Study Stopped
This study was terminated for administrative reasons. There were no safety concerns.
A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease
1 other identifier
interventional
27
1 country
40
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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started Mar 2007
40 active sites
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
February 11, 2007
CompletedFirst Posted
Study publicly available on registry
February 13, 2007
CompletedStudy Start
First participant enrolled
March 1, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2008
CompletedResults Posted
Study results publicly available
November 16, 2010
CompletedMay 16, 2017
May 1, 2017
1.7 years
February 11, 2007
October 18, 2010
May 12, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change in Distance Walked in the 6-minute Walk Test
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.
From baseline to Day 14
Secondary Outcomes (1)
Change in Pulmonary Function as Measured by FEV1 and FVC
From baseline to Day 14
Study Arms (2)
Placebo
PLACEBO COMPARATORDornase alfa
EXPERIMENTALInterventions
2.5 mg inhalation dose twice daily for 14±2 days
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Genentech, Inc.lead
Study Sites (40)
University of Alabama
Birmingham, Alabama, 35233, United States
Childrens Hospital of LA
Los Angeles, California, 90027, United States
USC Adult CF Center
Los Angeles, California, 90033, United States
CHOC
Orange, California, 92868, United States
Capital Allergy Resp Dis Ctr
Sacramento, California, 95819, United States
Ventura County Medical Ctr
Ventura, California, 93003, United States
Nat'l Jewish Med/Research Ctr
Denver, Colorado, 80206, United States
Pulm & Critical Care Assoc
Jacksonville, Florida, 32204, United States
Univ of Miami
Miami, Florida, 33136, United States
Central Florida Pulmonary Grou
Orlando, Florida, 32803, United States
Brown Univ School of Medicine
Atlanta, Georgia, 30322, United States
Medical College of Georgia
Augusta, Georgia, 30912, United States
Rush Medical Center
Chicago, Illinois, 60612, United States
Univ of Kentucky Med Ctr
Lexington, Kentucky, 40536, United States
Univ of Michigan Hlth System
Ann Arbor, Michigan, 48109-5212, United States
Wayne State University
Detroit, Michigan, 48201, United States
Spectrum Hospital
Grand Rapids, Michigan, 49503, United States
Michigan State University
Kalamazoo, Michigan, 49008, United States
Univ of Minnesota Dept of Derm
Minneapolis, Minnesota, 55455, United States
University of Mississippi
Jackson, Mississippi, 39216-4505, United States
Pulmonary & Critical Care Med
St Louis, Missouri, 63110, United States
Children's Lung Specialist
Las Vegas, Nevada, 89107, United States
Monmouth Medical Center
Long Branch, New Jersey, 07740, United States
Morristown Memorial Hospital
Morristown, New Jersey, 07962, United States
St. Peters Univ Hospital
New Brunswick, New Jersey, 08901, United States
University of New Mexico
Albuquerque, New Mexico, 87131-0001, United States
Albany Medical College
Albany, New York, 12208, United States
Long Island Jew Adult CF Ctr
New Hyde Park, New York, 11040, United States
SUNY Upstate Medical Univ
Syracuse, New York, 13210, United States
Duke Pediatric Clinical
Durham, North Carolina, 27710, United States
Rainbow Babies & Child Hosp
Cleveland, Ohio, 44106, United States
Toledo Childrens Hospital
Toledo, Ohio, 43606, United States
Santiago Reyes MD-Private Prac
Oklahoma City, Oklahoma, 73112, United States
CF Solutions, Inc
Tulsa, Oklahoma, 74145, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, 15260, United States
Medical Univ of South Carolina
Charleston, South Carolina, 29425, United States
Pediatric Pulmonary Assoc
Columbia, South Carolina, 29203, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Alamo Clinical Research Assoc
San Antonio, Texas, 78212, United States
W Virginia Univ Health Sci Ctr
Morgantown, West Virginia, 26506, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
Because the study was terminated (for administrative reasons and infeasibility of enrollment), no conclusions can be made about the effect of Pulmozyme on exercise tolerance in this population.
Results Point of Contact
- Title
- Medical Communications
- Organization
- Genentech, Inc.
Study Officials
- STUDY DIRECTOR
Michelle Freemer, M.D.
Genentech, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 11, 2007
First Posted
February 13, 2007
Study Start
March 1, 2007
Primary Completion
November 1, 2008
Study Completion
November 1, 2008
Last Updated
May 16, 2017
Results First Posted
November 16, 2010
Record last verified: 2017-05