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Most patients were unable to perform the test being used to measure lung function, thus it became clear the study would not yield clinically meaningful data
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This was a Phase IV, multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the effect of Pulmozyme on pulmonary function, health-related quality of life (HRQOL), and respiratory symptoms in 3- to 5-year-old children with cystic fibrosis (CF). Approximately 40 patients were planned to be enrolled in this study. However, only 3 patients were eligible for random allocation and received treatment: 1 patient in the Pulmozyme group and 2 patients in the placebo group. All 3 patients completed the study assessments but did not have usable pulmonary function test (PFT) data.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dornase alfa | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dornase alfa | Drug | 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment). | The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung. | from Visit 2 to Visit 3 (16 +/- 2 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10) | The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Michelle Freemer, M.D. | Genentech, Inc. | Study Director |
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| ID | Title | Description |
|---|---|---|
| FG000 | Dornase Alfa | 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days |
| FG001 | Placebo | 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Dornase Alfa | 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days |
| BG001 | Placebo | 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment). | The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung. | Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT. | Posted | from Visit 2 to Visit 3 (16 +/- 2 days) |
|
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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 | Dornase Alfa | 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Increase in cough | Respiratory, thoracic and mediastinal disorders |
Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Communications | Genentech, Inc. | 800-821-8590 |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C568813 | dornase alfa |
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| Placebo | Drug | 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days |
|
| from Visit 2 to Visit 3 (16 +/- 2 days) |
| Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10) | The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Resistance is complex measure that incorporates the lack of changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (10Hz was used for the secondary endpoint). | from Visit 2 to Visit 3 (16 +/- 2 days) |
| Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers | The CFQ-R for Preschoolers and the CFQ-R for Parents of Preschoolers was designed specifically to measure the impact of CF for patients with a diagnosis of CF. Each question is answered using a 4-point Likert scale. In order to calculate the domain/symptom scale scores, the following algorithm is followed
| from Visit 2 to Visit 3 (16 +/- 2 days) |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Gender | Count of Participants | Participants |
|
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
| OG001 | Placebo | 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days |
|
| Secondary | Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10) | The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung. | Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT. | Posted | from Visit 2 to Visit 3 (16 +/- 2 days) |
|
|
| Secondary | Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10) | The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Resistance is complex measure that incorporates the lack of changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (10Hz was used for the secondary endpoint). | Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT. | Posted | from Visit 2 to Visit 3 (16 +/- 2 days) |
|
|
| Secondary | Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers | The CFQ-R for Preschoolers and the CFQ-R for Parents of Preschoolers was designed specifically to measure the impact of CF for patients with a diagnosis of CF. Each question is answered using a 4-point Likert scale. In order to calculate the domain/symptom scale scores, the following algorithm is followed
| Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT. | Posted | from Visit 2 to Visit 3 (16 +/- 2 days) |
|
|
| 0 |
| 1 |
| 1 |
| 1 |
| EG001 | Placebo | 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days | 0 | 2 | 2 | 2 |
| Bad dreams | Psychiatric disorders |
|
| Flu symptoms | General disorders |
|
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |