TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension

Aberrant kinase signaling that involves platelet-derived growth factor receptor (PDGFR) α/β, colony stimulating factor 1 receptor (CSF1R), and stem cell factor receptor (c-KIT) pathways may be responsible for vascular remodeling in pulmonary arterial hypertension. Targeting these specific pathways m...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Robert P. Frantz, Raymond L. Benza, Richard N. Channick, Kelly Chin, Luke S. Howard, Vallerie V. McLaughlin, Olivier Sitbon, Roham T. Zamanian, Anna R. Hemnes, Matt Cravets, Jean-Marie Bruey, Robert Roscigno, David Mottola, Erin Elman, Lawrence S. Zisman, Hossein-Ardeschir Ghofrani
Formato:	article
Lenguaje:	EN
Publicado:	SAGE Publishing 2021
Materias:	Diseases of the circulatory (Cardiovascular) system RC666-701 Diseases of the respiratory system RC705-779
Acceso en línea:	https://doaj.org/article/e7c6ee3ea34a41f89721a2c6c04b54de
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:e7c6ee3ea34a41f89721a2c6c04b54de
record_format	dspace
spelling	oai:doaj.org-article:e7c6ee3ea34a41f89721a2c6c04b54de2021-11-13T23:03:26ZTORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension2045-894010.1177/20458940211057071https://doaj.org/article/e7c6ee3ea34a41f89721a2c6c04b54de2021-11-01T00:00:00Zhttps://doi.org/10.1177/20458940211057071https://doaj.org/toc/2045-8940Aberrant kinase signaling that involves platelet-derived growth factor receptor (PDGFR) α/β, colony stimulating factor 1 receptor (CSF1R), and stem cell factor receptor (c-KIT) pathways may be responsible for vascular remodeling in pulmonary arterial hypertension. Targeting these specific pathways may potentially reverse the pathological inflammation, cellular proliferation, and fibrosis associated with pulmonary arterial hypertension progression. Seralutinib (formerly known as GB002) is a novel, potent, clinical stage inhibitor of PDGFRα/β, CSF1R, and c-KIT delivered via inhalation that is being developed for patients with pulmonary arterial hypertension. Here, we report on an ongoing Phase 2 randomized, double-blind, placebo-controlled trial (NCT04456998) evaluating the efficacy and safety of seralutinib in subjects with World Health Organization Group 1 Pulmonary Hypertension who are classified as Functional Class II or III. A total of 80 subjects will be enrolled and randomized to receive either study drug or placebo for 24 weeks followed by an optional 72-week open-label extension study. The primary endpoint is the change from baseline to Week 24 in pulmonary vascular resistance by right heart catheterization. The secondary endpoint is the change in distance from baseline to Week 24 achieved in the 6-min walk test. A computerized tomography sub-study will examine the effect of seralutinib on pulmonary vascular remodelling. A separate heart rate monitoring sub-study will examine the effect of seralutinib on cardiac effort during the 6-min walk test.Robert P. FrantzRaymond L. BenzaRichard N. ChannickKelly ChinLuke S. HowardVallerie V. McLaughlinOlivier SitbonRoham T. ZamanianAnna R. HemnesMatt CravetsJean-Marie BrueyRobert RoscignoDavid MottolaErin ElmanLawrence S. ZismanHossein-Ardeschir GhofraniSAGE PublishingarticleDiseases of the circulatory (Cardiovascular) systemRC666-701Diseases of the respiratory systemRC705-779ENPulmonary Circulation, Vol 11 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Diseases of the circulatory (Cardiovascular) system RC666-701 Diseases of the respiratory system RC705-779
spellingShingle	Diseases of the circulatory (Cardiovascular) system RC666-701 Diseases of the respiratory system RC705-779 Robert P. Frantz Raymond L. Benza Richard N. Channick Kelly Chin Luke S. Howard Vallerie V. McLaughlin Olivier Sitbon Roham T. Zamanian Anna R. Hemnes Matt Cravets Jean-Marie Bruey Robert Roscigno David Mottola Erin Elman Lawrence S. Zisman Hossein-Ardeschir Ghofrani TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension
description	Aberrant kinase signaling that involves platelet-derived growth factor receptor (PDGFR) α/β, colony stimulating factor 1 receptor (CSF1R), and stem cell factor receptor (c-KIT) pathways may be responsible for vascular remodeling in pulmonary arterial hypertension. Targeting these specific pathways may potentially reverse the pathological inflammation, cellular proliferation, and fibrosis associated with pulmonary arterial hypertension progression. Seralutinib (formerly known as GB002) is a novel, potent, clinical stage inhibitor of PDGFRα/β, CSF1R, and c-KIT delivered via inhalation that is being developed for patients with pulmonary arterial hypertension. Here, we report on an ongoing Phase 2 randomized, double-blind, placebo-controlled trial (NCT04456998) evaluating the efficacy and safety of seralutinib in subjects with World Health Organization Group 1 Pulmonary Hypertension who are classified as Functional Class II or III. A total of 80 subjects will be enrolled and randomized to receive either study drug or placebo for 24 weeks followed by an optional 72-week open-label extension study. The primary endpoint is the change from baseline to Week 24 in pulmonary vascular resistance by right heart catheterization. The secondary endpoint is the change in distance from baseline to Week 24 achieved in the 6-min walk test. A computerized tomography sub-study will examine the effect of seralutinib on pulmonary vascular remodelling. A separate heart rate monitoring sub-study will examine the effect of seralutinib on cardiac effort during the 6-min walk test.
format	article
author	Robert P. Frantz Raymond L. Benza Richard N. Channick Kelly Chin Luke S. Howard Vallerie V. McLaughlin Olivier Sitbon Roham T. Zamanian Anna R. Hemnes Matt Cravets Jean-Marie Bruey Robert Roscigno David Mottola Erin Elman Lawrence S. Zisman Hossein-Ardeschir Ghofrani
author_facet	Robert P. Frantz Raymond L. Benza Richard N. Channick Kelly Chin Luke S. Howard Vallerie V. McLaughlin Olivier Sitbon Roham T. Zamanian Anna R. Hemnes Matt Cravets Jean-Marie Bruey Robert Roscigno David Mottola Erin Elman Lawrence S. Zisman Hossein-Ardeschir Ghofrani
author_sort	Robert P. Frantz
title	TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension
title_short	TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension
title_full	TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension
title_fullStr	TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension
title_full_unstemmed	TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension
title_sort	torrey, a phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension
publisher	SAGE Publishing
publishDate	2021
url	https://doaj.org/article/e7c6ee3ea34a41f89721a2c6c04b54de
work_keys_str_mv	AT robertpfrantz torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT raymondlbenza torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT richardnchannick torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT kellychin torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT lukeshoward torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT vallerievmclaughlin torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT oliviersitbon torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT rohamtzamanian torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT annarhemnes torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT mattcravets torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT jeanmariebruey torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT robertroscigno torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT davidmottola torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT erinelman torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT lawrenceszisman torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension AT hosseinardeschirghofrani torreyaphase2studytoevaluatetheefficacyandsafetyofinhaledseralutinibforthetreatmentofpulmonaryarterialhypertension
_version_	1718430110528307200

TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension

Ejemplares similares