期刊
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
卷 183, 期 5, 页码 627-634出版社
AMER THORACIC SOC
DOI: 10.1164/rccm.201008-1267OC
关键词
chloride channel activator; early intervention; P2Y(2) receptor agonist; ion channel regulator; ENaC inhibition
资金
- Inspire Pharmaceuticals
- Inspire Pharmaceuticals Inc.
- Vertex Pharmaceuticals
- PTC Therapeutics
- Digestive Care
- Gilead Sciences
- Altus Pharmaceuticals
- Kalobios Pharmaceuticals
- Axcan Pharmaceuticals
- INO therapeutics
- Yahoo Health
- MPEX Pharmaceuticals
- PTC Corp.
- Vertex
- Gilead
- Novartis
- UpToDate
- Boehringer-Ingelheim
- Cystic Fibrosis Foundation
- Bayer Pharmaceuticals
- Genentech
- Axcan
- Altus
- AlgiPharma AS
- Altana Pharma Inc.
- Amgen Inc.
- Aradigm Corporation
- Axcan Pharma
- Bayer Healthcare AG
- Berna Bio Tech AG
- Chiesi Pharmaceuticals Inc.
- Chiron Corporation
- Corus Pharma Inc.
- CSL Behring LLC
- Digestive Care Inc.
- Eurand
- Extera Partners LLC
- Galephar PR
- Genaera Corporation
- Genentech Inc.
- Gilead Sciences Inc.
- GlaxoSmithKline
- PLC
- GlycoMimetics
- JJ PRO LLC
- Kalo Bios Pharmaceuticals Inc.
- MerLion Pharmaceuticals GmbH
- Mpex Pharmaceuticals Inc.
- MPM Asset Management LLC
- N30 Pharmaceuticals LLC
- NITROX LLC
- Novartis Pharmaceuticals Corp.
- Peninsula Pharmaceuticals Inc.
- Pharmaxis Ltd
- PTC Therapeutics Inc.
- Seed-One Ventures LLC
- SEER Pharmaceuticals LLC
- Solvay Pharmaceuticals Inc.
- Sucampo Pharmaceuticals Inc.
- Syngenta AG
- Seeds Division
- Transave Inc.
- Vectura Ltd
- Vertex Pharmaceuticals Inc.
- ViaSys Manufacturing Inc.
- National Institutes of Health
Rationale: Intervention for cystic fibrosis lung disease early in its course has the potential to delay or prevent progressive changes that lead to irreversible airflow obstruction. Denufosol is a novel ion channel regulator designed to correct the ion transport defect and increase the overall mucociliary clearance in cystic fibrosis lung disease by increasing chloride secretion, inhibiting sodium absorption, and increasing ciliary beat frequency in the airway epithelium independently of cystic fibrosis transmembrane conductance regulator genotype. Objectives: To evaluate the efficacy and safety of denufosol in patients with cystic fibrosis who had normal to mildly impaired lung function characteristic of early cystic fibrosis Methods: A total of 352 patients greater than or equal to 5 years old with cystic fibrosis who had FEV1 greater than or equal to 75% of predicted normal were randomized to receive inhaled denufosol, 60 mg, or placebo three times daily in a Phase 3, randomized, double-blind, placebo-controlled, 24-week trial. Measurements and Main Results: Main outcome measures included change in FEV1 from baseline to Week 24 endpoint and adverse events. Mean change from baseline to Week 24 endpoint in FEV1 (primary efficacy endpoint) was 0.048 L for denufosol (n = 178) and 0.003 L for placebo (n = 174; P = 0.047). No significant differences between groups were observed for secondary endpoints including exacerbation rate and other measures of lung function. Denufosol was well tolerated with adverse event and growth profiles similar to placebo. Conclusions: Denufosol improved lung function relative to placebo in cystic fibrosis patients with normal to mildly impaired lung function.
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