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

Cystic Fibrosis Clinical Trial

Official title:

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00434278
• Other study ID #: Z3877g
• Status: Terminated
• Phase: Phase 4
• First received: February 11, 2007
• Last updated: May 12, 2017
• Start date: March 2007
• Est. completion date: November 2008

Study information

• Verified date: May 2017
• Source: Genentech, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 27
• Est. completion date: November 2008
• Est. primary completion date: November 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis
• Lung Diseases

Intervention

Drug:
• Dornase alfa
2.5 mg inhalation dose twice daily for 14±2 days
• placebo
2.5 mg inhalation dose twice daily for 14±2 days

Locations

Country	Name	City	State
United States	Albany Medical College	Albany	New York
United States	University of New Mexico	Albuquerque	New Mexico
United States	Univ of Michigan Hlth System	Ann Arbor	Michigan
United States	Brown Univ School of Medicine	Atlanta	Georgia
United States	Medical College of Georgia	Augusta	Georgia
United States	University of Alabama	Birmingham	Alabama
United States	Medical Univ of South Carolina	Charleston	South Carolina
United States	Rush Medical Center	Chicago	Illinois
United States	Rainbow Babies & Child Hosp	Cleveland	Ohio
United States	Pediatric Pulmonary Assoc	Columbia	South Carolina
United States	Nat'l Jewish Med/Research Ctr	Denver	Colorado
United States	Wayne State University	Detroit	Michigan
United States	Duke Pediatric Clinical	Durham	North Carolina
United States	Spectrum Hospital	Grand Rapids	Michigan
United States	Baylor College of Medicine	Houston	Texas
United States	University of Mississippi	Jackson	Mississippi
United States	Pulm & Critical Care Assoc	Jacksonville	Florida
United States	Michigan State University	Kalamazoo	Michigan
United States	Children's Lung Specialist	Las Vegas	Nevada
United States	Univ of Kentucky Med Ctr	Lexington	Kentucky
United States	Monmouth Medical Center	Long Branch	New Jersey
United States	Childrens Hospital of LA	Los Angeles	California
United States	USC Adult CF Center	Los Angeles	California
United States	Univ of Miami	Miami	Florida
United States	Univ of Minnesota Dept of Derm	Minneapolis	Minnesota
United States	W Virginia Univ Health Sci Ctr	Morgantown	West Virginia
United States	Morristown Memorial Hospital	Morristown	New Jersey
United States	St. Peters Univ Hospital	New Brunswick	New Jersey
United States	Long Island Jew Adult CF Ctr	New Hyde Park	New York
United States	Santiago Reyes MD-Private Prac	Oklahoma City	Oklahoma
United States	CHOC	Orange	California
United States	Central Florida Pulmonary Grou	Orlando	Florida
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Capital Allergy Resp Dis Ctr	Sacramento	California
United States	Pulmonary & Critical Care Med	Saint Louis	Missouri
United States	Alamo Clinical Research Assoc	San Antonio	Texas
United States	SUNY Upstate Medical Univ	Syracuse	New York
United States	Toledo Childrens Hospital	Toledo	Ohio
United States	CF Solutions, Inc	Tulsa	Oklahoma
United States	Ventura County Medical Ctr	Ventura	California

Sponsors (1)

Lead Sponsor	Collaborator
Genentech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in Pulmonary Function as Measured by FEV1 and FVC	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.	From baseline to Day 14