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| ID | Type | Description | Link |
|---|---|---|---|
| 18-C-0093 |
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Background:
Neurofibromatosis type 1 (NF1) is a genetic disorder. It has a broad variety of effects on the body. Up to half of people with NF1 get plexiform neurofibromas (PNs). These are benign tumors. But they can have serious effects like pain and disfigurement. To treat PNs, a person may have to take medicine every day for a long period of time. Researchers think that it will be important for people to take the medicine regularly for it to work. They want to study how well people with NF1 follow their treatment plan for PNs.
Objective:
To study how often people with neurofibromatosis type 1 take medicine that has been prescribed to them for treating plexiform neurofibromas.
Eligibility:
People ages 3-59 already enrolled in an NF1 clinical trial
Design:
Participants will need access to the internet to do the study activities.
Parents or caregivers will do some study activities for child participants.
Participants will complete 5 questionnaires. They will take about 20 minutes total. The topics will be:
Demographic data
Recent life events
How much pain interferes with daily life
Ability to focus and pay attention to tasks
Emotional distress or depression
Participants will mark down every time they take a dose of the medicine in their clinical trial. They will use a form the researchers give them. The pill bottles they get in their trial will have a chip in the cap that will record when it is opened. Participants will keep a daily diary of their medicine. Their pills will be counted at clinical trial visits.
Participants may have more short questionnaires. They may have interviews by phone or video.
Background:
Objective:
- To establish the feasibility of using MEMS^TM to monitor medication adherence in the NF1 population
Eligibility:
Design:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adult Participants | Experimental | Questionnaires and use of the medication event monitoring system (MEMS^TM) |
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| Pediatric Participants 8+ Years | Experimental | Questionnaires and use of the medication event monitoring system (MEMS^TM) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Medication Event Monitoring System (MEMS^TM) | Behavioral | A computerized method of tracking the dates and times of a pill bottle being opened. |
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| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Enrolled Participants for Which we Are Able to Collect Data From the Medication Event Monitoring Systems (MEMS^TM) System for Two or More Cycles of Treatment (Target = 75%) | The proportion of enrolled participants are reported for individuals who had two full cycles of data recorded by the medication event monitoring systems (MEMS^TM) system. To monitor medication adherence the medication event monitoring system (MEMSTM) used to track the dates and times a pill bottle was opened. At least 2 cycles of medication adherence are considered a success. | Two cycles, approximately 56 days |
| Median Number of Cycles Monitored for Participants From the Medication Event Monitoring System (MEMS^TM) | To monitor medication adherence the medication event monitoring system (MEMSTM) was used to track the dates and times a pill bottle was opened. We calculated the median number of cycles monitored for all patients. | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Measure | Description | Time Frame |
|---|---|---|
| Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data | To monitor medication adherence the medication event monitoring system (MEMS^TM) was used to track the dates and times a pill bottle was opened. The number of times the bottle was opened was divided by the total number of times the bottle should be opened according to provider instructions. The mean of these numbers was computed across groups of cycles 1-4, 5-8, 9-12 and 13-18. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. Higher number is better adherence. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Serious and/or Non-serious Adverse Events | Here is the number of participants with serious and/or non-serious adverse events. A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. |
Inclusion Criteria for Patient
Inclusion Criteria for Parents or Caregivers
EXCLUSION CRITERIA:
Exclusion Criteria for Patient
Exclusion Criteria for Parent or Caregiver
None
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| Name | Affiliation | Role |
|---|---|---|
| Staci M Peron, Ph.D. | National Cancer Institute (NCI) | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Institutes of Health Clinical Center | Bethesda | Maryland | 20892 | United States |
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| Label | URL |
|---|---|
| NIH Clinical Center Detailed Web Page | View source |
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All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request.
Clinical data available during the study and indefinitely.
Clinical data will be made available via subscription to Biomedical Translational Research Information System (BTRIS) and with the permission of the study principal investigator (PI).
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No pediatric participants were enrolled on this trial.
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| ID | Title | Description |
|---|---|---|
| FG000 | Adult Participants (18+ Years) | Questionnaires and use of the medication event monitoring system (MEMS^TM) Participants must take an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s). Medication Event Monitoring System (MEMS^TM) caps will be used to monitor adherence over time along with patient diaries and pill counts. Medication Event Monitoring System (MEMS^TM): A computerized method of tracking the dates and times of a pill bottle being opened. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 16, 2020 |
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| Questionnaires | Other | Adult and pediatric participants completed a series of questionnaires to assess medication adherence, demographics, life events, and barriers to adherence. |
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| Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count | A repeated-measures ANOVA was used to compare three adherence assessment methods over time. The medication event monitoring system (MEMS^TM) was used to track the dates and times a pill bottle was opened. The number of times the bottle was opened was divided by the total number of times the bottle should be opened according to provider instructions. Pill count was used to assess how many pills were returned at each restaging visit compared to the number that should be returned if all medication was taken as prescribed. The daily diary was completed by participants, documenting date and timing of doses. The mean of these numbers was computed across groups of cycles (1 cycle = 28 days) 1-4, 5-8, 9-12 and 13-18 for the MEMS^TM, Pill Count, and Daily Diary. | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), an range of approximately 112 days to 504 days |
| Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap and age, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4, cycles 5-8, cycles 9-12, and cycles 13-18. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap for years of education, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. Number of years of education was collected through participant self-report and was a continuous variable. | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Quality of Life (QOL) Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference T-scores | Outcome measures correlations between 2 variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap & PROMIS Pain Interference T- Scores (Same for all PROMIS measures) Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-13hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle & dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. PROMIS Pain Interference raw scores transformed to T-scores for analysis (mean:50,standard deviation: 10, higher scores is more pain interference, T of 55 is cutoff-mild pain interference). | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Quality of Life (QOL) Patient-Reported Outcomes Measurement Information System (PROMIS) Depression T-scores | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap and PROMIS Depression Scores, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. PROMIS Depression raw scores were transformed to T-scores for analysis (mean: 50, standard deviation: 10, higher scores is more depression symptoms, T of 55 is the cut off for mild depressive symptoms). | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Quality of Life (QOL) Patient-Reported Outcomes Measurement Information System (PROMIS) Cognitive Interference T-scores | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap and PROMIS Cognitive Interference Scores, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. PROMIS Cognitive Interference raw scores were transformed to T-scores for analysis (mean: 50, standard deviation: 10, higher scores is more cognitive interference, T of 60 represents mild interference). | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Barriers to Adherence Questionnaire | Participants completed a barriers questionnaire to assess barriers most frequently endorsed on the questionnaire. The questionnaire focused on any medication adherence issues during the trial (e.g., missed doses). Participants were given 16 possible reasons that they may have missed a dose and were asked to check all items that caused them to miss a dose of medication. Participants were also able to provide additional reasons for missing medication that were not on the form. | Baseline and follow-up (between cycles 8 and 12; or after cycle 18/when taken off of study (range of 224-504 days) |
| Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Barriers to Adherence Questionnaire | The outcome measures Spearman correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. MEMS^TM was defined as the average mean adherence, an adherent dose was taken 11-13 hours from the prior correct dose. The mean was computed by dividing medication adherence across the cycle by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data. Barriers to adherence was calculated by summing the number of barriers for each participant at follow-up. Participants were given 16 possible reasons that they may have missed a dose and were asked to check all items that apply to them. | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Medication Event Monitoring Systems (MEMS^TM) Cap and Stressful Life Events: Number of Stressful Life Events Endorsed in the Life Events Checklist | To assess the relationship between the adherence method MEMS^TM cap and stressful life events (i.e., the number of stressful life events endorsed in the Life Events Checklist), Spearman correlation was used. Participants complete a 22-item questionnaire to report stressful events that impact medication adherence. MEMS^TM was defined as the average mean adherence, an adherent dose was taken 11-13 hours from the prior correct dose. The mean was computed by dividing medication adherence across the cycle by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data. | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Medication Event Monitoring Systems (MEMS^TM) Cap and Rating of Overall Stress | To assess the relationship between the adherence method MEMS^TM cap and rating of overall stress, Spearman correlation was used. Participants complete a 1-item question asking them to rate their stress from 0 - 10, with higher numbers being more stressed. MEMS^TM was defined as the average mean adherence, an adherent dose was taken 11-13 hours from the prior correct dose. The mean was computed by dividing medication adherence across the cycle by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data. | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
| Baseline to cycle 18 (end of study), approximately 504 days (1.38 years) |
| FG001 | Pediatric Participants (3-17 Years) | Questionnaires and use of the medication event monitoring system (MEMS^TM) Participants must take an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s). Medication Event Monitoring System (MEMS^TM) caps will be used to monitor adherence over time along with patient diaries and pill counts. Medication Event Monitoring System (MEMS^TM): A computerized method of tracking the dates and times of a pill bottle being opened. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Adult Participants (18+ Years) | Questionnaires and use of the medication event monitoring system (MEMS^TM) Participants must take an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s). Medication Event Monitoring System (MEMS^TM) caps will be used to monitor adherence over time along with patient diaries and pill counts. Medication Event Monitoring System (MEMS^TM): A computerized method of tracking the dates and times of a pill bottle being opened. |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| 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 | Proportion of Enrolled Participants for Which we Are Able to Collect Data From the Medication Event Monitoring Systems (MEMS^TM) System for Two or More Cycles of Treatment (Target = 75%) | The proportion of enrolled participants are reported for individuals who had two full cycles of data recorded by the medication event monitoring systems (MEMS^TM) system. To monitor medication adherence the medication event monitoring system (MEMSTM) used to track the dates and times a pill bottle was opened. At least 2 cycles of medication adherence are considered a success. | 11/12 participants were analyzed because 1 participant's cap malfunctioned, so no data was collected. | Posted | Number | Proportion of participants | Two cycles, approximately 56 days |
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| Primary | Median Number of Cycles Monitored for Participants From the Medication Event Monitoring System (MEMS^TM) | To monitor medication adherence the medication event monitoring system (MEMSTM) was used to track the dates and times a pill bottle was opened. We calculated the median number of cycles monitored for all patients. | 11/12 participants were analyzed because 1 participant's cap malfunctioned, so no data was collected. | Posted | Median | Full Range | Cycles | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data | To monitor medication adherence the medication event monitoring system (MEMS^TM) was used to track the dates and times a pill bottle was opened. The number of times the bottle was opened was divided by the total number of times the bottle should be opened according to provider instructions. The mean of these numbers was computed across groups of cycles 1-4, 5-8, 9-12 and 13-18. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. Higher number is better adherence. | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Mean | Standard Deviation | percent | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count | A repeated-measures ANOVA was used to compare three adherence assessment methods over time. The medication event monitoring system (MEMS^TM) was used to track the dates and times a pill bottle was opened. The number of times the bottle was opened was divided by the total number of times the bottle should be opened according to provider instructions. Pill count was used to assess how many pills were returned at each restaging visit compared to the number that should be returned if all medication was taken as prescribed. The daily diary was completed by participants, documenting date and timing of doses. The mean of these numbers was computed across groups of cycles (1 cycle = 28 days) 1-4, 5-8, 9-12 and 13-18 for the MEMS^TM, Pill Count, and Daily Diary. | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8 . 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | ANOVA F-Value | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), an range of approximately 112 days to 504 days |
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| Secondary | Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap and age, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4, cycles 5-8, cycles 9-12, and cycles 13-18. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficient | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap for years of education, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. Number of years of education was collected through participant self-report and was a continuous variable. | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficient | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Quality of Life (QOL) Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference T-scores | Outcome measures correlations between 2 variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap & PROMIS Pain Interference T- Scores (Same for all PROMIS measures) Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-13hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle & dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. PROMIS Pain Interference raw scores transformed to T-scores for analysis (mean:50,standard deviation: 10, higher scores is more pain interference, T of 55 is cutoff-mild pain interference). | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficient | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Quality of Life (QOL) Patient-Reported Outcomes Measurement Information System (PROMIS) Depression T-scores | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap and PROMIS Depression Scores, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. PROMIS Depression raw scores were transformed to T-scores for analysis (mean: 50, standard deviation: 10, higher scores is more depression symptoms, T of 55 is the cut off for mild depressive symptoms). | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficients | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Quality of Life (QOL) Patient-Reported Outcomes Measurement Information System (PROMIS) Cognitive Interference T-scores | The outcome measures correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. To assess the relationship between the adherence method MEMS^TM cap and PROMIS Cognitive Interference Scores, Spearman correlation was used. For this analysis, MEMS^TM was defined as the average mean adherence for cycles 1-4. A dose was considered adherent if it was taken within an 11-to-13-hour interval from the prior correct dose. The mean was computed by summing medication adherence across the cycle and dividing it by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data to compute the average. PROMIS Cognitive Interference raw scores were transformed to T-scores for analysis (mean: 50, standard deviation: 10, higher scores is more cognitive interference, T of 60 represents mild interference). | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficient | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Barriers to Adherence Questionnaire | Participants completed a barriers questionnaire to assess barriers most frequently endorsed on the questionnaire. The questionnaire focused on any medication adherence issues during the trial (e.g., missed doses). Participants were given 16 possible reasons that they may have missed a dose and were asked to check all items that caused them to miss a dose of medication. Participants were also able to provide additional reasons for missing medication that were not on the form. | Six participants were given the barriers to adherence questionnaire at baseline due to error. One participant was not given the barriers to adherence questionnaire at follow-up due to error, so only 11/12 participants had this at follow-up. | Posted | Count of Participants | Participants | Baseline and follow-up (between cycles 8 and 12; or after cycle 18/when taken off of study (range of 224-504 days) |
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| Secondary | Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Barriers to Adherence Questionnaire | The outcome measures Spearman correlations between two variables and the resulting correlation coefficient describes the strength of the relationship between the variables. MEMS^TM was defined as the average mean adherence, an adherent dose was taken 11-13 hours from the prior correct dose. The mean was computed by dividing medication adherence across the cycle by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data. Barriers to adherence was calculated by summing the number of barriers for each participant at follow-up. Participants were given 16 possible reasons that they may have missed a dose and were asked to check all items that apply to them. | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficient | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Medication Event Monitoring Systems (MEMS^TM) Cap and Stressful Life Events: Number of Stressful Life Events Endorsed in the Life Events Checklist | To assess the relationship between the adherence method MEMS^TM cap and stressful life events (i.e., the number of stressful life events endorsed in the Life Events Checklist), Spearman correlation was used. Participants complete a 22-item questionnaire to report stressful events that impact medication adherence. MEMS^TM was defined as the average mean adherence, an adherent dose was taken 11-13 hours from the prior correct dose. The mean was computed by dividing medication adherence across the cycle by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data. | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8. 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficient | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Secondary | Medication Event Monitoring Systems (MEMS^TM) Cap and Rating of Overall Stress | To assess the relationship between the adherence method MEMS^TM cap and rating of overall stress, Spearman correlation was used. Participants complete a 1-item question asking them to rate their stress from 0 - 10, with higher numbers being more stressed. MEMS^TM was defined as the average mean adherence, an adherent dose was taken 11-13 hours from the prior correct dose. The mean was computed by dividing medication adherence across the cycle by the number of days within the cycle. Then, the average mean was calculated by using Statistical Package for the Social Sciences (SPSS) MEAN function, which uses all non-missing data. | 11/12 participants (pts) analyzed Cycles(C) 1-4. 1 pts MEMS cap malfunctioned (= 0 data across any C). 10/12 pts analyzed C5-8 . 1 additional pt withdrew from the study, resulting in 0 MEMS data beginning C5. 8/12 pts analyzed C9-12 & C13-18. 1 pt withdrew from study after C9 & 1 failed to return their MEMS Cap. Due to battery expiration between clinic visits, at least 1C was unable to be obtained for 5pts. And 1pt had missing data due to forgetting to replace the cap on the next cycle's bottle. | Posted | Number | Correlation coefficient | Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days |
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| Other Pre-specified | Number of Participants With Serious and/or Non-serious Adverse Events | Here is the number of participants with serious and/or non-serious adverse events. A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. | As pre-specified by the protocol, adverse events occurring because of treatment for the underlying condition or medical treatment for pain will NOT be recorded in this study. | Posted | Count of Participants | Participants | Baseline to cycle 18 (end of study), approximately 504 days (1.38 years) |
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Baseline to cycle 18 (end of study), approximately 504 days (1.38 years)
As pre-specified by the protocol, adverse events occurring as a result of treatment for the underlying condition or medical treatment for pain will NOT be recorded on this study.
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 | Adult Participants (18+ Years) | Questionnaires and use of the medication event monitoring system (MEMS^TM) Participants must take an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s). Medication Event Monitoring System (MEMS^TM) caps will be used to monitor adherence over time along with patient diaries and pill counts. Medication Event Monitoring System (MEMS^TM): A computerized method of tracking the dates and times of a pill bottle being opened. | 0 | 12 | 0 | 12 | 0 | 12 |
Not provided
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Staci M Peron, Ph.D. | National Cancer Institute | 240-760-6025 | martins@mail.nih.gov |
| Apr 16, 2024 |
| Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Nov 17, 2020 | Apr 16, 2024 | ICF_001.pdf |
| ID | Term |
|---|---|
| D009456 | Neurofibromatosis 1 |
| D018318 | Neurofibroma, Plexiform |
| ID | Term |
|---|---|
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009386 | Neoplastic Syndromes, Hereditary |
| D020752 | Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D010524 | Peripheral Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
Not provided
Not provided
| ID | Term |
|---|---|
| D011795 | Surveys and Questionnaires |
| ID | Term |
|---|---|
| D003625 | Data Collection |
| D004812 | Epidemiologic Methods |
| D008919 | Investigative Techniques |
| D017531 | Health Care Evaluation Mechanisms |
| D011787 | Quality of Health Care |
| D017530 | Health Care Quality, Access, and Evaluation |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
Not provided
Not provided
| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| OG000 |
| Adult Participants (18+ Years) |
Questionnaires and use of the medication event monitoring system (MEMS) Participants must take an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s). Medication Event Monitoring System (MEMS) caps will be used to monitor adherence over time along with patient diaries and pill counts. Medication Event Monitoring System (MEMS): A computerized method of tracking the dates and times of a pill bottle being opened. |
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| Adult Participants (18+ Years) |
Questionnaires and use of the medication event monitoring system (MEMS) Participants must take an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s). Medication Event Monitoring System (MEMS) caps will be used to monitor adherence over time along with patient diaries and pill counts. Medication Event Monitoring System (MEMS): A computerized method of tracking the dates and times of a pill bottle being opened. |
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| OG000 |
| Adult Participants (18+ Years) |
Questionnaires and use of the medication event monitoring system (MEMS) Participants must take an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s). Medication Event Monitoring System (MEMS) caps will be used to monitor adherence over time along with patient diaries and pill counts. Medication Event Monitoring System (MEMS): A computerized method of tracking the dates and times of a pill bottle being opened. |
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