Journalartikel
Autorenliste: Frantz, Robert P.; Benza, Raymond L.; Channick, Richard N.; Chin, Kelly; Howard, Luke S.; McLaughlin, Vallerie V.; Sitbon, Olivier; Zamanian, Roham T.; Hemnes, Anna R.; Cravets, Matt; Bruey, Jean-Marie; Roscigno, Robert; Mottola, David; Elman, Erin; Zisman, Lawrence S.; Ghofrani, Hossein-Ardeschir
Jahr der Veröffentlichung: 2021
Zeitschrift: Pulmonary Circulation
Bandnummer: 11
Heftnummer: 4
ISSN: 2045-8932
eISSN: 2045-8940
Open Access Status: Gold
DOI Link: https://doi.org/10.1177/20458940211057071
Verlag: Wiley
Abstract:
Aberrant kinase signaling that involves platelet-derived growth factor receptor (PDGFR) alpha/beta, 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 PDGFR alpha/beta, 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.
Zitierstile
Harvard-Zitierstil: Frantz, R., Benza, R., Channick, R., Chin, K., Howard, L., McLaughlin, V., et al. (2021) TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension, Pulmonary Circulation, 11(4), Article 20458940211057071. https://doi.org/10.1177/20458940211057071
APA-Zitierstil: Frantz, R., Benza, R., Channick, R., Chin, K., Howard, L., McLaughlin, V., Sitbon, O., Zamanian, R., Hemnes, A., Cravets, M., Bruey, J., Roscigno, R., Mottola, D., Elman, E., Zisman, L., & Ghofrani, H. (2021). TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension. Pulmonary Circulation. 11(4), Article 20458940211057071. https://doi.org/10.1177/20458940211057071
Schlagwörter
BMPR2; C-KIT; CSF1R; GB002; PDGFR; pulmonary vascular remodeling
