Cystic FibrosisPhase 2NCT00179998
Effectiveness of Pulmozyme in Infants With Cystic Fibrosis
A Phase 2 study testing Recombinant Human DNase (Pulmozyme) in people with Cystic Fibrosis, run by Nationwide Children's Hospital at 1 site in United States.
Trial summary
This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age. Read more about this study
- Who is it for?
- Ages 1 month to 30 months
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Recombinant Human DNase (Pulmozyme)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2005to Jul 2016
- Phase
- Phase 2
- 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 1 month to 30 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age < 30 months
- Diagnosis of CF based on clinical features consistent with CF as well as 1 of the 2 following criteria: a) two sweat chlorides >60 mEq/L (by quantitative pilocarpine iontophoresis), b) genotype with 2 identifiable mutations consistent with CF.
- Informed consent by parent or legal guardian
Who cannot join
- Previous treatment with Pulmozyme
- Hospitalization or treatment with IV antibiotics with 14 days of initial study visit
- Acute intercurrent respiratory infection, defined as any of the following symptoms within the preceding 48 hours: 1) fever > 38 degrees C, 2) new onset of coryza or other upper respiratory symptoms, 3) increase in cough, wheezing, or respiratory rate
- History of adverse reaction to sedation
- Oxyhemoglobin saturation <90% on room air
- Severe upper airway obstruction as determined by site PI (severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnea)
Show all 12Show fewer
- Hemodynamically significant congenital heart disease or diagnosed arrhythmias
- History of hemoptysis
- History of previous pulmonary air leak (pneumothorax)
- Diagnosed seizure disorder necessitating current anticonvulsive therapy. A history of febrile seizures is not an exclusion criterion.
- Use of Investigational drug(s) within 60 days or 5 half-lives of enrollment in this study.
- Known allergy to Chinese Hamster Ovary-derived biological products or any component of the placebo or active drug formulations.
Trial locations and contacts
1 site in 1 country.
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
Official title: Efficacy of Pulmozyme in Infants and Young Children With Cystic Fibrosis
This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
Read the detailed description
Pulmozyme is given using a nebulizer and is now widely used in older children and adults with cystic fibrosis. In adults and older children, studies have shown that daily use of Pulmozyme improves lung function and decreases the number of lung infections requiring hospital treatment. Pulmozyme has been approved by the Food and Drug Administration for use in children over 5 years old and adults with cystic fibrosis. Pulmozyme has also been approved by the FDA for use in children with cystic fibrosis less than 5 years old based upon studies showing that it is safe in this age group and that it does get into the airway tubes as well in infants and toddlers as it does in older children and adults. Currently Pulmozyme is not widely used in children with cystic fibrosis younger than 5 years because no study has clearly shown that inhaling Pulmozyme daily improves lung function or improves clearance of mucus from the airway tubes in very young children. This study will measure whether Pulmozyme improves lung function and mucous clearance from the lungs in children with cystic fibrosis less than 3 ½ years of age.
This study will compare Pulmozyme to a placebo. During the study infants and young children with cystic fibrosis will be treated with Pulmozyme for 6 months and placebo for 6 months. The study medicines will be inhaled at home once a day from a nebulizer for a period of one year. Half of the children will be treated with Pulmozyme for the first 6 months of the study and half will receive the placebo. At the 6 month point the group receiving Pulmozyme will be changed to the placebo and the group receiving placebo will be changed to Pulmozyme. The order of the 6 month treatment periods is randomized. This study is blinded. The study doctor and his staff will not know who is receiving Pulmozyme or placebo at any time during the study.
Whether Pulmozyme works will be measured using infant lung function tests and by doing a special 3-D x-ray of the child's chest (a high resolution CT or HRCT) at the beginning of the study, at 6 months and at 12 month after starting study. The study will not change the regular clinical care.
- Conditions
- Cystic Fibrosis
- Being tested
- Recombinant Human DNase (Pulmozyme), Placebos
- Keywords
- Cystic fibrosis, Infants, Children, Pulmozyme, Pulmonary function, Computed tomography
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Human DNase (Pulmozyme)Drug
Also called Pulmozyme
2.5 mg in 3 ml diluent delivered by nebulization given daily for 6 months with 3 ml diluent placebo delivered by nebulization given daily for 6 months
PlacebosDrug
2.5 ml sterile solution (8.77 mg/ml sodium chloride, 0.15 mg/ml calcium chloride, pH 7.0 +/- 2.0) delivered daily by nebulization for 6 months, either preceding or following 6 months of Pulmozyme depending on randomization of the subject
| Study group | Role | What they receive |
|---|---|---|
| Recombinant Human DNase (Pulmozyme) then Placebo | Active comparator | once daily nebulized rhDNAse Receives: Drug: Recombinant Human DNase (Pulmozyme); Drug: Placebos |
| Placebo (Nebulized Saline) then rhDNase | Placebo comparator | once daily nebulized vehicle Receives: Drug: Recombinant Human DNase (Pulmozyme); Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Chest CT (High Resolution Computed Tomography (HRCT) Score)
Change in Total HRCT Score from initiation of intervention to 6 months
Modified Maffessanti HRCT Scoring System
Airways
- Bronchial Wall Thickening:1 = mild, 2 = moderate, 3 = severe
- Bronchiectasis:1 = mild, 2 = moderate, 3 = severe
- Axial extent of 1 or 2: 1 = central/middle, 2 = also periphery
- Regional extent of 1 or 2: x 1 if < 50 %, x 2 if > 50 %
- Gas trapping score:0 if 1 sub-segment, 1 if < 25 %, 2 if 25 - 50 %, 3 if 50 - 75 %, 4 if > 75 % Multiply (# 1 + # 2 + # 3) by # 4 then add # 5
Parenchyma
- Airspace disease: 0 = none, 1 = present
- Ground glass opacity: 0 = none, 1 = present
- Mucous Plugging: 0 = none, 1 = present
Total Score = Airway + Parenchymal Scores for RUL, LUL, RLL, and LLL Sections. The Total Score ranges from 12 to 92, with higher scores indicating greater impairment.
Maximum Score = 4 x 23 = 92
Measured: 6 months
Infant Pulmonary Function Tests (FEV0.5)
Change in FEV0.5 from initiation of intervention to 6 months
Measured: 6 months
Secondary outcomes (1)
Antibiotic Treatment Days
Total number of days treated with IV, oral or nebulized antibiotics over 6 initial month interval
Measured: per 6 month interval
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study startJan 2005
- Primary completionOct 2008
- Study completionJul 2016
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 Nationwide Children's HospitalThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Robert G Castile, MD, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00179998
- Sponsor's protocol ID
- Z2910s
- Status last verified
- Apr 2019
- First submitted
- 10 Sep 2005
- Last updated
- 9 May 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Pulmozyme in Infants and Young Children With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00179998
- Acronym / sponsor ID
- Z2910s
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic fibrosis, Infants, Children, Pulmozyme, Pulmonary function, Computed tomography
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Jan 2005
- Primary completion
- Oct 2008 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 16 Sep 2005 (estimated)
- Results
- Posted 9 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
Detailed description
Pulmozyme is given using a nebulizer and is now widely used in older children and adults with cystic fibrosis. In adults and older children, studies have shown that daily use of Pulmozyme improves lung function and decreases the number of lung infections requiring hospital treatment. Pulmozyme has been approved by the Food and Drug Administration for use in children over 5 years old and adults with cystic fibrosis. Pulmozyme has also been approved by the FDA for use in children with cystic fibrosis less than 5 years old based upon studies showing that it is safe in this age group and that it does get into the airway tubes as well in infants and toddlers as it does in older children and adults. Currently Pulmozyme is not widely used in children with cystic fibrosis younger than 5 years because no study has clearly shown that inhaling Pulmozyme daily improves lung function or improves clearance of mucus from the airway tubes in very young children. This study will measure whether Pulmozyme improves lung function and mucous clearance from the lungs in children with cystic fibrosis less than 3 ½ years of age.
This study will compare Pulmozyme to a placebo. During the study infants and young children with cystic fibrosis will be treated with Pulmozyme for 6 months and placebo for 6 months. The study medicines will be inhaled at home once a day from a nebulizer for a period of one year. Half of the children will be treated with Pulmozyme for the first 6 months of the study and half will receive the placebo. At the 6 month point the group receiving Pulmozyme will be changed to the placebo and the group receiving placebo will be changed to Pulmozyme. The order of the 6 month treatment periods is randomized. This study is blinded. The study doctor and his staff will not know who is receiving Pulmozyme or placebo at any time during the study.
Whether Pulmozyme works will be measured using infant lung function tests and by doing a special 3-D x-ray of the child's chest (a high resolution CT or HRCT) at the beginning of the study, at 6 months and at 12 month after starting study. The study will not change the regular clinical care.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Recombinant Human DNase (Pulmozyme) then Placebo | Active comparator | Drug: Recombinant Human DNase (Pulmozyme), Drug: Placebosonce daily nebulized rhDNAse |
| Placebo (Nebulized Saline) then rhDNase | Placebo comparator | Drug: Recombinant Human DNase (Pulmozyme), Drug: Placebosonce daily nebulized vehicle |
Who may qualify
- Ages
- 1 month to 30 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Age < 30 months
- Diagnosis of CF based on clinical features consistent with CF as well as 1 of the 2 following criteria: a) two sweat chlorides >60 mEq/L (by quantitative pilocarpine iontophoresis), b) genotype with 2 identifiable mutations consistent with CF.
- Informed consent by parent or legal guardian
Key exclusion criteria (12 listed)
- Previous treatment with Pulmozyme
- Hospitalization or treatment with IV antibiotics with 14 days of initial study visit
- Acute intercurrent respiratory infection, defined as any of the following symptoms within the preceding 48 hours: 1) fever > 38 degrees C, 2) new onset of coryza or other upper respiratory symptoms, 3) increase in cough, wheezing, or respiratory rate
- History of adverse reaction to sedation
- Oxyhemoglobin saturation <90% on room air
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 |
|---|---|---|
| Recombinant Human DNase (Pulmozyme)Also: Pulmozyme2.5 mg in 3 ml diluent delivered by nebulization given daily for 6 months with 3 ml diluent placebo delivered by nebulization given daily for 6 months | Drug | Placebo (Nebulized Saline) then rhDNase, Recombinant Human DNase (Pulmozyme) then Placebo |
| Placebos2.5 ml sterile solution (8.77 mg/ml sodium chloride, 0.15 mg/ml calcium chloride, pH 7.0 +/- 2.0) delivered daily by nebulization for 6 months, either preceding or following 6 months of Pulmozyme depending on randomization of the subject | Drug | Placebo (Nebulized Saline) then rhDNase, Recombinant Human DNase (Pulmozyme) then Placebo |
Primary outcome
Chest CT (High Resolution Computed Tomography (HRCT) Score)
Time frame: 6 months
Change in Total HRCT Score from initiation of intervention to 6 months Modified Maffessanti HRCT Scoring System Airways 1. Bronchial Wall Thickening:1 = mild, 2 = moderate, 3 = severe 2. Bronchiectasis:1 = mild, 2 = moderate, 3 = severe 3. Axial extent of 1 or 2: 1 = central/middle, 2 = also periphery 4. Regional extent of 1 or 2: x 1 if < 50 %, x 2 if > 50 % 5. Gas trapping score:0 if 1 sub-segment, 1 if < 25 %, 2 if 25 - 50 %, 3 if 50 - 75 %, 4 if > 75 % Multiply (# 1 + # 2 + # 3) by # 4 then add # 5 Parenchyma 1. Airspace disease: 0 = none, 1 = present 2. Ground glass opacity: 0 = none, 1 = present 3. Mucous Plugging: 0 = none, 1 = present Total Score = Airway + Parenchymal Scores for RUL, LUL, RLL, and LLL Sections. The Total Score ranges from 12 to 92, with higher scores indicating greater impairment. Maximum Score = 4 x 23 = 92
Infant Pulmonary Function Tests (FEV0.5)
Time frame: 6 months
Change in FEV0.5 from initiation of intervention to 6 months
1 secondary outcome
Antibiotic Treatment Days
Time frame: per 6 month interval
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Robert G Castile, MD principal investigator
Nationwide Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Recombinant Human DNase (Pulmozyme) in people with Cystic Fibrosis, run by Nationwide Children's Hospital at 1 site 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 3 inclusion and 12 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: finished, as of the sponsor's last update on 9 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 9 May 2019). 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?
Chest CT (High Resolution Computed Tomography (HRCT) Score) (time frame: 6 months). The study lists 2 primary outcomes in all. 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 9 May 2019.