Clinical trial NCT00434278: A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Cystic Fibrosis
Keywords: Lung disease
CF
Pulmozyme
Lead sponsor: Genentech, Inc.
Sponsor class: INDUSTRY
First posted: 2007-02-13
Enrollment: 27
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This was a multicenter, randomized, double-blind, placebo-controlled study of patients with severe, though stable, cystic fibrosis (CF) whose routine treatment included Pulmozyme. Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group). Patients must have had stable CF symptoms without any change in therapy for 2 weeks prior to enrollment in order to participate.


Eligibility criteria: Inclusion Criteria:

* Signed Informed Consent Form and, if applicable, Assent Form
* Age ≥ 14 years old at screening
* Proven diagnosis of CF
* Ability to perform acceptable and reproducible spirometry maneuvers at screening
* FVC ≤ 45% predicted for race, height, age, and sex at screening
* Chronic use of Pulmozyme for at least 3 months in the last year and daily or twice daily use during the 14 days prior to screening
* Stable regimen of chest physiotherapy (CPT) for at least 14 days prior to screening
* Ability to complete the 6-minute walk test at screening
* Ability to complete the 6-minute walk test and spirometry at Visit 2
* If on routine tobramycin solution for inhalation (i.e., TOBI®) or other cycled antibiotic therapy, intention to either start or stop therapy at the time of Visit 2 (based on routine therapy cycle; no other planned change in this antibiotic regimen for 28 days before or 28 days after Visit 2)
* Clinically stable with no change in medications during the 14 days prior to screening

Exclusion Criteria:

* Use of an investigational drug or device within 28 days prior to screening
* Previous episode of acute respiratory failure requiring assisted ventilation within 2 years prior to screening
* Previous lung transplant
* Any cardiac disease that would contraindicate performing the 6-minute walk test
* Pregnancy or nursing
* Known hypersensitivity or other contraindication to the use of Pulmozyme
* Previous completion or premature discontinuation of study drug or withdrawal from this study
* More than one prior screening failure for this study at any time or any prior screening failure for this study within the last 28 days

Primary outcomes:
measure: Change in Distance Walked in the 6-minute Walk Test
timeFrame: From baseline to Day 14
Secondary outcomes:
measure: Change in Pulmonary Function as Measured by FEV1 and FVC
timeFrame: From baseline to Day 14
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Los Angeles
country: United States
facility: Childrens Hospital of LA
state: California
city: Los Angeles
country: United States
facility: USC Adult CF Center
state: California
city: Orange
country: United States
facility: CHOC
state: California
city: Sacramento
country: United States
facility: Capital Allergy Resp Dis Ctr
state: California
city: Ventura
country: United States
facility: Ventura County Medical Ctr
state: California
city: Denver
country: United States
facility: Nat'l Jewish Med/Research Ctr
state: Colorado
city: Jacksonville
country: United States
facility: Pulm & Critical Care Assoc
state: Florida
city: Miami
country: United States
facility: Univ of Miami
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Grou
state: Florida
city: Atlanta
country: United States
facility: Brown Univ School of Medicine
state: Georgia
city: Augusta
country: United States
facility: Medical College of Georgia
state: Georgia
city: Chicago
country: United States
facility: Rush Medical Center
state: Illinois
city: Lexington
country: United States
facility: Univ of Kentucky Med Ctr
state: Kentucky
city: Ann Arbor
country: United States
facility: Univ of Michigan Hlth System
state: Michigan
city: Detroit
country: United States
facility: Wayne State University
state: Michigan
city: Grand Rapids
country: United States
facility: Spectrum Hospital
state: Michigan
city: Kalamazoo
country: United States
facility: Michigan State University
state: Michigan
city: Minneapolis
country: United States
facility: Univ of Minnesota Dept of Derm
state: Minnesota
city: Jackson
country: United States
facility: University of Mississippi
state: Mississippi
city: Saint Louis
country: United States
facility: Pulmonary & Critical Care Med
state: Missouri
city: Las Vegas
country: United States
facility: Children's Lung Specialist
state: Nevada
city: Long Branch
country: United States
facility: Monmouth Medical Center
state: New Jersey
city: Morristown
country: United States
facility: Morristown Memorial Hospital
state: New Jersey
city: New Brunswick
country: United States
facility: St. Peters Univ Hospital
state: New Jersey
city: Albuquerque
country: United States
facility: University of New Mexico
state: New Mexico
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: New Hyde Park
country: United States
facility: Long Island Jew Adult CF Ctr
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical Univ
state: New York
city: Durham
country: United States
facility: Duke Pediatric Clinical
state: North Carolina
city: Cleveland
country: United States
facility: Rainbow Babies & Child Hosp
state: Ohio
city: Toledo
country: United States
facility: Toledo Childrens Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: Santiago Reyes MD-Private Prac
state: Oklahoma
city: Tulsa
country: United States
facility: CF Solutions, Inc
state: Oklahoma
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Charleston
country: United States
facility: Medical Univ of South Carolina
state: South Carolina
city: Columbia
country: United States
facility: Pediatric Pulmonary Assoc
state: South Carolina
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: San Antonio
country: United States
facility: Alamo Clinical Research Assoc
state: Texas
city: Morgantown
country: United States
facility: W Virginia Univ Health Sci Ctr
state: West Virginia
Study officials:
affiliation: Genentech, Inc.
name: Michelle Freemer, M.D.
role: STUDY_DIRECTOR
References: