A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)

• Status: Terminated
• Trial end date: 2008-11-01
• Target enrollment: 0
• Participant gender: All

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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genentech, Inc.

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