End-points and biomarkers for clinical trials in cystic fibrosis

European Respiratory Monograph

Volumn 64, Issue , 2014, Pages 104-115

  De Boeck, Kris ^a   Fajac, Isabelle ^b,c   Ratjen, Felix ^d  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b UNIVERSITÉ PARIS DESCARTES   (France)

^c Institut Cochin   (France)

^d UNIVERSITY OF TORONTO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBIOTIC AGENT; ANTIINFLAMMATORY AGENT; BIOLOGICAL MARKER; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE RECEPTOR; LEVOFLOXACIN; UNCLASSIFIED DRUG;

ARTICLE; BACTERIUM CULTURE; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; DISEASE EXACERBATION; DISEASE MARKER; DRUG EFFICACY; FORCED EXPIRATORY VOLUME; HUMAN; IMAGING; LUNG CLEARANCE; LUNG DISEASE; LUNG LAVAGE; MEDICAL RESEARCH; METHODOLOGY; MULTICENTER STUDY (TOPIC); NUCLEAR MAGNETIC RESONANCE IMAGING; OUTCOME ASSESSMENT; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); QUALITY OF LIFE INDEX; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAMPLE SIZE; SPUTUM EXAMINATION; STUDY DESIGN; STUDY END POINT; SURROGATE END POINT;

EID: 84905740139     PISSN: 1025448X     EISSN: 20756674     Source Type: Book Series    
DOI: 10.1183/1025448x.10009313     Document Type: Article

References (54)

1. De Gruttola VG, Clax P, DeMets DL, et al. Considerations in the evaluation of surrogate end-points in clinical trials. Summary of a National Institutes of Health workshop. Control Clin Trials 2001; 22: 485-502.
2. Atkinson A, Colburn W, De Gruttola V, et al. Biomarkers and surrogate end-points: preferred definitions and conceptual framework. Clin Pharmacol Ther 2001; 69: 89-95.
3. Bilton D, Canny G, Conway S, et al. Pulmonary exacerbation: towards a definition for use in clinical trials. Report from the EuroCareCF Working Group on outcome parameters in clinical trials. J Cyst Fibros 2011; 10: Suppl. 2, S79-S81.
4. Quittner AL, Modi AC, Wainwright C, et al. Determination of the minimal clinically important difference scores for the Cystic Fibrosis Questionnaire-Revised respiratory symptom scale in two populations of patients with cystic fibrosis and chronicPseudomonas aeruginosaairway infection. Chest 2009; 135: 1610-1618.
5. De Boeck K, Kent L, Davies J, et al. CFTR biomarkers: time for promotion to surrogate end-point. Eur Respir J2013; 41: 203-216.
6. Subbarao P, Stanojevic S, Brown M, et al. Lung clearance index as an outcome measure for clinical trials in young children with cystic fibrosis. A pilot study using inhaled hypertonic saline. Am J Respir Crit Care Med 2013; 188: 456-460.
7. Kent L, Reix P, Innes JA, et al. Lung clearance index: evidence for use in clinical trials in cystic fibrosis. J Cyst Fibros 2014; 13: 123-138.
8. Fayon M, Kent L, Bui S, et al. Clinimetric properties of bronchoalveolar lavage inflammatory markers in cystic fibrosis. Eur Respir J2014; 43: 610-626.
9. Loeve M, Krestin GP, Rosenfeld M, et al. Chest computed tomography: a validated surrogate end-point of cystic fibrosis lung disease?Eur Respir J2013; 42: 844-857.
10. Stick S, Tiddens H, Aurora P, et al. Early intervention studies in infants and preschool children with cystic fibrosis: are we ready?Eur Respir J2013; 42: 527-538.
11. Simpson SJ, Mott LS, Esther CR Jr, et al. Novel end points for clinical trials in young children with cystic fibrosis. Expert Rev Respir Med 2013; 7: 231-243.
12. Amin R, Ratjen F. Cystic fibrosis. In: Kolb M, Vogelmeier CF. eds. Outcomes in Clinical Trials. ERS Monogr 2013; 62: 54-69.
13. EMA. Workshop on end-points for cystic fibrosis clinical trials. www. ema. europa. eu/ema/index. jsp?curl5pages/news_and_events/events/2012/07/event_detail_000609. jsp&mid5WC0b01ac058004d5c3Date last updated: 2012. Date last accessed: November 22, 2013.
14. Tiddens HA, Stick SM, Davis S. Multi-modality monitoring of cystic fibrosis lung disease: the role of chest computed tomography. Paediatr Respir Rev 2014; 15: 92-97.
15. Mott LS, Park J, Murray CP, et al. Progression of early structural lung disease in young children with cystic fibrosis assessed using CT. Thorax 2012; 67: 509-516.
16. De Boeck K. Trying to find a cure for cystic fibrosis: CFTR biomarkers as outcomes. Eur Respir J2013; 42: 1156-1157.
17. Waters V, Stanojevic S, Atenafu EG, et al. Effect of pulmonary exacerbations on long-term lung function decline in cystic fibrosis. Eur Respir J2012; 40: 61-66.
18. Goss CH, Burns JL. Exacerbations in cystic fibrosis. 1: Epidemiology and pathogenesis. Thorax 2007; 62: 360-367.
19. Aurora P, Stanojevic S, Wade A, et al. Lung clearance index at 4 years predicts subsequent lung function in children with cystic fibrosis. Am J Respir Crit Care Med 2010; 183: 752-758.
20. Vermeulen F, Proesmans M, Boon M, et al. Lung clearance index predicts pulmonary exacerbations in young patients with cystic fibrosis. Thorax 2014; 69: 39-45.
21. Rowe SM, Liu B, Hill A, et al. Optimizing nasal potential difference analysis for CFTR modulator development: assessment of ivacaftor in CF subjects with the G551D-CFTR mutation. PLoS One 2013; 8: e66955.
22. Sly PD, Gangell CL, Chen L, et al. Risk factors for bronchiectasis in children with cystic fibrosis. N Engl J Med 2013; 368: 1963-1670.
23. Sagel SD, Wagner BD, Anthony MM, et al. Sputum biomarkers of inflammation and lung function decline in children with cystic fibrosis. Am J Respir Crit Care Med 2012; 186: 857-865.
24. Mayer-Hamblett N, Aitken ML, Accurso FJ, et al. Association between pulmonary function and sputum biomarkers in cystic fibrosis. Am J Respir Crit Care Med 2007; 175: 822-828.
25. Lynch SV, Bruce KD. The cystic fibrosis airway microbiome. Cold Spring Harb Perspect Med 2013; 3: a009738.
26. Mayer-Hamblett N, Ramsey BW, Kronmal RA. Advancing outcome measures for the new era of drug development in cystic fibrosis. Proc Am Thorac Soc 2007; 4: 370-377.
27. EMA. ICH Topic E 8-General Considerations for Clinical Trials. www. ema. europa. eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500002877. pdf Date last updated: 2006. Date last accessed: March 25, 2014.
28. Kerem E, Reisman J, Corey M, et al. Prediction of mortality in patients with cystic fibrosis. N Engl J Med 1992; 326: 1187-1191.
29. Huang NN, Schidlow DV, Szatrowski TH, et al. Clinical features, survival rate, and prognostic factors in young adults with cystic fibrosis. Am J Med 1987; 82: 871-879.
30. Corey M, McLaughlin FJ, Williams M, et al. A comparison of survival, growth, and pulmonary function in patients with cystic fibrosis in Boston and Toronto. J Clin Epidemiol 1988; 41: 583-591.
31. Postmarketing Studies and Clinical Trials: Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act. www. fda. gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM172001. pdf Date last updated: 2011. Date last accessed: November 22, 2013.
32. Guideline on the clinical development of medicinal products for the treatment of cystic fibrosis. www. ema. europa. eu/docs/en_GB/document_library/Scientific_guideline/2009/12/WC500017055. pdf Date last updated: 2009. Date last accessed: November 22, 2013.
33. Que C, Cullinan P, Geddes D. Improving rate of decline of FEV1in young adults with cystic fibrosis. Thorax 2006; 61: 155-157.
34. Rosenfeld M, Ratjen F, Brumback L, et al. Inhaled hypertonic saline in infants and children younger than 6 years with cystic fibrosis: the ISIS randomized controlled trial. JAMA 2012; 307: 2269-2277.
35. Robinson TE, Goris ML, Zhu HJ, et al. Dornase alfa reduces air trapping in children with mild cystic fibrosis lung disease: a quantitative analysis. Chest 2005; 128: 2327-2335.
36. Konstan MW, Wagener JS, Yegin A, et al. Design and powering of cystic fibrosis clinical trials using rate of FEV1 decline as an efficacy end-point. J Cyst Fibros 2010; 9: 332-338.
37. Willemink MJ, de Jong PA. Pediatric chest computed tomography at a radiation dose approaching a chest radiograph. Am J Respir Crit Care Med 2013; 188: 626-627.
38. Konstan MW, Byard PJ, Hoppel CL, et al. Effect of high-dose ibuprofen in patients with cystic fibrosis. N Engl J Med 1995; 332: 848-854.
39. Konstan MW, Schluchter MD, Xue W, et al. Clinical use of Ibuprofen is associated with slower FEV1 decline in children with cystic fibrosis. Am J Respir Crit Care Med 2007; 176: 1084-1089.
40. Ratjen F, Saiman L, Mayer-Hamblett N, et al. Effect of azithromycin on systemic markers of inflammation in patients with cystic fibrosis uninfected withPseudomonas aeruginosa. Chest 2012; 142: 1259-1266.
41. Geller DE, Flume PA, Staab D, et al. Levofloxacin inhalation solution (MP-376) in patients with cystic fibrosis with Pseudomonas aeruginosa. Am J Respir Crit Care Med 2011; 183: 1510-1516.
42. Clancy JP, Dupont L, Konstan MW, et al. Phase II studies of nebulised Arikace in CF patients withPseudomonas aeruginosainfection. Thorax 2013; 68: 818-825.
43. Trapnell BC, McColley SA, Kissner DG, et al. Fosfomycin/tobramycin for inhalation in patients with cystic fibrosis with pseudomonas airway infection. Am J Respir Crit Care Med 2011; 185: 171-178.
44. Maiz L, Giron RM, Olveira C, et al. Inhaled antibiotics for the treatment of chronic bronchopulmonaryPseudomonas aeruginosainfection in cystic fibrosis: systematic review of randomised controlled trials. Expert Opin Pharmacother 2013; 14: 1135-1149.
45. Fleming TR, Odem-Davis K, Rothmann MD, et al. Some essential considerations in the design and conduct of noninferiority trials. Clin Trials 2011; 8: 432-439.
46. Ballmann M, Rabsch P, von der Hardt H. Long-term follow up of changes in FEV1and treatment intensity during Pseudomonas aeruginosacolonisation in patients with cystic fibrosis. Thorax 1998; 53: 732-737.
47. Taylor-Robinson D, Whitehead M, Diderichsen F, et al. Understanding the natural progression in % FEV1decline in patients with cystic fibrosis: a longitudinal study. Thorax 2012; 67: 860-866.
48. Littlewood JM, Miller MG, Ghoneim AT, et al. Nebulised colomycin for early pseudomonas colonisation in cystic fibrosis. Lancet 1985; 1: 865.
49. Valerius NH, Koch C, Hoiby N. Prevention of chronicPseudomonas aeruginosacolonisation in cystic fibrosis by early treatment. Lancet 1991; 338: 725-726.
50. Ratjen F, Doring G, Nikolaizik WH. Effect of inhaled tobramycin on earlyPseudomonas aeruginosacolonisation in patients with cystic fibrosis. Lancet 2001; 358: 983-984.
51. Ratjen F, Munck A, Kho P, et al. Treatment of earlyPseudomonas aeruginosainfection in patients with cystic fibrosis: the ELITE trial. Thorax 2010; 65: 286-291.
52. Taccetti G, Bianchini E, Cariani L, et al. Early antibiotic treatment forPseudomonas aeruginosaeradication in patients with cystic fibrosis: a randomised multicentre study comparing two different protocols. Thorax 2012; 67: 853-859.
53. Accurso FJ, Rowe SM, Clancy JP, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med 2010; 363: 1991-2003.
54. Dekkers JF, Wiegerinck CL, de Jonge HR, et al. A functional CFTR assay using primary cystic fibrosis intestinal organoids. Nat Med 2013; 19: 939-945.