Pitfalls of Drug Development: Lessons Learned from Trials of Denufosol in Cystic Fibrosis

被引：36

作者：

Moss, Richard B. ^{[1
]}

机构：

[1] Stanford Univ, Dept Pediat, Sch Med, Stanford, CA 94305 USA

来源：

JOURNAL OF PEDIATRICS | 2013年 / 162卷 / 04期

关键词：

INHALED HYPERTONIC SALINE; P2Y(2) RECEPTOR AGONIST; INHALATION SOLUTION; LUNG-FUNCTION; TETRASODIUM; SAFETY; CLEARANCE; INFANTS; TMEM16A; STRESS;

D O I：

10.1016/j.jpeds.2012.11.034

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

引用

页码：676 / 680

页数：5

共 42 条

[1] Denufosol Tetrasodium in Patients with Cystic Fibrosis and Normal to Mildly Impaired Lung Function [J].

Accurso, Frank J. ;

Moss, Richard B. ;

Wilmott, Robert W. ;

Anbar, Ran D. ;

Schaberg, Amy E. ;

Durham, Todd A. ;

Ramsey, Bonnie W. .

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2011, 183 (05) :627-634

[2] Long-Term Inhaled Dry Powder Mannitol in Cystic Fibrosis An International Randomized Study [J].

Aitken, Moira L. ;

Bellon, Gabriel ;

De Boeck, Kris ;

Flume, Patrick A. ;

Fox, Howard G. ;

Geller, David E. ;

Haarman, Eric G. ;

Hebestreit, Helge U. ;

Lapey, Allen ;

Schou, I. Manjula ;

Zuckerman, Jonathan B. ;

Charlton, Brett .

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2012, 185 (06) :645-652

[3] Evidence for airway surface dehydration as the initiating event in CF airway disease [J].

Boucher, R. C. .

JOURNAL OF INTERNAL MEDICINE, 2007, 261 (01) :5-16

[4]

Büscher R, 2006, PHARMACOGENET GENOM, V16, P199

[5] Role of mechanical stress in regulating airway surface hydration and mucus clearance rates [J].

Button, Brian ;

Boucher, Richard C. .

RESPIRATORY PHYSIOLOGY & NEUROBIOLOGY, 2008, 163 (1-3) :189-201

[6] TMEM16A, a membrane protein associated with calcium-dependent chloride channel activity [J].

Caputo, Antonella ;

Caci, Emanuela ;

Ferrera, Loretta ;

Pedemonte, Nicoletta ;

Barsanti, Cristina ;

Sondo, Elvira ;

Pfeffer, Ulrich ;

Ravazzolo, Roberto ;

Zegarra-Moran, Olga ;

Galietta, Luis J. V. .

SCIENCE, 2008, 322 (5901) :590-594

[7] Identifying treatments that halt progression of pulmonary disease in cystic fibrosis [J].

Davis, PB ;

Byard, PJ ;

Konstan, MW .

PEDIATRIC RESEARCH, 1997, 41 (02) :161-165

[8] Hitting the Target New Treatments for Cystic Fibrosis [J].

Davis, Stephanie D. ;

Ferkol, Thomas W. .

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2010, 182 (12) :1460-1461

[9] Safety and tolerability of denufosol tetrasodium inhalation solution, a novel P2Y2 receptor agonist:: Results of a phase 1/phase 2 multicenter study in mild to moderate cystic fibrosis [J].

Deterding, R ;

Retsch-Bogart, G ;

Milgram, L ;

Gibson, R ;

Daines, C ;

Zeitlin, PL ;

Milla, C ;

Marshall, B ;

LaVange, L ;

Engels, J ;

Mathews, D ;

Gorden, J ;

Schaberg, A ;

Williams, J ;

Ramsey, B .

PEDIATRIC PULMONOLOGY, 2005, 39 (04) :339-348

[10] Phase 2 randomized safety and efficacy trial of nebulized denufosol tetrasodium in cystic fibrosis [J].

Deterding, Robin R. ;

LaVange, Lisa M. ;

Engels, Jean M. ;

Mathews, Dave W. ;

Coquillette, Sarah J. ;

Brody, Alan S. ;

Millard, Steve P. ;

Ramsey, Bonnie W. .

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2007, 176 (04) :362-369

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