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

NCT ID: NCT00434278

Last Updated: 2017-05-16

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE4

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-03-31

Study Completion Date

2008-11-30

Brief Summary

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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group).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

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Placebo

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type DRUG

2.5 mg inhalation dose twice daily for 14±2 days

Dornase alfa

Group Type EXPERIMENTAL

Dornase alfa

Intervention Type DRUG

2.5 mg inhalation dose twice daily for 14±2 days

Interventions

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Dornase alfa

2.5 mg inhalation dose twice daily for 14±2 days

Intervention Type DRUG

placebo

2.5 mg inhalation dose twice daily for 14±2 days

Intervention Type DRUG

Other Intervention Names

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Pulmozyme

Eligibility Criteria

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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
Minimum Eligible Age

14 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genentech, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Michelle Freemer, M.D.

Role: STUDY_DIRECTOR

Genentech, Inc.

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

Childrens Hospital of LA

Los Angeles, California, United States

Site Status

USC Adult CF Center

Los Angeles, California, United States

Site Status

CHOC

Orange, California, United States

Site Status

Capital Allergy Resp Dis Ctr

Sacramento, California, United States

Site Status

Ventura County Medical Ctr

Ventura, California, United States

Site Status

Nat'l Jewish Med/Research Ctr

Denver, Colorado, United States

Site Status

Pulm & Critical Care Assoc

Jacksonville, Florida, United States

Site Status

Univ of Miami

Miami, Florida, United States

Site Status

Central Florida Pulmonary Grou

Orlando, Florida, United States

Site Status

Brown Univ School of Medicine

Atlanta, Georgia, United States

Site Status

Medical College of Georgia

Augusta, Georgia, United States

Site Status

Rush Medical Center

Chicago, Illinois, United States

Site Status

Univ of Kentucky Med Ctr

Lexington, Kentucky, United States

Site Status

Univ of Michigan Hlth System

Ann Arbor, Michigan, United States

Site Status

Wayne State University

Detroit, Michigan, United States

Site Status

Spectrum Hospital

Grand Rapids, Michigan, United States

Site Status

Michigan State University

Kalamazoo, Michigan, United States

Site Status

Univ of Minnesota Dept of Derm

Minneapolis, Minnesota, United States

Site Status

University of Mississippi

Jackson, Mississippi, United States

Site Status

Pulmonary & Critical Care Med

St Louis, Missouri, United States

Site Status

Children's Lung Specialist

Las Vegas, Nevada, United States

Site Status

Monmouth Medical Center

Long Branch, New Jersey, United States

Site Status

Morristown Memorial Hospital

Morristown, New Jersey, United States

Site Status

St. Peters Univ Hospital

New Brunswick, New Jersey, United States

Site Status

University of New Mexico

Albuquerque, New Mexico, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

Long Island Jew Adult CF Ctr

New Hyde Park, New York, United States

Site Status

SUNY Upstate Medical Univ

Syracuse, New York, United States

Site Status

Duke Pediatric Clinical

Durham, North Carolina, United States

Site Status

Rainbow Babies & Child Hosp

Cleveland, Ohio, United States

Site Status

Toledo Childrens Hospital

Toledo, Ohio, United States

Site Status

Santiago Reyes MD-Private Prac

Oklahoma City, Oklahoma, United States

Site Status

CF Solutions, Inc

Tulsa, Oklahoma, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Medical Univ of South Carolina

Charleston, South Carolina, United States

Site Status

Pediatric Pulmonary Assoc

Columbia, South Carolina, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Alamo Clinical Research Assoc

San Antonio, Texas, United States

Site Status

W Virginia Univ Health Sci Ctr

Morgantown, West Virginia, United States

Site Status

Countries

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United States

Other Identifiers

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Z3877g

Identifier Type: -

Identifier Source: org_study_id

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