培维索孟(Pegvisomant) - 药物靶点：GHR_在研适应症：肢端肥大症_专利_临床

概要

基本信息

药物类型

激素

别名

Pegvisomant (USAN/INN)、Pegvisomant (genetical recombination) (JAN)、pegviosomant

+ [6]

靶点

GHR

作用机制

GHR拮抗剂(生长激素受体拮抗剂)

治疗领域

神经系统疾病内分泌与代谢疾病皮肤和肌肉骨骼疾病

在研适应症

肢端肥大症

非在研适应症

晚期恶性实体瘤乳腺癌去势抵抗性前列腺癌+ [5]

原研机构

Sensus USA, Inc.

在研机构

Pfizer Europe MA EEIGPharmacia & Upjohn LLC

Pfizer Inc.

非在研机构

Sensus Drug Development Corp.

最高研发阶段批准上市

首次获批日期

欧盟 (2002-11-12),

肢端肥大症

最高研发阶段(中国)-

特殊审评孤儿药 (日本)

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序列信息

Sequence Code 61626

来源: *****

关联

37

项与培维索孟相关的临床试验

NCT05470504 / Recruiting临床2期IIT

Phase II Study of Growth Hormone Inhibition Using Pegvisomant In Severe Insulin Resistance

Background:
Lipodystrophy (LD) syndromes are a group of rare disorders that affect how a person s body can store and use fat tissue. Many people with LDs become severely insulin resistant. Some people are insulin resistant because of a variant in the insulin receptor gene. Insulin resistance causes many health problems.
Objective:
To learn if blocking the effects of growth hormone in the body will help people with severe insulin resistance.
Eligibility:
Adults aged 18 to 65 years with either a known variant in the insulin receptor gene or with a diagnosis of partial LD.
Design:
Participants will have 2 hospital stays, about 1 month apart. Each stay will be 3 or 4 nights.
During each hospital stay, participants will have many tests. They will have a physical exam with blood tests. They will have all of their urine collected for a 24-hour period. They will have scans to measure their muscle, bone, and fat tissues. They will have tests to measure metabolism and insulin sensitivity. They may have an optional biopsy of fat tissue.
During the first hospital visit, participants will learn how to give themselves shots of a drug (pegvisomant) that blocks growth hormone. The drug is injected under the skin. Participants will continue to give themselves these shots once a day at home.
After the first hospital visit, participants will talk on the phone with members of the study team once each week. After 2 weeks they will have blood drawn for tests.
Participants will stop the shots after the second hospital visit.

开始日期2023-01-23

申办/合作机构

National Institute of Diabetes & Digestive & Kidney Diseases

NCT05131100 / RecruitingN/A

Korean Post-marketing Surveillance for Somavert

Non-interventional observational study, to identify safety and effectiveness of Somavert during the post-marketing period based on the Korean RMP as required by the regulations of Ministry of Food and Drug Safety (MFDS)

开始日期2022-07-13

申办/合作机构

Pfizer Inc.

NCT04998500 / Active, not recruitingN/AIIT

Growth Hormone as a Model for Reversible Activation of Adipose Tissue Fibrosis

Background: Adipose tissue fibrosis denotes excessive pathological accumulation of extracellular matrix (ECM) in adipose tissue and is a marker of dysfunction. Growth hormone (GH) activates adipose tissue lipolysis and stimulates collagen synthesis in lean tissues. Intriguingly, we have novel pilot data to suggest that GH excess (acromegaly) also induces reversible fibrosis in vivo and potently activates the expression of fibroblast activation protein alpha (FAPα).
Hypothesis: GH induces adipose tissue fibrosis by increased FAPα expression together with proliferation and fibrogenic differentiation of fibro-adipogenic progenitor (FAP) cells.
Aim: To unravel the mechanisms underlying GH-induced adipose tissue fibrosis with emphasis on FAPα expression and proliferation of FAP cells.
Subjects and methods: In a single blinded, randomized, double-dummy crossover design, 10 adult, moderately overweight individuals will be subjected to one week of GH and GH receptor blockade (Pegvisomant). We will use single-cell technologies, fluorescence-activated cell sorting (FACS), RNA sequencing, and cell culture studies on adipose tissue samples, combined with in vivo assessment of adipose tissue turnover and metabolism.
Perspectives: Understanding fibrosis formation in human models may identify new targets for treatment of obesity-associated disorders.

开始日期2021-08-01

申办/合作机构

University of Aarhus

[+1]

100 项与培维索孟相关的临床结果

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100 项与培维索孟相关的转化医学

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100 项与培维索孟相关的专利（医药）

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551

项与培维索孟相关的文献（医药）

2024-04-01·BMJ Case Reports

Beware of epistaxis: fatal pseudoaneurysm rupture 30 years after treatment of acromegaly

Article

作者: Korbonits, Márta ; Carreira, Ana ; Grossman, Ashley B ; Muna, Solomon

We present a fatal complication of treatment in a patient with early-onset acromegaly, treated with two transsphenoidal operations, radiotherapy, radiosurgery and pegvisomant. He was diagnosed in his 30s, and controlled from his 40s, with stable residual tumour within the left cavernous sinus. In his 60s, 30 years after surgery/radiotherapy and 14 years after radiosurgery, he developed recurrent episodes of mild epistaxis. A week later, he presented at his local hospital’s emergency department with severe epistaxis and altered consciousness. He was diagnosed with a ruptured internal carotid artery (ICA) pseudoaneurysm, but unfortunately died before treatment could be attempted.ICA pseudoaneurysms are rare complications of surgery or radiotherapy and can present with several years of delay, often with epistaxis. This case highlights the importance of life-long monitoring in patients with previous pituitary interventions and early recognition of epistaxis as a herald sign of a potentially catastrophic event, thus leading to timely treatment.

2024-02-01·Best Practice & Research Clinical Endocrinology & Metabolism

Real-world value of cabergoline in the treatment of acromegaly

Review

作者: Shimon, Ilan

Cabergoline is an ergot derivative long-acting dopamine receptor 2 (DR2) selective agonist administered orally and widely used for the treatment of prolactin-secreting adenomas and Parkinson's disease. DR2 is expressed in most somatotroph adenomas. In acromegaly, cabergoline is used off-label and its role is limited by the relatively modest efficacy for achieving hormonal remission and thus, it is largely indicated in patients with mild elevation of GH/IGF-I postoperatively. It can be given as monotherapy, usually at a higher weekly dose than usually required to treat prolactinomas, but also as an add-on treatment in patients partially responding to the somatostatin receptor ligands octreotide or lanreotide. IGF-1 normalization with cabergoline can be achieved in about a third of the patients. Low baseline IGF-1 level (below 1.5 x ULN) before cabergoline initiation is a good predictor for remission. Combination treatment with the GH receptor antagonist pegvisomant can also be beneficial. The inexpensive, well-tolerated and convenient oral administration of cabergoline makes it an attractive medical therapy for active acromegaly.

2024-01-03·European Journal of Endocrinology

Pasireotide: potential treatment option for McCune–Albright-associated acromegaly

Article

作者: Raverot, Gérald ; Brac de la Perrière, Aude ; Ilie, Mirela-Diana

AbstractOnly 30% of patients with McCune–Albright syndrome (MAS)–associated acromegaly achieve biochemical control under first-generation somatostatin receptor ligands (fg-SRLs), while pegvisomant fails to normalize insulin-like growth factor 1 (IGF-I) in >20% of cases. Here, we report all the patients with MAS-associated acromegaly treated with pasireotide long-acting release (LAR) in our center. Pasireotide LAR 20 mg/month resulted in rapid and long-term IGF-I normalization in patients #1 and #3. Patient #3 was resistant to fg-SRLs, while patient #1 was also controlled on fg-SRLs. In patient #2, resistant to fg-SRLs and uncontrolled on pegvisomant 40 mg/day combined with cabergoline 0.5 mg/day, pegvisomant was replaced with pasireotide LAR 40 mg/month, resulting in the near normalization of IGF-I levels. All 3 patients developed intermittent impaired fasting glucose, without the need for glucose-lowering drugs. Thus, pasireotide LAR is clearly useful as third-line therapy, and potentially even as second-line therapy, in MAS-associated acromegaly.

1

项与培维索孟相关的新闻（医药）

2024-03-19

·BioSpace

Crinetics Bags Second Phase III Win for Acromegaly, Heads for NDA Submission

Pictured: Blood sample for insulin-like growth factor-1 (IGF-1)/iStock, Jarun011 Crinetics Pharmaceuticals has secured another late-stage win as its oral, once-daily investigational drug paltusotine aced a second Phase III study for acromegaly, a rare hormonal disorder. The biotech’s stock shot up over 10% Tuesday morning following the announcement. In the late-stage trial, 56% of patients taking paltusotine achieved the primary endpoint for the insulin-like growth factor 1 (IGF-1) level compared to 5% on placebo. The treatment also helped reduce symptoms of the disease—a secondary endpoint of the study. No serious adverse events were reported. Acromegaly, sometimes referred to as gigantism, is a serious hormonal disorder caused by a benign tumor in the pituitary gland that secretes growth hormone. Over 25,000 patients in the U.S. suffer from the rare disease, which results in enlargement of face, hands and feet. Left untreated, it can cause type 2 diabetes, hypertension, heart disease and other serious complications. Surgical removal of the tumor is the preferred treatment but many patients are not candidates for surgery. Current pharmacological options include painful monthly injections or Pfizer’s subcutaneous daily injection of Somavert. The Phase III data are the second positive trial results for Crinetics in acromegaly. In September 2023, the biotech shared data for a study of paltusotine in patients who switched from their current injections to Crinetics’ pill. Among treated patients, 83% were able to maintain their IGF-1 levels within the upper limit of normal, compared to only 4% of those on placebo. With these data in hand, Crinetics plans to submit its New Drug Application to the FDA in the second half of this year, while prepping for a 2025 launch. Jefferies analysts believe the market opportunity could be around $300 million for the company. Crinetics is also testing the drug candidate in carcinoid syndrome, reporting positive Phase II topline results last week. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

临床3期临床结果临床2期申请上市

100 项与培维索孟相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D05394	培维索孟	H01AX01	DB00082

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
肢端肥大症	欧盟	Pfizer Europe MA EEIG	2002-11-12
肢端肥大症	冰岛	Pfizer Europe MA EEIG	2002-11-12
肢端肥大症	列支敦士登	Pfizer Europe MA EEIG	2002-11-12
肢端肥大症	挪威	Pfizer Europe MA EEIG	2002-11-12

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
肉瘤	临床2期	美国	Pfizer Inc.	2009-11-01
肉瘤	临床2期	芬兰	Pfizer Inc.	2009-11-01
肉瘤	临床2期	加拿大	Pfizer Inc.	2009-11-01
糖尿病肾病	临床1期	美国	Sensus Drug Development Corp.	1998-09-01
去势抵抗性前列腺癌	临床前	德国	Pfizer Inc.	2009-11-01
去势抵抗性前列腺癌	临床前	美国	Pfizer Inc.	2009-11-01
去势抵抗性前列腺癌	临床前	加拿大	Pfizer Inc.	2009-11-01
糖尿病性视网膜病变	临床前	美国	Sensus Drug Development Corp.	1998-09-01
去势抵抗性前列腺癌	药物发现	芬兰	Pfizer Inc.	2009-11-01

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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


NCT00976508 (CTgov)	临床1期	结直肠癌 \| 晚期恶性实体瘤 \| 乳腺癌 \| 肉瘤 \| 肺癌 \| 去势抵抗性前列腺癌	24	Figitumumab+Pegvisomant (Figitumumab 20mg/kg + Pegvisomant 10 mg)	(鬱積淵憲鬱選築範憲網) = 鏇醖簾餘鹹衊糧壓蓋鬱壓願蓋鏇壓鏇顧糧願夢 (鏇積觸鬱齋憲觸齋糧衊, 艱蓋餘積選選鬱構簾願 ~ 衊獵廠蓋網製網顧壓鬱) 更多	-	2013-12-13
				Figitumumab+Pegvisomant (Figitumumab 20mg/kg + Pegvisomant 20 mg)	(鬱積淵憲鬱選築範憲網) = 製淵鹹淵築簾廠壓蓋築壓願蓋鏇壓鏇顧糧願夢 (鏇積觸鬱齋憲觸齋糧衊, 衊膚繭遞網願鹽構鹽構 ~ 夢選鹹艱網壓醖廠壓憲) 更多
ESPE2016	N/A	AIP mutation	2	Pegvisomant	(糧構觸廠餘鏇簾醖糧齋) = Her clinical course was complicated by cholelithiasis and abnormal liver function, induced by somatostatin and which resolved after cholecystectomy. 糧範網鏇膚願觸淵顧鹹 (齋餘襯繭襯築範窪淵範 ) 更多	-	2016-09-10
				Cabergoline
Pubmed \| J Clin Endocrinol Metab	N/A	肢端肥大症	-	Surgical therapy	網鏇遞鏇餘築醖鏇繭鹽(繭積衊衊鑰簾構廠顧獵) = 鹹壓鏇鏇夢壓鹹鬱簾廠夢繭廠簾壓衊醖壓蓋繭 (窪艱餘艱鬱蓋願鑰獵鹽 )	-	2019-11-01
				Pegvisomant therapy	網鏇遞鏇餘築醖鏇繭鹽(繭積衊衊鑰簾構廠顧獵) = 廠鬱鬱淵夢艱淵鑰夢衊夢繭廠簾壓衊醖壓蓋繭 (窪艱餘艱鬱蓋願鑰獵鹽 )
ESPE2015	N/A	肢端肥大症 GH \| IGF1 \| prolactin	-	Pegvisomant	(廠壓膚願鏇鏇顧鹹選襯) = 淵遞願顧鏇範鏇遞襯齋夢鬱餘窪夢鏇廠窪簾糧 (餘衊築餘網齋鹽窪襯夢 ) 更多	-	2015-10-01
				Cabergoline	(鬱簾選糧醖觸糧顧選糧) = 鬱憲鹹膚憲構製膚構蓋簾選淵獵窪艱顧願齋顧 (醖願廠膚醖積糧遞窪繭 )
ESPE2018	N/A	生长激素-分泌垂体腺瘤 IGF1 \| GH \| Prolactin ... 更多	5	Somatostatin anologue	鬱廠積繭鬱醖鹹糧壓膚(齋觸網構製製構製膚顧) = 繭觸衊壓觸鹽觸構窪鏇鏇廠築繭築構製遞範蓋 (積醖襯網襯衊壓顧窪網 ) 更多	-	2018-09-27
				Cabergoline	獵膚鑰築壓膚憲構築蓋(製鑰選簾餘膚觸鹽積製) = 鏇積齋艱鬱網願網遞製願蓋憲壓觸艱遞繭鑰觸 (積選繭鹹網餘簾艱蓋淵 ) 更多