Clinical Trial Results:
Expanded Access Program for Aztreonam Lysine for Inhalation in Canadian Patients with Cystic Fibrosis and Pseudomonas aeruginosa Airway Infection Who Have Limited Treatment Options and are at Risk for Disease Progression
Summary
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EudraCT number |
2015-000397-36 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
27 Nov 2012
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Results information
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Results version number |
v1(current) |
This version publication date |
22 Mar 2016
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First version publication date |
05 Aug 2015
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
EA-US-205-0122
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT00989807 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Gilead Sciences
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Sponsor organisation address |
333 Lakeside Drive, Foster City, CA, United States, 94404
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Public contact |
Clinical Trial Mailbox, Gilead Sciences International Ltd, ClinicalTrialDisclosures@gilead.com
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Scientific contact |
Clinical Trial Mailbox, Gilead Sciences International Ltd, ClinicalTrialDisclosures@gilead.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
27 Nov 2012
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
27 Nov 2012
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Global end of trial reached? |
Yes
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Global end of trial date |
27 Nov 2012
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The primary objective of this study is to provide expanded access to AZLI 75 mg prior to its commercial availability and establishment of reimbursement programs through Provincial Ministries of Health to patients in Canada with CF and PA airway infection who have limited treatment options and are at risk for disease progression.
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Protection of trial subjects |
The protocol and consent/assent forms were submitted by each investigator to a duly constituted Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for review and approval before study initiation. All revisions to the consent/assent forms (if applicable) after initial IEC/IRB approval were submitted by the investigator to the IEC/IRB for review and approval before implementation in accordance with regulatory requirements.
This study was conducted in accordance with recognized international scientific and ethical standards, including but not limited to the International Conference on Harmonization guideline for Good Clinical Practice (ICH GCP) and the original principles embodied in the Declaration of Helsinki.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
08 Feb 2010
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Canada: 45
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Worldwide total number of subjects |
45
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
3
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Adults (18-64 years) |
41
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
Participants were enrolled at study sites in Canada. The first participant was screened on 08 February 2010. The last study visit occurred on 27 November 2012. | ||||||||||||||||
Pre-assignment
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Screening details |
45 participants were screened. | ||||||||||||||||
Period 1
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Period 1 title |
Overall study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||||
Arms
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Arm title
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All participants | ||||||||||||||||
Arm description |
Aztreonam for inhalation solution (AZLI) 3 times daily in 56-day cycles (28 days on treatment followed by 28 days off treatment). | ||||||||||||||||
Arm type |
Experimental | ||||||||||||||||
Investigational medicinal product name |
Aztreonam for inhalation solution
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Investigational medicinal product code |
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Other name |
AZLI, Cayston®
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Pharmaceutical forms |
Powder for nebuliser solution
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Routes of administration |
Inhalation use
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Dosage and administration details |
AZLI 75 mg administered 3 times daily usung the eFlow nebulizer
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Baseline characteristics reporting groups
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Reporting group title |
All participants
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Reporting group description |
Aztreonam for inhalation solution (AZLI) 3 times daily in 56-day cycles (28 days on treatment followed by 28 days off treatment). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
All participants
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Reporting group description |
Aztreonam for inhalation solution (AZLI) 3 times daily in 56-day cycles (28 days on treatment followed by 28 days off treatment). |
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End point title |
Percentage of participants experiencing any serious adverse event [1] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
From date of first dose to study discontinuation (average 73 weeks) plus 30 days
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No statistical analysis was planned or performed. |
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No statistical analyses for this end point |
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Adverse events information [1]
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Timeframe for reporting adverse events |
From date of first dose to study discontinuation (average 73 weeks) plus 30 days
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Adverse event reporting additional description |
All AEs are reported by system order class and preferred term as determined by the investigator.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
17.0
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Reporting groups
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Reporting group title |
All participants
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: Per protocol, non-serious adverse events were not collected in this expanded access study. |
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |