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Phase 3 N=274 Treatment

Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis (CF) Patients With Pseudomonas Aeruginosa (PA)

Cystic Fibrosis

Enrolled (actual)
274
Serious AEs
50.4%
Results posted
Mar 2011
Primary outcome: Primary: Number of Participants Reporting Adverse Events (AEs) — 83; 185; 30; 79 participants

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
AZLI 75 mg two times a day (BID)/ three times a day (TID) (Drug)
Age
Pediatric, Adult, Older Adult · 6+ yrs
Sex
All
Sponsor
Gilead Sciences
Primary completion
Nov 2008

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Participants Reporting Adverse Events (AEs)
83; 185; 30; 79; 38; 100
PRIMARY
Number of Subjects With <15% or ≥15% Decline in Forced Expiratory Volume in 1 Second [FEV1] From Pretreatment to 30 Minutes After Treatment With AZLI
76; 175; 9; 14
PRIMARY
Change in Heart Rate (HR)
1.08; -0.07; -0.53; 0.13; 3.87; -0.06
PRIMARY
Change in Systolic and Diastolic Blood Pressure (BP)
0.75; 1.19; -0.67; 0.29; -1.06; -0.33
PRIMARY
Change in Temperature
-0.05; 0.00; -0.09; 0.01; 0.04; 0.05
PRIMARY
Change in Respiratory Rate (RR)
-0.07; 0.17; -0.03; 0.02; 0.02; 0.33
PRIMARY
Serum Hematology - Concentration of White Blood Cells (WBC), Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, and Platelets
9.28; 10.11; 9.47; 9.53; 0.05; 0.06
PRIMARY
Serum Hematology - Percent of Differential for Basophils, Eosinophils, Lymphocytes, Monocytes, and Neutrophils
0.59; 0.61; 0.59; 0.62; 2.05; 1.95
PRIMARY
Serum Hematology - Number of Red Blood Cells (RBC)
5.15; 4.94; 4.93; 4.77
PRIMARY
Serum Hematology - Hematocrit
43.43; 42.45; 42.00; 41.63
PRIMARY
Serum Hematology - Hemoglobin
14.06; 13.70; 13.89; 13.71
PRIMARY
Serum Hematology - Mean Corpuscular Volume (MCV)
84.33; 86.08; 85.38; 87.72
PRIMARY
Serum Hematology - Mean Corpuscular Hemoglobin (MCH)
27.43; 27.84; 28.35; 28.89
PRIMARY
Serum Hematology - Mean Corpuscular Hemoglobin Concentration (MCHC)
32.41; 32.34; 33.13; 32.99
PRIMARY
Serum Chemistry - Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), and Gamma-glutamlytransferase (GGT)
31.21; 26.65; 30.78; 27.81; 27.83; 25.77
PRIMARY
Serum Chemistry - Concentration of Calcium, Creatinine, Direct Bilirubin, Total Bilirubin, Serum Glucose, and Blood Urea Nitrogen
9.67; 9.56; 9.43; 9.47; 0.80; 0.81
PRIMARY
Serum Chemistry - Concentration of Chloride, Potassium, and Sodium
102.17; 102.22; 101.81; 102.16; 4.20; 4.29
PRIMARY
Serum Chemistry - Concentration of Total Protein
7.63; 7.58; 7.47; 7.53
SECONDARY
Change From Baseline in Pseudomonas Aeruginosa (PA) log10 Colony-forming Units (CFU) Per Gram of Sputum
-0.20; -0.81; -0.43; -0.53; -0.47; -0.60
SECONDARY
Number of Participants With Other Pathogens
33; 65; 34; 66; 41; 68
SECONDARY
Minimum Inhibitory Concentration (MIC) of Aztreonam
4; 4; 4; 8; 8; 8
SECONDARY
Percent Change in Pulmonary Function (FEV1, FEV1 Percent Predicted, FVC, FEF25-75)
4.87; 7.98; 3.55; 6.04; 1.31; 3.98
SECONDARY
Change in Clinical Symptoms as Assessed by the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Scale (CFQ-R RSS)
3.53; 6.83; 0.37; 7.34; 0.30; 6.01
SECONDARY
Time to First Hospitalization Due to a Respiratory Event
359; 417
SECONDARY
Change in Body Weight
0.58; 0.85; 1.31; 1.38; 3.24; 3.55
SECONDARY
Missed School/Work Days Due to CF Symptoms
2.00; 3.19
SECONDARY
Time to Intravenous (IV) Antipseudomonal Antibiotics
276; 232

Summary

The purpose of this study was to evaluate the safety and efficacy of multiple courses of AZLI in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).

Eligibility Criteria

Inclusion Criteria

  • Compliance with Studies CP-AI-005 (NCT00104520) or CP-AI-007 (NCT00112359) by taking at least 50% of expected study medication.
  • Completion of CP-AI-005 or CP-AI-007 or was withdrawn due to need for antipseudomonal antibiotics or for an AE unrelated to study medication tolerance.
  • Ability to provide written informed consent/assent prior to initiation of study-related procedures.
  • Ability to perform reproducible pulmonary function tests.

Exclusion Criteria

  • Use of any investigational medication or device between the last visit of CP-AI-005 or CP-AI-007 and Visit 1 of this study.
  • Concurrent participation in a study of another investigational drug or device.
  • Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg prednisone/day or 20 mg prednisone every other day.
  • History of sputum or throat swab culture yielding Burkholderia cepacia in the previous 2 years.
  • History of daily continuous oxygen supplementation or requirement for more than 2 liters/minute at night.
  • Inability to tolerate study medication in CP-AI-005 or CP-AI-007.
  • Known local or systemic hypersensitivity to aztreonam.
  • Inability to tolerate inhalation of a short acting beta-2 agonist.
  • Abnormal renal or hepatic function based on results of most recent test.
  • Female of child-bearing potential who was pregnant, lactating, or not (in the opinion of the investigator) practicing an acceptable method of birth control.
  • Any serious or active medical or psychiatric illness which, in the opinion of the investigator, would have interfered with participant treatment, assessment, or compliance with the protocol.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00128492). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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