Cystic FibrosisPhase 4NCT00434278
A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)
A Phase 4 study testing Dornase alfa in people with Cystic Fibrosis, run by Genentech, Inc. at 40 sites in United States.
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 Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group). Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Dornase alfa
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 40 sitesUnited States
- Study dates
- Mar 2007to Nov 2008
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Show all 11Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
40 sites in 1 country.
40 sites in 1 country
United States40 sites
Albany Medical College
Albany, New York, 12208
University of New Mexico
Albuquerque, New Mexico, 87131-0001
Univ of Michigan Hlth System
Ann Arbor, Michigan, 48109-5212
Brown Univ School of Medicine
Atlanta, Georgia, 30322
Medical College of Georgia
Augusta, Georgia, 30912
University of Alabama
Birmingham, Alabama, 35233
Medical Univ of South Carolina
Charleston, South Carolina, 29425
Rush Medical Center
Chicago, Illinois, 60612
Rainbow Babies & Child Hosp
Cleveland, Ohio, 44106
Pediatric Pulmonary Assoc
Columbia, South Carolina, 29203
Nat'l Jewish Med/Research Ctr
Denver, Colorado, 80206
Wayne State University
Detroit, Michigan, 48201
Duke Pediatric Clinical
Durham, North Carolina, 27710
Spectrum Hospital
Grand Rapids, Michigan, 49503
Baylor College of Medicine
Houston, Texas, 77030
University of Mississippi
Jackson, Mississippi, 39216-4505
Pulm & Critical Care Assoc
Jacksonville, Florida, 32204
Michigan State University
Kalamazoo, Michigan, 49008
Children's Lung Specialist
Las Vegas, Nevada, 89107
Univ of Kentucky Med Ctr
Lexington, Kentucky, 40536
Monmouth Medical Center
Long Branch, New Jersey, 07740
Childrens Hospital of LA
Los Angeles, California, 90027
USC Adult CF Center
Los Angeles, California, 90033
Univ of Miami
Miami, Florida, 33136
Univ of Minnesota Dept of Derm
Minneapolis, Minnesota, 55455
W Virginia Univ Health Sci Ctr
Morgantown, West Virginia, 26506
Morristown Memorial Hospital
Morristown, New Jersey, 07962
St. Peters Univ Hospital
New Brunswick, New Jersey, 08901
Long Island Jew Adult CF Ctr
New Hyde Park, New York, 11040
Santiago Reyes MD-Private Prac
Oklahoma City, Oklahoma, 73112
CHOC
Orange, California, 92868
Central Florida Pulmonary Grou
Orlando, Florida, 32803
University of Pittsburgh
Pittsburgh, Pennsylvania, 15260
Capital Allergy Resp Dis Ctr
Sacramento, California, 95819
Alamo Clinical Research Assoc
San Antonio, Texas, 78212
Pulmonary & Critical Care Med
St Louis, Missouri, 63110
SUNY Upstate Medical Univ
Syracuse, New York, 13210
Toledo Childrens Hospital
Toledo, Ohio, 43606
CF Solutions, Inc
Tulsa, Oklahoma, 74145
Ventura County Medical Ctr
Ventura, California, 93003
About this study
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease
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.
- Conditions
- Cystic Fibrosis
- Being tested
- Dornase alfa, placebo
- Keywords
- Lung disease, CF, Pulmozyme
- Medical subject headings
- Cystic Fibrosis, Lung Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dornase alfaDrug
Also called Pulmozyme
2.5 mg inhalation dose twice daily for 14±2 days
placeboDrug
2.5 mg inhalation dose twice daily for 14±2 days
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: placebo |
| Dornase alfa | Experimental | Not described Receives: Drug: Dornase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to Day 14
Secondary outcomes (1)
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.
Measured: From baseline to Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2007 (estimated)
- Study startMar 2007
- Primary completionNov 2008
- Study completionNov 2008
Why it stopped: This study was terminated for administrative reasons. There were no safety concerns.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Genentech, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genentech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michelle Freemer, M.D., Study director, Genentech, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00434278
- Sponsor's protocol ID
- Z3877g
- Status last verified
- May 2017
- First submitted
- 11 Feb 2007
- Last updated
- 16 May 2017
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease
- ClinicalTrials.gov ID
- NCT00434278
- Acronym / sponsor ID
- Z3877g
- Conditions
- Cystic Fibrosis
- Keywords
- Lung disease, CF, Pulmozyme
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was terminated for administrative reasons. There were no safety concerns.
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Genentech, Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2007
- Primary completion
- Nov 2008 (actual)
- Study completion
- Nov 2008 (actual)
- First posted
- 13 Feb 2007 (estimated)
- Results
- Posted 16 Nov 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: placebo |
| Dornase alfa | Experimental | Drug: Dornase alfa |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dornase alfaAlso: Pulmozyme2.5 mg inhalation dose twice daily for 14±2 days | Drug | Dornase alfa |
| placebo2.5 mg inhalation dose twice daily for 14±2 days | Drug | Placebo |
Primary outcome
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.
1 secondary outcome
Change in Pulmonary Function as Measured by FEV1 and FVC
Time frame: From baseline to Day 14
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michelle Freemer, M.D. study director
Genentech, Inc.
40 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Dornase alfa in people with Cystic Fibrosis, run by Genentech, Inc. at 40 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 16 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Nov 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Distance Walked in the 6-minute Walk Test (time frame: From baseline to Day 14). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2017.