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.

Start Date23 Jan 2023

Sponsor / Collaborator

National Institute of Diabetes & Digestive & Kidney Diseases

NCT05131100 / RecruitingNot Applicable

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)

Start Date13 Jul 2022

Sponsor / Collaborator

Pfizer Inc.

NCT04998500 / Active, not recruitingNot ApplicableIIT

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.

Start Date01 Aug 2021

Sponsor / Collaborator

University of Aarhus

[+1]

100 Clinical Results associated with Pegvisomant

100 Translational Medicine associated with Pegvisomant

100 Patents (Medical) associated with Pegvisomant

564

Literatures (Medical) associated with Pegvisomant

01 Oct 2024·Growth Hormone & IGF Research

Medical treatment of acromegaly – When the tumor size matters: A narrative review

Review

Author: Stojanovic, Marko ; Milicevic, Mihajlo ; Doknic, Mirjana ; Miljic, Dragana

Medical treatment of acromegaly is generally positioned as a second line of treatment after pituitary adenoma surgery. With the rising availability and variety of medications for acromegaly increases our understanding of their effectiveness and safety. Volume of the published data on the impact of medical therapy on biochemical control of acromegaly, contrasts a relative lack of publications which comprehensively address pituitary tumor alterations under different drug modalities. Assessment of changes in GH-secreting adenoma volume is often overshadowed by clinicians' focus on GH and IGF-I levels during acromegaly treatment. Close analysis of studies published in the last two decades, reveals that both an increase and decrease in somatotropinoma volume are possible during treatment with any of available drugs for acromegaly. Changes in pituitary tumor size may arise from the biological nature of adenoma itself, independently of the administered medications. Therefore, an individual approach is necessary in the treatment of patients with acromegaly, based on repeated insight to their clinical, biochemical, pathological and imaging characteristics. In this review, we summarize and comment how pituitary tumor size is affected by the treatment with all currently available drugs in acromegaly: long-acting somatostatin receptor ligands of the first generation (octreotide LAR and lanreotide autogel) and the second generation (pasireotide-LAR), as well as pegvisomant (PEG) and cabergoline (CAB).

12 Sep 2024·Endocrine Reviews

The Genetic Pathophysiology and Clinical Management of the TADopathy, X-Linked Acrogigantism

Review

Author: Beckers, Albert ; Daly, Adrian F

AbstractPituitary gigantism is a rare manifestation of chronic growth hormone (GH) excess that begins before closure of the growth plates. Nearly half of patients with pituitary gigantism have an identifiable genetic cause. X-linked acrogigantism (X-LAG; 10% of pituitary gigantism) typically begins during infancy and can lead to the tallest individuals described. In the 10 years since its discovery, about 40 patients have been identified. Patients with X-LAG usually develop mixed GH and prolactin macroadenomas with occasional hyperplasia that secrete copious amounts of GH, and frequently prolactin. Circulating GH-releasing hormone is also elevated in a proportion of patients. X-LAG is caused by constitutive or sporadic mosaic duplications at chromosome Xq26.3 that disrupt the normal chromatin architecture of a topologically associating domain (TAD) around the orphan G-protein–coupled receptor, GPR101. This leads to the formation of a neo-TAD in which GPR101 overexpression is driven by ectopic enhancers (“TADopathy”). X-LAG has been seen in 3 families due to transmission of the duplication from affected mothers to sons. GPR101 is a constitutively active receptor with an unknown natural ligand that signals via multiple G proteins and protein kinases A and C to promote GH/prolactin hypersecretion. Treatment of X-LAG is challenging due to the young patient population and resistance to somatostatin analogs; the GH receptor antagonist pegvisomant is often an effective option. GH, insulin-like growth factor 1, and prolactin hypersecretion and physical overgrowth can be controlled before definitive adult gigantism occurs, often at the cost of permanent hypopituitarism.

01 Sep 2024·Clinical Endocrinology

Acromegaly management in the Nordic countries: A Delphi consensus survey

Article

Author: Akerman, Anna-Karin ; Burman, Pia ; Ragnarsson, Oskar ; Rintamäki, Reeta ; Immonen, Heidi ; Olsson, Daniel ; Nevalainen, Pasi ; Cederberg-Tamminen, Henna ; Jørgensen, Jens Otto Lunde ; Klose, Marianne Christina ; Bollerslev, Jens ; Engstrom, Britt Eden ; Oksnes, Marianne ; Schalin-Jäntti, Camilla ; Krogh, Jesper ; Berinder, Katarina ; Metso, Saara ; Olsson, Tommy ; Sigurjónsdóttir, Helga Ágústa ; Hoybye, Charlotte ; Jarlov, Anne ; Dahlqvist, Per ; Dal, Jakob ; Nielsen, Torben Leo ; Arlien-Søborg, Mai C ; Vehkavaara, Satu ; Petersson, Maria ; Nermoen, Ingrid ; Andreassen, Mikkel ; Jaatinen, Pia ; Nuutila, Pirjo ; Husted, Eigil ; Stochholm, Kirstine ; Heck, Ansgar ; Feltoft, Claus Larsen ; Johannsson, Gudmundur ; Andersen, Marianne Skovsager ; Matikainen, Niina ; Yliaska, Iina ; Hämäläinen, Päivi ; Rasmussen, Åse Krogh ; Mäkimattila, Sari ; Feldt-Rasmussen, Ulla ; Wahlberg, Jeanette ; Christensen, Louise Lehmann ; Fougner, Stine Lyngvi ; Ekman, Bertil ; Viukari, Marianna

AbstractObjectiveAcromegaly is associated with increased morbidity and mortality if left untreated. The therapeutic options include surgery, medical treatment, and radiotherapy. Several guidelines and recommendations on treatment algorithms and follow‐up exist. However, not all recommendations are strictly evidence‐based. To evaluate consensus on the treatment and follow‐up of patients with acromegaly in the Nordic countries.MethodsA Delphi process was used to map the landscape of acromegaly management in Denmark, Sweden, Norway, Finland, and Iceland. An expert panel developed 37 statements on the treatment and follow‐up of patients with acromegaly. Dedicated endocrinologists (n = 47) from the Nordic countries were invited to rate their extent of agreement with the statements, using a Likert‐type scale (1−7). Consensus was defined as ≥80% of panelists rating their agreement as ≥5 or ≤3 on the Likert‐type scale.ResultsConsensus was reached in 41% (15/37) of the statements. Panelists agreed that pituitary surgery remains first line treatment. There was general agreement to recommend first‐generation somatostatin analog (SSA) treatment after failed surgery and to consider repeat surgery. In addition, there was agreement to recommend combination therapy with first‐generation SSA and pegvisomant as second‐ or third‐line treatment. In more than 50% of the statements, consensus was not achieved. Considerable disagreement existed regarding pegvisomant monotherapy, and treatment with pasireotide and dopamine agonists.ConclusionThis consensus exploration study on the management of patients with acromegaly in the Nordic countries revealed a relatively large degree of disagreement among experts, which mirrors the complexity of the disease and the shortage of evidence‐based data.

News (Medical) associated with Pegvisomant

19 Mar 2024

·www.biospace.com

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.

Phase 3Clinical ResultPhase 2NDA

100 Deals associated with Pegvisomant

External Link

KEGG	Wiki	ATC	Drug Bank
D05394	Pegvisomant	H01AX01	DB00082

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Acromegaly	EU	Pfizer Europe MA EEIG	12 Nov 2002
Acromegaly	NO	Pfizer Europe MA EEIG	12 Nov 2002
Acromegaly	LI	Pfizer Europe MA EEIG	12 Nov 2002
Acromegaly	IS	Pfizer Europe MA EEIG	12 Nov 2002

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Colorectal Cancer	Preclinical	CA	Pfizer Inc.	01 Nov 2009
Colorectal Cancer	Preclinical	FI	Pfizer Inc.	01 Nov 2009
Lung Cancer	Preclinical	CA	Pfizer Inc.	01 Nov 2009
Lung Cancer	Preclinical	DE	Pfizer Inc.	01 Nov 2009
Lung Cancer	Preclinical	US	Pfizer Inc.	01 Nov 2009
Lung Cancer	Preclinical	FI	Pfizer Inc.	01 Nov 2009
Sarcoma	Preclinical	DE	Pfizer Inc.	01 Nov 2009
Sarcoma	Preclinical	FI	Pfizer Inc.	01 Nov 2009
Sarcoma	Preclinical	CA	Pfizer Inc.	01 Nov 2009
Sarcoma	Preclinical	US	Pfizer Inc.	01 Nov 2009

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Pubmed \| J Clin Endocrinol Metab	Not Applicable	Acromegaly	-	Surgical therapy	gkuqdbmyxr(sxyefeaqya) = xupehxpzjo oafvvlkaxk (wotvhtebya )	-	01 Nov 2019
				Pegvisomant therapy	gkuqdbmyxr(sxyefeaqya) = wajqyhasuc oafvvlkaxk (wotvhtebya )
ESPE2016	Not Applicable	AIP mutation	2	Pegvisomant	(lcyuezzzbz) = Her clinical course was complicated by cholelithiasis and abnormal liver function, induced by somatostatin and which resolved after cholecystectomy. mzhabfpsmb (vfuoacpgbj ) View more	-	10 Sep 2016
				Cabergoline
ESPE2015	Not Applicable	Acromegaly GH \| IGF1 \| prolactin	-	Pegvisomant	(nigmokseuv) = dfjgmvpbfv cebbfgfhsj (trwkuofwau ) View more	-	01 Oct 2015
				Cabergoline	(opmvbxhtzb) = cflhbqzigh tmulwcybkb (phcprihwdp )

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis