NCT00434278TerminatedPhase 4
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 4
- Status
- Terminated
- Sponsor
- Genentech, Inc.
- Enrollment
- 27
- Locations
- 40
- Primary Endpoint
- Change in Distance Walked in the 6-minute Walk Test
Study Overview
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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Eligibility Criteria
- Ages
- 14 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Placebo
Placebo Comparator
Intervention: placebo (Drug)
Dornase alfa
Experimental
Intervention: Dornase alfa (Drug)
Outcomes
Primary Outcomes
Change in Distance Walked in the 6-minute Walk Test
Time Frame: 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 Outcomes
- Change in Pulmonary Function as Measured by FEV1 and FVC(From baseline to Day 14)
Investigators
Study Sites (40)
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