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| Name | Class |
|---|---|
| Genentech, Inc. | INDUSTRY |
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Chronic sinusitis is a frequent complication in cystic fibrosis. The aim of this study is to determine whether Pulmozyme(dornase alfa) would maintain sinus health (compared to placebo) in patients with cystic fibrosis who have recently undergone sinus surgery.
AIM: To evaluate the effectiveness of Pulmozyme(dornase alfa) in decreasing post-operative sinusitis symptoms in patients with cystic fibrosis (CF)
PROCEDURES: 20 patients with CF will be randomized to receive either Pulmozyme or placebo via nasal inhalation daily for 12 months. Consent will be obtained following surgery and treatment will begin 1 week post-operatively.
Monitoring will include examination and recording of adverse effects and follow up weekly for one month and then at 2.5, 6, 9 and 12 months.
Outcome measures will include ciliary function testing, pulmonary function testing, sinus questionnaires and CT scan.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pulmozyme | Experimental | 2.5 mg Pulmozyme (dornase alfa) delivered intranasally once daily |
|
| placebo | Placebo Comparator | 2.5 mg/2mL placebo administered intranasally once daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pulmozyme (dornase alfa) | Drug | 2.5 mg/2.5 mL of Pulmozyme (dornase alfa) delivered via Sinustar nasal nebulizer device |
|
| Measure | Description | Time Frame |
|---|---|---|
| Computed Tomography Evidence of Less Sinus Disease | compare sinus CT pre-op (baseline) to one year after initiation of study drug Difference in pre and post scores by Lund-McKay scoring system are reported (1 year minus baseline) The Lund-Mackay scoring system was used to evaluate the extent and severity of sinusitis. The scale ranges from 0 (best possible outcome with complete lucency of all sinuses) to 24 (worst possible outcome with complete opacification of all sinuses) | baseline and 1 year |
| Improvement in Appearance of Nasal Passages/Sinuses | periodic endoscopic photos of sinuses by ear-nose-throat (ENT) surgeon. The scale for scoring severity of disease ranges from 0 (best possible outcome) to 2 (worst possible outcome). independent blinded scoring by 2 surgeons difference in scores pre and post are reported (1 year minus baseline) | baseline and 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Chronic Sinusitis Survey Score | pre-surgery and end of trial (12 months) Reduction in scores (baseline minus 1 year) are recorded The chronic sinusitis survey consists of 6 questions, ranges from 0-24, a lower score indicates the best possible outcome. | baseline and 1 year |
| Pulmonary Function |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Thomas Lahiri, MD | University of Vermont | Principal Investigator |
| Sandra Diehl, MS | University of Vermont Medical Center | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Fletcher Allen Health Care | Burlington | Vermont | 05401 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| Result | Lahiri T, Herrington H, Diehl S, Landrigan G. The effect of intranasal dornase alfa on chronic sinusitis in patients with cystic fibrosis: a pilot study. Pediatr Pulmonol S35:354, 2012. | ||
| 33735508 | Derived | Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5. |
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upon providing informed consent, subjects were randomized and blinded to treatment or placebo groups
Patients undergoing sinus surgery meeting inclusion criteria were approached in the outpatient or hospital setting
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| ID | Title | Description |
|---|---|---|
| FG000 | Pulmozyme (Dornase Alfa) | 2.5 mg/2.5mL of Pulmozyme administered intranasally once daily |
| FG001 | Placebo | 2.5mg/2.5mL placebo administered intranasally once daily |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Pulmozyme (Dornase Alfa) | 2.5 mg/2.5 mL of intranasal Pulmozyme |
| BG001 | Placebo | 2.5 mL of placebo comparator |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Computed Tomography Evidence of Less Sinus Disease | compare sinus CT pre-op (baseline) to one year after initiation of study drug Difference in pre and post scores by Lund-McKay scoring system are reported (1 year minus baseline) The Lund-Mackay scoring system was used to evaluate the extent and severity of sinusitis. The scale ranges from 0 (best possible outcome with complete lucency of all sinuses) to 24 (worst possible outcome with complete opacification of all sinuses) | Posted | Mean | 95% Confidence Interval | units on a scale | baseline and 1 year |
|
1 year (study period)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Pulmozyme | patients receiving once daily intranasal Pulmozyme |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| hospitalization for pulmonary exacerbation | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| sinus surgery | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
recruitment for a long trial was difficult so enrollment was truncated.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Thomas Lahiri, Principal Investigator | Vermont Children's Hospital at Fletcher Allen Health Care | 802-847-8600 | thomas.lahiri@vtmednet.org |
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| ID | Term |
|---|---|
| D012852 | Sinusitis |
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D012141 | Respiratory Tract Infections |
| D007239 | Infections |
| D010254 | Paranasal Sinus Diseases |
| D009668 | Nose Diseases |
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| ID | Term |
|---|---|
| C568813 | dornase alfa |
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| Placebo | Drug | 2.5 mL of placebo delivered via Sinustar nebulizer device |
|
|
prior to surgery and end of study spirometry as measured by forced expiratory volume in 1 second (FEV1) percent predicted. The change over the course of the study (1 year minus baseline) is reported. A higher value indicates a better outcome. |
| baseline and 1 year |
| BG002 |
| Total |
Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
|
| Primary | Improvement in Appearance of Nasal Passages/Sinuses | periodic endoscopic photos of sinuses by ear-nose-throat (ENT) surgeon. The scale for scoring severity of disease ranges from 0 (best possible outcome) to 2 (worst possible outcome). independent blinded scoring by 2 surgeons difference in scores pre and post are reported (1 year minus baseline) | Posted | Mean | 95% Confidence Interval | units | baseline and 1 year |
|
|
|
|
| Secondary | Chronic Sinusitis Survey Score | pre-surgery and end of trial (12 months) Reduction in scores (baseline minus 1 year) are recorded The chronic sinusitis survey consists of 6 questions, ranges from 0-24, a lower score indicates the best possible outcome. | Posted | Mean | 95% Confidence Interval | units on a scale | baseline and 1 year |
|
|
|
|
| Secondary | Pulmonary Function | prior to surgery and end of study spirometry as measured by forced expiratory volume in 1 second (FEV1) percent predicted. The change over the course of the study (1 year minus baseline) is reported. A higher value indicates a better outcome. | Posted | Mean | Full Range | percentage of predicted FEV1 | baseline and 1 year |
|
|
|
|
| 1 |
| 8 |
| 1 |
| 8 |
| EG001 | Placebo | patients receiving once daily intranasal placebo | 2 | 8 | 0 | 8 |
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| D012140 |
| Respiratory Tract Diseases |
| D010038 | Otorhinolaryngologic Diseases |
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |