Program to Give the Drug Aztreonam Lysine for Inhalation to 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: EUCTR2015-000396-26-Outside-EU/EEA
• Date of registration: 02/02/2015
• Prospective Registration: Yes
• Primary sponsor: Gilead Sciences, Inc.
• Public title: Program to Give the Drug Aztreonam Lysine for Inhalation to 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 Patients with Cystic Fibrosis and Pseudomonas aeruginosa Airway Infection Who Have Limited Treatment Options and are at Risk for Disease Progression
• Target sample size: 603
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000396-26
• 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

United States

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:
> 6years 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:
-First Cohort Criteria:
Those who are waitlisted or eligible for lung transplant based on FEV1 criteria.
Patients who have a level of lung function impairment consistent with lung transplantation criteria, but who are ineligible for transplantation for other reasons, can enroll in this program.
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.
- Second Cohort Criteria:
Meets first cohort criteria; or
FEV1 < 40 % predicted at the time of consent
- Third Cohort Criteria:
Meets first or second cohort criteria; or
FEV1 < 50 % predicted at the time of consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 108
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 495
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.

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)

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

Main Objective: The primary objective of this program is to provide expanded access to aztreonam lysine for inhalation 75 mng prior to its
commercial availability to patients with cystic fibrosis and chronic P. aeruginosa airway infection who have limited treatment options and are at risk for disease progression.

Secondary Objective: Not applicable

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-205-0111

Source(s) of Monetary Support

Gilead Sciences, Inc.