Ignite Creation Date:
2024-05-06 @ 11:29 AM
Last Modification Date:
2024-10-26 @ 12:46 PM
Study NCT ID:
NCT03531814
Status:
COMPLETED
Last Update Posted:
2024-06-11
First Post:
2018-05-19
Brief Title:
Medication Adherence in Children Adolescents and Adults With Neurofibromatosis Type 1NF1 on Clinical Treatment Trials
Sponsor:
National Cancer Institute NCI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Pilot Study of Medication Adherence in Children Adolescents and Adults With Neurofibromatosis Type 1 NF1 on Clinical Treatment Trials
Status:
COMPLETED
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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 3500 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 human immunodeficiency virus 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 neurofibromas
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
Neurofibromatosis type 1 NF1 is a genetic disorder that affects approximately 1 in 3500 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 human immunodeficiency virus 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 neurofibromas
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 18-C-0093 | None | None | None |