A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)
- Registration Number
- NCT00434278
- Lead Sponsor
- Genentech, Inc.
- 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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 27
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo placebo - Dornase alfa Dornase alfa -
- Primary Outcome Measures
Name Time Method Change in Distance Walked in the 6-minute Walk Test From baseline to Day 14 Change in distance walked was defined as (distance walked in 6 minutes at baseline \[Day 0\]) - (distance walked in 6 minutes at Day 14) in meters.
- Secondary Outcome Measures
Name Time Method Change in Pulmonary Function as Measured by FEV1 and FVC From baseline to Day 14 FEV1 (forced expiratory volume in 1 second) and FVC (forced vital capacity) were recorded at Day 0 (baseline) and Day 14. Change in FEV1 and FVC from baseline to Day 14 was reported as a percentage of values predicted for age, height, and race.
Trial Locations
- Locations (40)
Ventura County Medical Ctr
🇺🇸Ventura, California, United States
Pulm & Critical Care Assoc
🇺🇸Jacksonville, Florida, United States
Morristown Memorial Hospital
🇺🇸Morristown, New Jersey, United States
Toledo Childrens Hospital
🇺🇸Toledo, Ohio, United States
Univ of Kentucky Med Ctr
🇺🇸Lexington, Kentucky, United States
Pediatric Pulmonary Assoc
🇺🇸Columbia, South Carolina, United States
W Virginia Univ Health Sci Ctr
🇺🇸Morgantown, West Virginia, United States
USC Adult CF Center
🇺🇸Los Angeles, California, United States
Brown Univ School of Medicine
🇺🇸Atlanta, Georgia, United States
Rush Medical Center
🇺🇸Chicago, Illinois, United States
Rainbow Babies & Child Hosp
🇺🇸Cleveland, Ohio, United States
Nat'l Jewish Med/Research Ctr
🇺🇸Denver, Colorado, United States
Univ of Miami
🇺🇸Miami, Florida, United States
Children's Lung Specialist
🇺🇸Las Vegas, Nevada, United States
Duke Pediatric Clinical
🇺🇸Durham, North Carolina, United States
Santiago Reyes MD-Private Prac
🇺🇸Oklahoma City, Oklahoma, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
Alamo Clinical Research Assoc
🇺🇸San Antonio, Texas, United States
University of Alabama
🇺🇸Birmingham, Alabama, United States
Childrens Hospital of LA
🇺🇸Los Angeles, California, United States
SUNY Upstate Medical Univ
🇺🇸Syracuse, New York, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
CHOC
🇺🇸Orange, California, United States
Medical College of Georgia
🇺🇸Augusta, Georgia, United States
Wayne State University
🇺🇸Detroit, Michigan, United States
Michigan State University
🇺🇸Kalamazoo, Michigan, United States
Univ of Minnesota Dept of Derm
🇺🇸Minneapolis, Minnesota, United States
Pulmonary & Critical Care Med
🇺🇸Saint Louis, Missouri, United States
Spectrum Hospital
🇺🇸Grand Rapids, Michigan, United States
University of Mississippi
🇺🇸Jackson, Mississippi, United States
St. Peters Univ Hospital
🇺🇸New Brunswick, New Jersey, United States
University of New Mexico
🇺🇸Albuquerque, New Mexico, United States
CF Solutions, Inc
🇺🇸Tulsa, Oklahoma, United States
Long Island Jew Adult CF Ctr
🇺🇸New Hyde Park, New York, United States
Monmouth Medical Center
🇺🇸Long Branch, New Jersey, United States
Albany Medical College
🇺🇸Albany, New York, United States
Central Florida Pulmonary Grou
🇺🇸Orlando, Florida, United States
Capital Allergy Resp Dis Ctr
🇺🇸Sacramento, California, United States
Univ of Michigan Hlth System
🇺🇸Ann Arbor, Michigan, United States
Medical Univ of South Carolina
🇺🇸Charleston, South Carolina, United States