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

This study has been terminated.
(This study was terminated for administrative reasons. There were no safety concerns.)
Sponsor:
Information provided by:
Genentech
ClinicalTrials.gov Identifier:
NCT00434278
First received: February 11, 2007
Last updated: October 18, 2010
Last verified: October 2010
  Purpose

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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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


Condition Intervention Phase
Cystic Fibrosis
Drug: Dornase alfa
Drug: placebo
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease

Resource links provided by NLM:


Further study details as provided by Genentech:

Primary Outcome Measures:
  • Change in Distance Walked in the 6-minute Walk Test [ Time Frame: From baseline to Day 14 ] [ Designated as safety issue: No ]
    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:
  • Change in Pulmonary Function as Measured by FEV1 and FVC [ Time Frame: From baseline to Day 14 ] [ Designated as safety issue: No ]
    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.


Enrollment: 27
Study Start Date: March 2007
Study Completion Date: November 2008
Primary Completion Date: November 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo Drug: placebo
2.5 mg inhalation dose twice daily for 14±2 days
Experimental: Dornase alfa Drug: Dornase alfa
2.5 mg inhalation dose twice daily for 14±2 days
Other Name: Pulmozyme

  Eligibility

Ages Eligible for Study:   14 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00434278

  Show 40 Study Locations
Sponsors and Collaborators
Genentech
Investigators
Study Director: Michelle Freemer, M.D. Genentech
  More Information

No publications provided

Responsible Party: Disclosures Group, Genentech, Inc.
ClinicalTrials.gov Identifier: NCT00434278     History of Changes
Other Study ID Numbers: Z3877g
Study First Received: February 11, 2007
Results First Received: October 18, 2010
Last Updated: October 18, 2010
Health Authority: United States: Food and Drug Administration

Keywords provided by Genentech:
Lung disease
CF
Pulmozyme

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Lung Diseases
Pancreatic Diseases
Digestive System Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on September 16, 2014