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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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.
Related Research Topics
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Trial Locations
- Locations (40)
University of Alabama
🇺🇸Birmingham, Alabama, United States
Childrens Hospital of LA
🇺🇸Los Angeles, California, United States
USC Adult CF Center
🇺🇸Los Angeles, California, United States
CHOC
🇺🇸Orange, California, United States
Capital Allergy Resp Dis Ctr
🇺🇸Sacramento, California, United States
Ventura County Medical Ctr
🇺🇸Ventura, California, United States
Nat'l Jewish Med/Research Ctr
🇺🇸Denver, Colorado, United States
Pulm & Critical Care Assoc
🇺🇸Jacksonville, Florida, United States
Univ of Miami
🇺🇸Miami, Florida, United States
Central Florida Pulmonary Grou
🇺🇸Orlando, Florida, United States
Scroll for more (30 remaining)University of Alabama🇺🇸Birmingham, Alabama, United States