Program to Give the Drug Aztreonamn Lysine for Inhalation to Canadian Patients with Cystic Fibrosis and infection by the Bacteria Pseudomonas aeruginosa Who Have Limited Treatment Options and are at Risk for Disease Progression

• Register: EUCTR
• Last refreshed on: 16 February 2015
• Main ID: EUCTR2015-000397-36-Outside-EU/EEA
• Date of registration: 03/02/2015
• Prospective Registration: Yes
• Primary sponsor: Gilead Sciences, Inc.
• Public title: Program to Give the Drug Aztreonamn Lysine for Inhalation to Canadian Patients with Cystic Fibrosis and infection by the Bacteria Pseudomonas aeruginosa Who Have Limited Treatment Options and are at Risk for Disease Progression
• Scientific title: 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
• Target sample size: 45
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000397-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Canada

Contacts

• Name: Clinical Trials Information
• Affiliation: Gilead Sciences International Ltd

• Name: Clinical Trials Information
• Affiliation: Gilead Sciences International Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
> 6 years of age
Patient has CF as diagnosed by one of the following:
-Documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis
test, or
-Two well characterized genetic mutations in the CFTR gene, or
-Abnormal nasal potential difference with accompanying symptoms characteristic of
CF
PA present in expectorated sputum or throat swab culture within 2 months prior to
consent
Patient must be able to provide written informed consent/assent prior to any study related
procedure; parent/guardian must be able to give written informed consent as necessary
prior to any study related procedure
At high risk for disease progression as defined by one of the following patient
populations:
FEV1 < 50 00 predicted at the time of consent
Or
Completed participation in CP-Al-006 (through Visit 20). Patients who withdraw
from CP-Al-006 prior to completing all courses of AZLI and all study visits will
not be eligible for this protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 3
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 42
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients with any serious or active medical or psychiatric illness that, in the opinion of the investigator, would interfere with patient treatment, assessment or compliance with the protocol or dosing requirements
Patients with hypersensitivity to any of the components of the drug product
Currently enrolled in another clinical trial
Pregnant or lactating females


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Adult and paediatric cystic fibrosis (CF) patients with pulmonary Pseudomonas aeruginosa (PA) infection.
MedDRA version: 17.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Intervention(s)

Trade Name: Cayston
Aztreonam 75 mg powder and solvent for nebuliser solution
Pharmaceutical Form: Powder and solvent for nebuliser solution
INN or Proposed INN: AZTREONAM
CAS Number: 78110-38-0
Current Sponsor code: AZLI
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-

Primary Outcome(s)

Secondary Objective: Not applicable.

Main Objective: 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.

Primary end point(s): Not applicable: Expanded Access Program

Timepoint(s) of evaluation of this end point: Not applicable

Secondary Outcome(s)

Secondary end point(s): Not applicable

Timepoint(s) of evaluation of this end point: Not applicable

Secondary ID(s)

EA-US-205-0122

Source(s) of Monetary Support

Gilead Sciences, Inc.