Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The investigators know that adherence to medications in children with cystic fibrosis (CF) is poor. Forgetfulness has often been reported as a barrier to adherence by both CF patients and their parents. Many of the investigators patients also report being motivated by the results of their lung function studies (PFTs) to stay adherent to their medications. In this study, the investigators would like to see if providing medication reminders and allowing patients to measure their lung function at home will lead to better adherence. This will be a pilot study to determine the feasibility of providing such as a device to children with CF.
This is a randomized controlled trial to assess the impact of home lung function monitoring and medication reminders on adherence, clinical outcomes and quality of life in children with cystic fibrosis (CF) who are between 10 and 21 years of age.
Participants will be randomly assigned to either an intervention group or a control group. Those in the intervention group will receive a personal spirometer device that provides medication reminders and allows for lung function monitoring at home. The control group will receive the current standard of care. All participants will be aware that their adherence is being monitored over the course of the study using prescription refill data. Changes in lung function, body mass index and rate of hospitalization will be used as measures of clinical outcome. Responses to age-appropriate well-validated patient questionnaires will be used to assess the impact of the investigators interventions on quality of life and perceptions of treatment burden.
Since this device has never been previously studied in this patient population, the investigators would like to do a pilot study with 5 patients (3 in the intervention group and 2 in the control group) over a 3-month period to determine feasibility. This will also help us with the investigator power calculations and ultimately deciding the number of participants that will be needed for the larger study.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intervention Group | Active Comparator | Participants will receive a Spiro PD personal spirometer that will allow them to measure their lung function at home and provide medication reminders. Participants will be instructed to use the device to check their lung function once a week. They will also be asked to use the medication reminder feature of their device daily. Participants in this group will receive a telephone call once a week from the research team to review lung function results and answer questions. All participants need to fill out a quality of life questionnaire at the time of enrollment and at the end of the study. Participants will be asked to sign a release form so their pharmacies can be contacted for prescription refill data to monitor adherence over the course of the study. All participants will be asked to come to their quarterly clinic visits with their pediatric pulmonologist where their height, weight, body mass index, lung function and frequency of hospitalizations will be assessed. |
|
| Control | No Intervention | Participants will be asked to fill out a quality of life questionnaire at the time of enrollment and at the end of the study. All participants will be asked to come to their quarterly clinic visits with their pediatric pulmonologist where their height, weight, body mass index, lung function and frequency of hospitalizations will be assessed. All participants will be asked to sign a release form so their pharmacies can be contacted for prescription refill data to monitor adherence over the course of the study. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Spiro PD personal spirometer | Device |
|
| Measure | Description | Time Frame |
|---|---|---|
| Medication Adherence | Overall adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins based on prescription refill data. The actual number of prescriptions of each of the three medications filled in the 3-month period was divided by the number that should have been filled based on the prescribed amount of each medication and that value was multiplied by a 100 to generate a percentage. | 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Cystic Fibrosis Questionnaire-Revised (CFQ-R)Treatment Burden Domain Score (Child) | Response of the participants to the treatment burden domain of the CFQ-R at the end of 3 months | 3 months |
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Parent) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Aarti Shakkottai, MD | University of Michigan | Principal Investigator |
| Samya Nasr, MD | University of Michigan | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Intervention Group | Subjects in this group were asked to use the Spiro PD personal spirometer to check their lung function once a week for 3 months. They were also asked to use the medication reminder feature of their device daily. Participants were trained on the appropriate use of their device at the time of enrollment. They also received weekly telephone calls from a respiratory therapist to review that week's lung function results. They filled out a quality of life questionnaire at enrollment and at 3 months. Information regarding their height, weight, lung function and frequency of hospitalizations over the previous 3 months were obtained at enrollment and 3-months from review of their medical records. Pharmacies were contacted for refill data during the 3-month study duration for inhaled hypertonic saline, dornase alfa and CF multivitamins. |
| FG001 | Control | Subjects in this group had their adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins during the 3-month study duration monitored through the use of prescription refill histories. They filled out a quality of life questionnaire at enrollment and 3 months. Their height, weight, lung function and frequency of hospitalizations over the previous 3 months was obtained at enrollment and 3-months from review of their medical records. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Intervention Group | Subjects in this group were asked to use the Spiro PD personal spirometer to check their lung function once a week for 3 months. They were also asked to use the medication reminder feature of their device daily. Participants were trained on the appropriate use of their device at the time of enrollment. They also received weekly telephone calls from a respiratory therapist to review that week's lung function results. They filled out a quality of life questionnaire at enrollment and at 3 months. Information regarding their height, weight, lung function and frequency of hospitalizations over the previous 3 months were obtained at enrollment and 3-months from review of their medical records. Pharmacies were contacted for refill data during the 3-month study duration for inhaled hypertonic saline, dornase alfa and CF multivitamins. |
| 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 | Medication Adherence | Overall adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins based on prescription refill data. The actual number of prescriptions of each of the three medications filled in the 3-month period was divided by the number that should have been filled based on the prescribed amount of each medication and that value was multiplied by a 100 to generate a percentage. | Posted | Mean | Standard Deviation | Percent Adherence | 3 months |
|
3 months
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Intervention Group | Subjects in this group were asked to use the Spiro PD personal spirometer to check their lung function once a week for 3 months. They were also asked to use the medication reminder feature of their device daily. Participants were trained on the appropriate use of their device at the time of enrollment. They also received weekly telephone calls from a respiratory therapist to review that week's lung function results. They filled out a quality of life questionnaire at enrollment and at 3 months. Information regarding their height, weight, lung function and frequency of hospitalizations over the previous 3 months were obtained at enrollment and 3-months from review of their medical records. Pharmacies were contacted for refill data during the 3-month study duration for inhaled hypertonic saline, dornase alfa and CF multivitamins. |
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Aarti Shakkottai | University of Michigan | 734-764-4123 | aartik@med.umich.edu |
Not provided
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Response of the parents/caregivers to the treatment burden domain of the CFQ-R at the end of 3-months |
| 3 months |
| BG001 | Control | Subjects in this group had their adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins during the 3-month study duration monitored through the use of prescription refill histories. They filled out a quality of life questionnaire at enrollment and 3 months. Their height, weight, lung function and frequency of hospitalizations over the previous 3 months was obtained at enrollment and 3-months from review of their medical records. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Forced expiratory volume in 1 second (FEV1) | Mean | Standard Deviation | %predicted |
|
| Body Mass Index (BMI) Percentile | Mean | Standard Deviation | Percentile |
|
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Child) | This is a well-validated measure of health-related quality of life among children with CF. It assesses functionality in several disease-specific domains including treatment burden. A total score is calculated for each domain, which varies based on the number of questions in that domain. This is then converted to a scaled score between 0-100 for better comparison between the domains. Higher scores reflect better quality of life. | Mean | Standard Deviation | units on a scale |
|
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain (Parent) | This is a well-validated measure of health-related quality of life among children with CF. It assesses functionality in several disease-specific domains including treatment burden. A total score is calculated for each domain, which varies based on the number of questions in that domain. This is then converted to a scaled score between 0-100 for better comparison between the domains. Higher scores reflect better quality of life. | Mean | Standard Deviation | units on a scale |
|
| OG001 | Control | Subjects in this group had their adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins during the 3-month study duration monitored through the use of prescription refill histories. They filled out a quality of life questionnaire at enrollment and 3 months. Their height, weight, lung function and frequency of hospitalizations over the previous 3 months was obtained at enrollment and 3-months from review of their medical records. |
|
|
| Secondary | Cystic Fibrosis Questionnaire-Revised (CFQ-R)Treatment Burden Domain Score (Child) | Response of the participants to the treatment burden domain of the CFQ-R at the end of 3 months | Posted | Mean | Standard Deviation | units on a scale | 3 months |
|
|
|
| Secondary | Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Parent) | Response of the parents/caregivers to the treatment burden domain of the CFQ-R at the end of 3-months | Posted | Mean | Standard Deviation | units on a scale | 3 months |
|
|
|
| 0 |
| 3 |
| 0 |
| 3 |
| EG001 | Control | Subjects in this group had their adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins during the 3-month study duration monitored through the use of prescription refill histories. They filled out a quality of life questionnaire at enrollment and 3 months. Their height, weight, lung function and frequency of hospitalizations over the previous 3 months was obtained at enrollment and 3-months from review of their medical records. | 0 | 2 | 0 | 2 |
Not provided
Not provided
Not provided
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |