A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy (CAT) Regimen of Inhaled Antibiotics for the Treatment of Chronic Pulmonary Pseudomonas aeruginosa Infection in Subjects with Cystic Fibrosis

Study ID
STU 092012-074

Cancer Related
No

Healthy Volunteers
No

Study Sites

  • UT Southwestern-Clinical Translational Research Center (CTRC)
  • UT Southwestern Ambulatory Services
  • UT Southwestern University Hospital—St. Paul
  • Children's Medical Center-Dallas

Contact
Ashley Keller
214-648-2817
ashley.keller@utsouthwestern.edu

Principal Investigator
Raksha Jain

Summary

This study is a 7-month, randomized, double-blind, placebo-controlled, multicenter trial to be
conducted at approximately 80 centers in the uS. The study period will last up to 30 weeks
and involve up to 9 visits (Screening Visit 1 and enrollment Visit 2 may be combined).
enrolled subjects will receive 28 days of TiS during the run-in period. Subjects that continue
to be eligible for the study will be randomized with a 1:1 allocation to receive Treatment 1
(3 cycles of aZLi alternating with TiS) or Treatment 2 (3 cycles of placebo alternating with
TiS). TiS treatment will be open-label while study drug treatment will be double-blinded.

Participant Eligibility

1. Males or females >= 6 years of age
2. Diagnosis of CF as determined by the 1997 CF Consensus Conference criteria: At least
1 or more accompanying clinical features consistent with CF AND
- Documented sweat chloride >= 60 mEq/L by quantitative pilocarpine iontophoresis test
OR
- Abnormal nasal transepithelial potential difference (NPD) test
OR
- Two well-characterized, disease-causing genetic mutations in the CF transmembrane
conductance regulator (CFTR) gene
3. Documented presence of PA in 2 lower respiratory tract cultures within the 12 months
prior to or at Screening (Visit 1). Cultures must be a minimum of 3 months apart, with
the most recent PA-positive culture within 3 months prior to Screening Visit 1. A
PA-positive sputum or throat swab culture at Screening (Visit 1) can qualify as the most
recent PA-positive culture.
4. Subject must be able to perform reproducible pulmonary function tests (PFTs)
5. FEV1 >= 25% and <= 75% predicted at Screening (Visit 1)
6. History of at least 1 hospitalization or 1 course of IV antibiotics for an acute respiratory
exacerbation within the previous 12 months from Screening
7. Clinically stable with no evidence of significant respiratory symptoms or, if obtained for
clinical evaluation, no acute chest radiograph findings at Screening (Visit 1) or
Enrollment (Visit 2) that would require administration of IV antibiotics, oxygen
supplementation, or hospitalization
8. Chest radiograph without significant acute findings (eg, infiltrates [lobar or diffuse
interstitial], pleural effusion, pneumothorax); or chest radiograph, CT, or MRI obtained
and interpreted within the 90 days prior to randomization, without acute findings and no
significant intercurrent illness; chronic, stable findings (eg, chronic scarring or
atelectasis) are allowed
9. Subjects must be able to provide written informed consent (or assent if applicable) prior
to any study related procedures; parent/guardian must be able to give written informed
consent prior to any study related procedure
10. A negative urine pregnancy test is required for female subjects of childbearing potential
(see Section 1)
11. All sexually active female subjects who are not postmenopausal, or surgically sterile, or
who do not have medically documented ovarian failure and are of childbearing potential
must agree to use highly effective contraception (per Section 7.8.2) during heterosexual
intercourse throughout the study and for 30 days after the last dose of TIS or study drug;
females utilizing hormonal contraceptives as a birth control method must have used the
same method for at least 3 months prior to start of TIS treatment
12. Male subjects who are sexually active are required to use barrier contraception (condom
with spermicide) during heterosexual intercourse and refrain from sperm donation from
screening through to study completion and for 90 days from the last dose of TIS or study
drug