Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials
Primary Purpose
Neurofibromatosis 1, Neurofibroma, Plexiform
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Medication Event Monitoring System (MEMS)
Sponsored by
About this trial
This is an interventional supportive care trial for Neurofibromatosis 1 focused on measuring Plexiform Neurofibromas, Medication Event Monitoring Systems, Oral Medication
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 an 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 PI or an 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 takingroutine would be significantly different from the general population of patients with NF1.
- Patients receiving the study drug in liquid form, since the use of MEMSTM caps prohibits liquid dosing.
Exclusion Criteria for Parent or Caregiver
None
Sites / Locations
- National Institutes of Health Clinical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
1/Intervention
Arm Description
Questionnaires and use of the medication event monitoring system (MEMS)
Outcomes
Primary Outcome Measures
Feasibility
Proportion of enrolled patients for which we are able to collect data from the MEMSTM system for two or more cycles of treatment (target = 75%)
Secondary Outcome Measures
Pattern
Average percent adherence based on the MEMSTM data only for cycles 1-4, 5-8, 9-12 and 13-18
Patient Characteristics
Relationships between adherence (correlations, t-tests, etc.) and demographics, life events and barriers to adherence will be used to identify patient characteristics that may predispose to poor medication adherence
Full Information
NCT ID
NCT03531814
First Posted
May 19, 2018
Last Updated
September 29, 2023
Sponsor
National Cancer Institute (NCI)
1. Study Identification
Unique Protocol Identification Number
NCT03531814
Brief Title
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials
Official Title
Pilot Study of Medication Adherence in Children, Adolescents, and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials
Study Type
Interventional
2. Study Status
Record Verification Date
September 28, 2023
Overall Recruitment Status
Completed
Study Start Date
October 23, 2018 (Actual)
Primary Completion Date
March 20, 2023 (Actual)
Study Completion Date
March 20, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Cancer Institute (NCI)
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief 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.
Detailed Description
Background:
Neurofibromatosis type 1 (NF1) is a genetic disorder that affects approximately 1 in 3,500 individuals and is associated with a broad variety of symptoms and physical findings.
Plexiform neurofibromas (PN) are histologically benign tumors which occur in 25-50% of patients with NF1 and can lead to significant morbidity.
Oral therapeutic options for the treatment of plexiform neurofibromas are being actively developed, however early clinical data indicate that prolonged treatment over the course of months to years will likely be needed to maintain clinical efficacy
Long-term medication adherence is an ongoing challenge for patients with many types of chronic illness, and clinical experience makes us strongly suspect patients with NF1 will likely have this issue as well.
In other diseases, such as HIV and Acute Lymphoblastic Leukemia, decreased medication adherence has been associated with poorer clinical outcomes, and this may be the case for NF1 as well.
The medication event monitoring systems (MEMSTM) uses a computerized method of tracking the dates and times of a pill bottle being opened, and has been shown to be a more accurate measure of medication adherence than patient diaries or pill counts in other patient populations.
Assessing medication adherence over time in this unique population will be essential for assessing any impact on medical outcomes, identifying potential behavioral interventions, and targeting patients most at risk for nonadherence moving forward.
Objective:
- To establish the feasibility of using MEMSTM to monitor medication adherence in the NF1 population
Eligibility:
Subjects must have a diagnosis of NF1 and be between 3 and 59 years of age
Participants must be enrolled on a clinical trial for an oral medication in pill (tablet or capsule) form directed at the treatment of plexiform neurofibroma(s)
Design:
This single-site, longitudinal study will recruit children and adults with NF1 who are currently enrolled in a treatment protocol for a drug targeting PN volume reduction.
MEMSTM caps will be used to monitor adherence over time along with patient diaries and pill counts.
Patients with MEMSTM cap data indicating <90% adherence at any study visit (typically across 3 - 6 cycles) will be administered a measure assessing barriers to adherence electronically and will be interviewed to evaluate what factors might contribute to decreased medication adherence and what potential interventions they consider useful.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis 1, Neurofibroma, Plexiform
Keywords
Plexiform Neurofibromas, Medication Event Monitoring Systems, Oral Medication
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1/Intervention
Arm Type
Experimental
Arm Description
Questionnaires and use of the medication event monitoring system (MEMS)
Intervention Type
Behavioral
Intervention Name(s)
Medication Event Monitoring System (MEMS)
Intervention Description
A computerized method of tracking the dates and times of a pill bottle being opened.
Primary Outcome Measure Information:
Title
Feasibility
Description
Proportion of enrolled patients for which we are able to collect data from the MEMSTM system for two or more cycles of treatment (target = 75%)
Time Frame
Two cycles
Secondary Outcome Measure Information:
Title
Pattern
Description
Average percent adherence based on the MEMSTM data only for cycles 1-4, 5-8, 9-12 and 13-18
Time Frame
Cycles 1-4, 5-8, 9-12 and 13-18
Title
Patient Characteristics
Description
Relationships between adherence (correlations, t-tests, etc.) and demographics, life events and barriers to adherence will be used to identify patient characteristics that may predispose to poor medication adherence
Time Frame
Two cycles
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
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 an 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 PI or an 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 takingroutine would be significantly different from the general population of patients with NF1.
Patients receiving the study drug in liquid form, since the use of MEMSTM caps prohibits liquid dosing.
Exclusion Criteria for Parent or Caregiver
None
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Staci M Peron, Ph.D.
Organizational Affiliation
National Cancer Institute (NCI)
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Institutes of Health Clinical Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
.All IPD recorded in the medical record will be shared with intramural investigators upon request.
IPD Sharing Time Frame
Clinical data available during the study and indefinitely.
IPD Sharing Access Criteria
Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.
Links:
URL
https://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_2018-C-0093.html
Description
NIH Clinical Center Detailed Web Page
Learn more about this trial
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials
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