atresia so ~ 1% follicles reach the ovulation stage. In the present study, we used buffalo granulosa cells to examine the effects of KGF and KITLG in apoptosis regulation and investigated potential mechanism on Fas-FasL and Bcl-2 signaling pathways.ResultIsolated buffalo granulosa cells were cultured with KGF and KITLG proteins using different doses (0, 10, 20, and 50 ng/ml) independently or in combination. Expression analysis for both anti-apoptotic (Bcl-2,Bcl-xL, andcFLIP) and pro-apoptotic (Bax, Fas,andFasL) genes at transcriptional levels were carried out by real-time PCR. Upon treatments, expression levels of anti-apoptotic genes were significantly upregulated in a dose-dependent manner, showing an upregulation at 50 ng/ml (independently), and at 10 ng/ml in combination. Additionally, upregulation of growth-promoting factors,bFGF, andα-Inhibinwas also observed.ConclusionsOur findings suggest the potential roles of KGF and KITLG in determining granulosa cell growth and regulating apoptosis.

2023-12-01·Biomedicine & Pharmacotherapy

Mesenchymal stem cell conditioned medium alleviates acute lung injury through KGF-mediated regulation of epithelial sodium channels

Article

作者: Hao, Liying ; Cui, Yong ; Nie, Hongguang ; Yu, Tong ; Fu, Yunmei ; Xin, Shuning ; Ding, Yan

Acute lung injury (ALI) is a progressive inflammatory injury, and mesenchymal stem cells (MSCs) can be used to treat ALI. MSC-conditioned medium (MSC-CM) contains many cytokines, in which keratinocyte growth factor (KGF) is a soluble factor that plays a role in lung development. We aim to explore the protective effects of MSCs secreted KGF on ALI, and investigate the involvement of epithelial sodium channel (ENaC), which are important in alveolar fluid reabsorption. Both lipopolysaccharides (LPS)-induced mouse and alveolar organoid ALI models were established to confirm the potential therapeutic effect of MSCs secreted KGF. Meanwhile, the expression and regulation of ENaC were determined in alveolar type II epithelial (ATII) cells. The results demonstrated that MSC-CM and KGF could alleviate the extent of inflammation-related pulmonary edema in ALI mice, which was abrogated by a KGF neutralizing antibody. In an alveolar organoid ALI model, KGF in MSC-CM could improve the proliferation and decrease the differentiation of ATII cells. At the cellular level, the LPS-inhibited protein expression of ENaC could be reversed by KGF in MSC-CM. In addition, bioinformatics analysis and our experimental data provided the evidence that the NF-κB signaling pathway may be involved in the regulation of ENaC. Our research confirmed that the therapeutic effect of MSC-CM on edematous ALI was closely related to KGF, which may be involved in the proliferation and differentiation of ATII cells, as well as the upregulation of ENaC expression by the inhibition of NF-κB signaling pathway.

100 项与帕利夫明相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D05338	帕利夫明	V03AF08	DB00039

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
口腔炎	美国	Swedish Orphan Biovitrum AB	2004-12-15

未上市

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
口腔炎	临床1期	冰岛	Swedish Orphan Biovitrum AB	2005-10-25
口腔炎	临床1期	挪威	Swedish Orphan Biovitrum AB	2005-10-25
头颈部肿瘤	临床前	-	Amgen, Inc.	2005-01-01
口腔炎	药物发现	列支敦士登	Swedish Orphan Biovitrum AB	2005-10-25
口腔炎	药物发现	欧盟	Swedish Orphan Biovitrum AB	2005-10-25

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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


ASCO2014	N/A	急性淋巴细胞白血病 \| 实体瘤	816	Palifermin	(廠窪願願鹽積艱糧餘遞) = 範鹽蓋選衊壓築壓齋淵鏇醖顧顧鬱襯觸願遞構 (夢糧遞繭憲構壓願繭選 ) 更多	-	2014-05-20
				No Palifermin	(廠窪願願鹽積艱糧餘遞) = 淵繭淵選顧鑰鏇網築構鏇醖顧顧鬱襯觸願遞構 (夢糧遞繭憲構壓願繭選 ) 更多
NCT00626639 (CTgov)	临床1/2期	粘膜炎 \| 局部晚期头颈部鳞状细胞癌 \| 局部晚期恶性肿瘤 \| 口腔炎	5	Radiotherapy+Cisplatin (Placebo)	(壓築窪觸築蓋蓋遞選鹹) = 夢鏇鏇襯積廠淵膚艱醖觸獵選餘齋蓋鬱艱鹽憲 (網壓夢齋鏇鏇衊醖襯夢, 鬱獵蓋顧廠淵構餘築襯 ~ 淵網膚鹹艱製鬱衊選壓) 更多	-	2014-03-06
				Radiotherapy+palifermin+Cisplatin (Palifermin)	(壓築窪觸築蓋蓋遞選鹹) = 範襯窪醖顧齋顧構鹽窪觸獵選餘齋蓋鬱艱鹽憲 (網壓夢齋鏇鏇衊醖襯夢, 製窪繭餘廠餘夢齋構衊 ~ 簾簾鏇遞遞膚壓醖獵夢) 更多
NCT00041665 (Pubmed \| Br Dent J)	临床3期	化疗性口腔黏膜炎	212	Palifermin	(蓋壓廠淵窪繭艱淵願構) = Adverse events, mainly rash, pruritus, erythema, mouth and tongue disorders, and taste alteration, were mild to moderate in severity and were transient 築繭範壓範廠鹽廠鹹製 (鹹選窪壓築壓鬱願廠製 )	积极	2004-12-16
				Placebo
EBMT2020	N/A	淋巴瘤	73	Palifermin + BEAM conditioning	(膚醖鹽獵鬱齋艱構淵觸) = 膚觸網鏇壓選憲鑰糧糧窪糧簾淵蓋觸壓鏇積築 (選遞構蓋鏇餘夢遞糧築 ) 更多	-	2020-08-29
				BEAM conditioning	(膚醖鹽獵鬱齋艱構淵觸) = 選網窪鏇衊廠遞廠壓網窪糧簾淵蓋觸壓鏇積築 (選遞構蓋鏇餘夢遞糧築 ) 更多
NCT00094861 (CTgov)	临床2期	局部晚期非小细胞肺癌 \| 吞咽障碍	100	Radiotherapy+Carboplatin+Paclitaxel (Placebo)	(醖壓襯鏇齋壓齋廠選網): Chi-Square Statistic = 0.8553 (95% CI) 更多	-	2014-03-06
				Radiotherapy+Carboplatin+Paclitaxel+Palifermin (Palifermin)
NCT02037347 (CTgov)	临床1/2期	Stevens-Johnson综合征	1	Palifermin	廠窪齋簾艱製遞醖繭蓋(壓艱夢襯築淵壓築膚齋) = 憲廠糧鹽簾夢衊願鏇鏇積觸鑰簾範餘選醖艱鹹 (齋襯夢醖積憲壓壓網齋, 積夢餘築獵壓鏇鬱範網 ~ 簾觸鏇淵獵餘鏇願廠獵) 更多	-	2016-11-30
NCT00109031 (CTgov)	临床3期	淋巴瘤 \| 口腔炎 \| 白血病	47	Placebo+Etoposide+Cyclophosphamide+palifermin (Palifermin 60 µg/kg for 3 Days)	選艱襯觸衊夢襯願鬱觸(廠蓋構齋蓋範網醖網願) = 觸夢鬱簾積夢簾壓膚廠選選獵觸憲蓋遞鏇鑰繭 (醖遞積鹹製淵廠遞鬱網, 醖範廠衊簾簾選憲積壓 ~ 憲築簾觸衊淵築簾襯夢) 更多	-	2014-09-15
				Placebo+Etoposide+Cyclophosphamide+palifermin (Palifermin 180 μg/kg on Day -1)	選艱襯觸衊夢襯願鬱觸(廠蓋構齋蓋範網醖網願) = 製窪築遞選餘選壓鹹廠選選獵觸憲蓋遞鏇鑰繭 (醖遞積鹹製淵廠遞鬱網, 觸簾鹹襯繭鏇襯願範鹹 ~ 積醖憲鏇構壓夢憲齋鹹) 更多
NCT00101582 (CTgov)	临床3期	FGF19阳性实体瘤 \| 鳞状细胞癌 \| 口腔炎 \| 头颈部肿瘤 \| 粘膜炎	188	Radiotherapy+cisplatin chemotherapy (Placebo)	築構積餘廠餘鹽膚製襯(齋窪觸繭糧顧繭鹽網製): Chi-Square Statistic = 4.1764 (95% CI) 更多	-	2014-09-15
				Radiotherapy+palifermin+cisplatin chemotherapy (Palifermin)
NCT00360971 (CTgov)	临床3期	头颈部肿瘤 \| 局部晚期恶性肿瘤 \| 粘膜炎 \| 疼痛 \| 口腔炎 \| 辐射损伤	21	placebo+cisplatin (Placebo)	築鹹願選鹽艱簾鬱廠壓(齋鹽鹹夢製壓廠壓構餘) = 選壓範獵鏇製鹽構觸遞餘餘製襯構網繭鬱構蓋 (窪鑰鬱鹹鏇鑰艱蓋築艱, 鬱願築衊觸襯衊鏇襯觸 ~ 網遞憲願鬱壓築艱襯衊) 更多	-	2014-05-15
				neck dissection+palifermin+cisplatin (Palifermin)	築鹹願選鹽艱簾鬱廠壓(齋鹽鹹夢製壓廠壓構餘) = 願積襯鹹積膚鹽遞鹹窪餘餘製襯構網繭鬱構蓋 (窪鑰鬱鹹鏇鑰艱蓋築艱, 餘鏇選製餘簾艱鏇鹹齋 ~ 簾選壓糧壓窪鬱鹹鏇鏇) 更多
ISRCTN95690673 (KARE, Pubmed)	临床2期	成人呼吸窘迫综合征	60	Recombinant human KGF (palifermin 60 μg/kg)Recombinant human KGF (palifermin 60 μg/kg)	(顧餘醖齋鑰艱憲獵網網) = Adverse events were more frequent in the KGF group than the placebo group (14 vs 5 events; odds ratio 4·9, 95% CI 1·3 to 20·3, p=0·008). The two adverse events assessed as related to KGF were due to pyrexia. 網夢壓蓋選廠築餘壓壓 (襯糧廠鹹鏇醖製廠積醖 )	不佳	2017-06-01
				Placebo (0·9% sodium chloride solution)