NCT01375036

Brief Summary

This is a prospective, longitudinal, 5-year study that will enroll participants from the existing Cystic Fibrosis Foundation (CFF) patient registry. Each enrolled participant will provide samples for microbiological evaluation, obtained upon enrollment and then once per year thereafter for 5 years.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
510

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Aug 2011

Longer than P75 for all trials

Geographic Reach
1 country

31 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 15, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 17, 2011

Completed
2 months until next milestone

Study Start

First participant enrolled

August 1, 2011

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2016

Completed
Last Updated

January 11, 2017

Status Verified

January 1, 2017

Enrollment Period

5.3 years

First QC Date

June 15, 2011

Last Update Submit

January 9, 2017

Conditions

Keywords

Cystic fibrosisPAPseudomonas aeruginosaCFaztreonam

Outcome Measures

Primary Outcomes (1)

  • Proportion of participants whose least susceptible Pseudomonas aeruginosa (PA) isolate has a ≥ 4-fold increase in aztreonam minimum inhibitory concentration over 1 year and is above the parenteral breakpoint (> 8 μg/mL)

    This proportion will be compared annually over 5 years.

    Up to 5 years

Secondary Outcomes (5)

  • Annual mean change and mean change from baseline in FEV1 (liters) and FEV1 % predicted

    Baseline to Year 5

  • Annual number of hospitalizations and the total number of hospitalizations at the end of each year

    Up to 5 years

  • Annual number of days hospitalized and the total number of hospitalization days at the end of each year

    Up to 5 years

  • Annual mean change and mean change from baseline in body mass index (BMI)

    Baseline to Year 5

  • Annual number of Cayston treatment courses per participant and the total number of Cayston treatment courses at the end of each year in participants that used Cayston

    Up to 5 years

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

A representative cross-section of individuals with mild (FEV1 75% to ≤ 90% predicted), moderate (FEV1 40% to 74% predicted), or severe (FEV1 \<40% predicted) lung disease and a history of ≥ 2 lower respiratory tract cultures positive for PA (at any time) will be enrolled. This will include approximately 100 individuals with pre-study exposure to Cayston (defined as having received at least one prior 28-day course of Cayston at any time before enrolling into the study). Enrollment will be monitored and potentially modified to ensure that adequate numbers of participants in each disease severity category are included. At the Investigator's discretion, participants enrolled in this study can be allowed to participate in clinical trials with other investigational therapies as appropriate.

You may qualify if:

  • Current participant or willingness to participate in the CFF patient registry database
  • ≥ 6 years of age
  • Subject 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 (NPD) AND accompanying clinical characteristics consistent with CF. For subjects who lack documentation of either a positive sweat chloride test or an abnormal NPD, and who have only one well-characterized genetic mutation of the CFTR gene, the diagnosis of CF is determined by the Investigator.
  • FEV1 ≥ 25% predicted and ≤ 90% predicted.
  • ≥ 2 lower respiratory tract cultures positive for PA with results documented in the subject's medical history.
  • Subject 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.

You may not qualify if:

  • Any serious active medical or psychiatric illness that, in the opinion of the Investigator, would interfere with subject assessment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (31)

Providence Alaska Medical Center

Anchorage, Alaska, 99508, United States

Location

University of Arizona

Tucson, Arizona, 85724, United States

Location

Arkansas Children's Hospital Research Institute

Little Rock, Arkansas, 72202, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

The Children's Hospital

Denver, Colorado, 80045, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

Central Florida Pulmonary Group

Altamonte, Florida, 32701, United States

Location

Nemours Children's Clinic

Orlando, Florida, 32801, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Hospitals & Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Children's Lung Specialists

Las Vegas, Nevada, 89107, United States

Location

Albany Medical College

Albany, New York, 12208, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Cincinati Children's Hosptial Medical Center

Cincinnati, Ohio, 45229, United States

Location

University of Cincinnati Medical Center

Cincinnati, Ohio, 45267, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

The Children's Medical Center of Dayton

Dayton, Ohio, 45404, United States

Location

Drexel University College of Medicine

Philadelphia, Pennsylvania, 19102, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53201, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (1)

  • Keating CL, Zuckerman JB, Singh PK, McKevitt M, Gurtovaya O, Bresnik M, Marshall BC, Saiman L. Pseudomonas aeruginosa Susceptibility Patterns and Associated Clinical Outcomes in People with Cystic Fibrosis following Approval of Aztreonam Lysine for Inhalation. Antimicrob Agents Chemother. 2021 Feb 17;65(3):e02327-20. doi: 10.1128/AAC.02327-20. Print 2021 Feb 17.

Biospecimen

Retention: SAMPLES WITHOUT DNA

Pseudomonas aeruginosa isolates from sputum or throat swab specimens

MeSH Terms

Conditions

Cystic FibrosisPseudomonas Infections

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesGram-Negative Bacterial InfectionsBacterial InfectionsBacterial Infections and MycosesInfections

Study Officials

  • Gilead Study Director

    Gilead Sciences

    STUDY DIRECTOR

Study Design

Study Type
observational
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 15, 2011

First Posted

June 17, 2011

Study Start

August 1, 2011

Primary Completion

December 1, 2016

Study Completion

December 1, 2016

Last Updated

January 11, 2017

Record last verified: 2017-01

Locations