Regulatory aspects of phase 3 endpoints for new inhaled antibiotics for cystic fibrosis patients with chronic pseudomonas aeruginosa infections

Journal of Aerosol Medicine and Pulmonary Drug Delivery

Volumn 25, Issue 4, 2012, Pages 198-203

  Montgomery, Alan Bruce ^a   Abuan, Tammy ^a   Yeager, Melissa A ^a  

^a Cardeas Pharma   (United States)

Author keywords

aztreonam solution for inhalation; clinical endpoints; cystic fibrosis; inhaled antibiotics; pseudomonas endobronchial infections; regulatory approval; tobramycin solution for inhalation

Indexed keywords

ANTIBIOTIC AGENT; ANTIINFECTIVE AGENT; AZTREONAM; PLACEBO; TOBRAMYCIN;

AEROSOL; CHRONIC DISEASE; CYSTIC FIBROSIS; DISEASE ASSOCIATION; DISEASE EXACERBATION; DRUG EFFICACY; FOOD AND DRUG ADMINISTRATION; FORCED EXPIRATORY VOLUME; HUMAN; LUNG FUNCTION; MORBIDITY; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRACTICE GUIDELINE; PSEUDOMONAS INFECTION; REVIEW; SPIROMETRY; STATISTICAL SIGNIFICANCE; TREATMENT OUTCOME; UNSPECIFIED SIDE EFFECT;

ADMINISTRATION, INHALATION; ANTI-BACTERIAL AGENTS; CLINICAL TRIALS, PHASE III AS TOPIC; COMORBIDITY; CYSTIC FIBROSIS; ENDPOINT DETERMINATION; HUMANS; LUNG; PRACTICE GUIDELINES AS TOPIC; PSEUDOMONAS AERUGINOSA; PSEUDOMONAS INFECTIONS; RESPIRATORY FUNCTION TESTS;

EID: 84864710041     PISSN: 19412711     EISSN: 19412703     Source Type: Journal    
DOI: 10.1089/jamp.2011.0911     Document Type: Review

References (23)

1. Flume PA, O'Sullivan BP, Robinson KA, Goss CH, Mogayzel PJ Jr, Willey-Courand DB, Bujan J, Finder J, Lester M, Quittell L, Rosenblatt R, Vender RL, Hazle L, Sabadosa K, Marshall B, and the Cystic Fibrosis Foundation, Pulmonary Therapies Committee: Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2007;176:957-969.
2. EMA Guidelines for development of drugs for cystic fibrosis: Available at: http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/ 2009/12/WC500017055.pdf (Accessed August 18, 2011).
3. Workshop on endpoints for CF drugs: Available at: (www.fda.gov/downloads/ Drugs/NewsEvents/UCM231055.pdf + endpoints + CF + transcripts&client= FDAgov&lr=&proxystylesheet=FDAgov&output=xml-no-dtd&ie= UTF8&site=FDAgov&access=p&oe = UTF-8) (Accessed August 18, 2011).
4. Cystic Fibrosis Foundation Patient Registry: 2009 Annual Data Report to the Center Directors. Cystic Fibrosis Foundation, Bethesda, MD; 2010.
5. Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams-Warren J, Vasiljev-KM, Borowitz D, Bowman CM, Marshall BC, and Smith AL: Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. N Engl J Med. 1999;341:23-30.
6. Retsch-Bogart GZ, Quittner AL, Gibson RL, Oermann CM, McCoy KS, Montgomery AB, and Cooper PJ: Efficacy and safety of inhaled aztreonam lysine for airway Pseudomonas in cystic fibrosis. Chest. 2009;135:1222-1232.
7. Oermann CM, Assael B, Nakamura C, Boas SR, Bresnik M, Montgomery AB, and Pressler T: Aztreonam for inhalation solution (AZLI) vs. tobramycin inhalation solution, a 6 month comparative trial in cystic fibrosis. Pediatr Pulmonol. 2010;S33:327.
8. McCoy KS, Quittner AL, Oermann CM, Gibson RL, Retsch-Bogart GZ, and Montgomery AB: Inhaled aztreonam lysine for chronic airway Pseudomonas aeruginosa in cystic fibrosis. Am J Respir Crit Care Med. 2008;178:921-928.
9. Kerem E, Reisman J, Corey M, Canny GJ, and Levison H: Prediction of mortality in patients with cystic fibrosis. N Engl J Med. 1992;326:1187-1191.
10. Milla CE, and Warwick WJ: Risk of death in cystic fibrosis patients with severely compromised lung function. Chest. 1998;113:1230-1234.
11. Wainwright CE, Quittner AL, Geller DE, Nakamura C, Wooldridge JL, Gibson RL, Lewis S, and Montgomery AB: Aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis, mild lung impairment, and P. aeruginosa. J Cystic Fibrosis. 2011;10:234-242.
12. Murphy TD, Anbar RD, Lester LA, Nasr SZ, Nickerson B, VanDevanter DR, and Colin AA: Treatment with tobramycin solution for inhalation reduces hospitalizations in young CF subjects with mild lung disease. Pediatr Pulmonol. 2004;38:314-320.
13. Quittner AL, Buu A, Messer MA, Modi AC, and Watrous M: Development and validation of the cystic fibrosis questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis. Chest. 2005;128:2347-2354.
14. FDA Guidance on Patient Reported Outcomes: Available at: http://www.fda.gov/cder/guidance/5460dft.pdf (Accessed August 18, 2011).
15. Quittner AL, Modi AC,Wainwright C, Otto K, Kirihara J, and Montgomery AB: 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 chronic Pseudomonas aeruginosa airway infection. Chest. 2009;135:1610-1618.
16. Oermann CM, Retsch-Bogart GZ, Quittner AL, Gibson RL, McCoy KS, Montgomery AB, and Cooper PJ: An 18-month study, AIR-CF3, of the safety and improvement in pulmonary function and respiratory symptoms with repeated courses of aztreonam for inhalation solution in patients with cystic fibrosis and airway Pseudomonas aeruginosa. Pediatr Pulmonol. 2010;45:1121-1134.
17. Goss CH, and Quittner AL: Patient reported outcomes in cystic fibrosis. Proc Am Thorac Soc. 2007;4:378-386.
18. Donaldson SH, Bennett WD, Zeman KL, Knowles MR, Tarran R, and Boucher RC: Mucus clearance and lung function in cystic fibrosis with hypertonic saline. N Engl J Med. 2006;354:241-250.
19. Goss CH, Edwards TC, Ramsey BW, Aitken ML, and Patrick DL: Patient-reported respiratory symptoms in cystic fibrosis. J Cystic Fibrosis. 2009;8:245-252.
20. Dakin C, Henry R, Field P, and Morton J: Defining an exacerbation of pulmonary disease in cystic fibrosis. Pediatr Pulmonol. 2001 31:436-442.
21. Rosenfeld M, Emerson J, Williams-Warren J, Pepe M, Smith A, Montgomery AB, and Ramsey B. Defining a pulmonary exacerbation in cystic fibrosis. J Pediatr. 2001;139:359-365.
22. Trapnell BC, Rolfe M, McColley S, Montgomery AB, Moorehead L, and Geller D. Fosformycin/tobramycin for inhalation: efficacy results of a phase 2 placebo-controlled trial in patients with cystic fibrosis. Pediatr Pulmonol. 2010;S33:302.
23. Tobramycin inhalation solution summary basis for approval. Available at: www.accessdata.fda.gov/drugsatfda-docs/nda/97/50753-mr.pdf (Accessed August 18, 2011).