Trial Outcomes & Findings for Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials (NCT NCT03531814)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

12 participants

Primary outcome timeframe

Two cycles, approximately 56 days

Results posted on

2024-06-11

Participant Flow

No pediatric participants were enrolled on this trial.

Participant milestones

Participant milestones
Measure	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.	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.
Overall Study STARTED	12	0
Overall Study COMPLETED	12	0
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Adult Participants (18+ Years) n=12 Participants 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.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	34.36 years STANDARD_DEVIATION 8.91 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants

PRIMARY outcome

Timeframe: Two cycles, approximately 56 days

Population: 11/12 participants were analyzed because 1 participant's cap malfunctioned, so no data was collected.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
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%)	0.9167 Proportion of participants

PRIMARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 11/12 participants were analyzed because 1 participant's cap malfunctioned, so no data was collected.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Median Number of Cycles Monitored for Participants From the Medication Event Monitoring System (MEMS^TM)	16 Cycles Interval 0.0 to 18.0

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data Cycles 1-4	83.39 percent Standard Deviation 16.71
Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data Cycles 5-8	87.01 percent Standard Deviation 14.62
Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data Cycles 9-12	84.77 percent Standard Deviation 15.44
Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data Cycles 13-18	79.68 percent Standard Deviation 20.85

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), an range of approximately 112 days to 504 days

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count Cycles 1-4	11.944 ANOVA F-Value
ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count Cycles 5-8	7.770 ANOVA F-Value
ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count Cycles 9-12	6.670 ANOVA F-Value
ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count Cycles 13-18	6.333 ANOVA F-Value

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age Cycles 1-4	-0.210 Correlation coefficient
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age Cycles 5-8	0.134 Correlation coefficient
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age Cycles 9-12	.095 Correlation coefficient
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age Cycles 13-18	.333 Correlation coefficient

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education Cycles 1-4	-0.333 Correlation coefficient
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education Cycles 5-8	-0.309 Correlation coefficient
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education Cycles 9-12	-0.259 Correlation coefficient
Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education Cycles 13-18	-0.222 Correlation coefficient

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
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 Cycles 1-4	-.215 Correlation coefficient
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 Cycles 5-8	0.049 Correlation coefficient
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 Cycles 9-12	-0.048 Correlation coefficient
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 Cycles 13-18	-0.167 Correlation coefficient

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
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 Cycles 1-4	-.607 Correlation coefficients
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 Cycles 5-8	-0.658 Correlation coefficients
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 Cycles 9-12	-0.708 Correlation coefficients
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 Cycles 13-18	-0.878 Correlation coefficients

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
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 Cycles 1-4	0.293 Correlation coefficient
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 Cycles 5-8	0.278 Correlation coefficient
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 Cycles 9-12	0.229 Correlation coefficient
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 Cycles 13-18	0.530 Correlation coefficient

SECONDARY outcome

Timeframe: Baseline and follow-up (between cycles 8 and 12; or after cycle 18/when taken off of study (range of 224-504 days)

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Away from home	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Busy with other things	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Forgot	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Had too many pills to take	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Wanted to avoid having side effects	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Did not want others to see taking medication	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Change in daily routine	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Too tired or slept through dose time	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Felt sick or ill	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Felt sad or stressed out	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Had problem taking medicine with clear liquid	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Had difficulty swallowing pills	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Ran out of pills	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Didn't like taste of medicine	0 Participants
Barriers to Adherence Questionnaire Didn't think medicine was working	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Didn't feel like it/Needed a break	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at baseline: Other reason	0 Participants
Barriers to Adherence Questionnaire Most commonly reported barrier at follow-up: Forgetting	6 Participants

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Barriers to Adherence Questionnaire Cycles 9-12	-0.835 Correlation coefficient
Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Barriers to Adherence Questionnaire Cycles 13-18	-0.933 Correlation coefficient
Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Barriers to Adherence Questionnaire Cycles 5-8	-0.680 Correlation coefficient
Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Barriers to Adherence Questionnaire Cycles 1-4	-0.576 Correlation coefficient

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Medication Event Monitoring Systems (MEMS^TM) Cap and Stressful Life Events: Number of Stressful Life Events Endorsed in the Life Events Checklist Cycles 1-4	-0.035 Correlation coefficient
Medication Event Monitoring Systems (MEMS^TM) Cap and Stressful Life Events: Number of Stressful Life Events Endorsed in the Life Events Checklist Cycles 5-8	0.227 Correlation coefficient
Medication Event Monitoring Systems (MEMS^TM) Cap and Stressful Life Events: Number of Stressful Life Events Endorsed in the Life Events Checklist Cycles 9-12	0.179 Correlation coefficient
Medication Event Monitoring Systems (MEMS^TM) Cap and Stressful Life Events: Number of Stressful Life Events Endorsed in the Life Events Checklist Cycles 13-18	0.041 Correlation coefficient

SECONDARY outcome

Timeframe: Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=11 Participants 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.
Medication Event Monitoring Systems (MEMS^TM) Cap and Rating of Overall Stress Cycles 1-4	-0.638 Correlation coefficient
Medication Event Monitoring Systems (MEMS^TM) Cap and Rating of Overall Stress Cycles 5-8	-0.361 Correlation coefficient
Medication Event Monitoring Systems (MEMS^TM) Cap and Rating of Overall Stress Cycles 9-12	-0.407 Correlation coefficient
Medication Event Monitoring Systems (MEMS^TM) Cap and Rating of Overall Stress Cycles 13-18	-0.180 Correlation coefficient

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to cycle 18 (end of study), approximately 504 days (1.38 years)

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Adult Participants (18+ Years) n=12 Participants 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.
Number of Participants With Serious and/or Non-serious Adverse Events	0 Participants

Adverse Events

Adult Participants (18+ Years)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Staci M Peron, Ph.D.

National Cancer Institute

Phone: 240-760-6025

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place