N/A
N=12
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials
Neurofibromatosis 1 · Neurofibroma, Plexiform
Bottom Line
View on ClinicalTrials.gov: NCT03531814 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jun 2024
Primary outcome: 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%) — 0.9167 Proportion of participants
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Medication Event Monitoring System (MEMS^TM) (Behavioral); Questionnaires (Other)
- Age
- Pediatric, Adult · 3+ yrs
- Sex
- All
- Sponsor
- National Cancer Institute (NCI)
- Primary completion
- Mar 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| 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%) |
0.9167 | — |
| PRIMARY Median Number of Cycles Monitored for Participants From the Medication Event Monitoring System (MEMS^TM) |
16 | — |
| SECONDARY Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data |
83.39; 87.01; 84.77; 79.68 | — |
| SECONDARY ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count |
11.944; 7.770; 6.670; 6.333 | 0.006 sig |
| SECONDARY Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age |
-0.210; 0.134; .095; .333 | 0.536 |
| SECONDARY Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education |
-0.333; -0.309; -0.259; -0.222 | 0.317 |
| 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 |
-.215; 0.049; -0.048; -0.167 | 0.526 |
| 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 |
-.607; -0.658; -0.708; -0.878 | 0.048 sig |
| 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 |
0.293; 0.278; 0.229; 0.530 | 0.382 |
| SECONDARY Barriers to Adherence Questionnaire |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Barriers to Adherence Questionnaire |
-0.576; -0.680; -0.835; -0.933 | 0.063 |
| SECONDARY Medication Event Monitoring Systems (MEMS^TM) Cap and Stressful Life Events: Number of Stressful Life Events Endorsed in the Life Events Checklist |
-0.035; 0.227; 0.179; 0.041 | 0.919 |
| SECONDARY Medication Event Monitoring Systems (MEMS^TM) Cap and Rating of Overall Stress |
-0.638; -0.361; -0.407; -0.180 | 0.035 sig |
Summary
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.
Eligibility Criteria
- INCLUSION CRITERIA:
Inclusion Criteria for Patient
- Patients must be between 3 and 59 years of age at the time of the baseline assessment.
- Patients must be enrolled on a neurofibromatosis Type 1 (NF1) clinical trial for an oral medication directed at the treatment of plexiform neurofibroma(s) (enrollment on this study to occur ideally within 1st cycle) Patients must have regular access to a computer or electronic device (e.g., smartphone, tablet) with internet access.
- Must have a parent or adult primary caregiver willing to participate in the study.
- Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
- Subjects must be able to read and comprehend the English language.
Inclusion Criteria for Parents or Caregivers
- Must be a parent or primary caregiver of a child (or if applicable adult patient) of diagnosed with NF1 and enrolled on a clinical trial for oral medication.
- Must have a child (or if applicable adult patient) willing to participate in the study
- Must have regular access to a computer or electronic device (e.g., smartphone, tablet) with internet access.
- Must be able to speak and understand English.
- Ability of subject to understand and the willing to sign a written informed consent document.
EXCLUSION CRITERIA
Exclusion Criteria for Patient
- In the opinion of the principal investigator (PI) or an associate investigator (AI), the subject has significant cognitive or emotional difficulties that would prevent them from being able to understand and/or participate fully in the study or complete the measures. Though these patients might be receiving assistance in taking medication from a caregiver, it is likely that their medication taking routine would be significantly different from the general population of patients with NF1.
- Patients receiving the study drug in liquid form, since the use of medication event monitoring systems (MEMS^TM) caps prohibits liquid dosing.
Exclusion Criteria for Parent or Caregiver
None
Data sourced from ClinicalTrials.gov (NCT03531814). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.