Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03531814|
Recruitment Status : Recruiting
First Posted : May 22, 2018
Last Update Posted : October 31, 2018
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.
To study how often people with neurofibromatosis type 1 take medicine that has been prescribed to them for treating plexiform neurofibromas.
People ages 3-59 already enrolled in an NF1 clinical trial
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:
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.
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis 1 Neurofibroma, Plexiform||Behavioral: Medication Event Monitoring System (MEMS)||Not Applicable|
- 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.
- To establish the feasibility of using MEMSTM to monitor medication adherence in the NF1 population
- 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)
- This multi-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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Supportive Care|
|Official Title:||Pilot Study of Medication Adherence in Children, Adolescents, and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials|
|Actual Study Start Date :||October 23, 2018|
|Estimated Primary Completion Date :||September 30, 2019|
|Estimated Study Completion Date :||September 30, 2019|
Questionnaires and use of the medication event monitoring system (MEMS)
Behavioral: Medication Event Monitoring System (MEMS)
A computerized method of tracking the dates and times of a pill bottle being opened.
- Feasibility [ Time Frame: Two cycles ]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%)
- Patient Characteristics [ Time Frame: Two cycles ]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
- Pattern [ Time Frame: Cycles 1-4, 5-8, 9-12 and 13-18 ]Average percent adherence based on the MEMSTM data only for cycles 1-4, 5-8, 9-12 and 13-18
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03531814
|Contact: Andrea M Gross, M.D.||(301) email@example.com|
|United States, Maryland|
|Johns Hopkins University||Recruiting|
|Baltimore, Maryland, United States, 21205|
|Contact: Jaishri Blakeley 410-955-6827 firstname.lastname@example.org|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937|
|United States, Massachusetts|
|Boston Children s Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Nicole Ullrich 617-355-3193 email@example.com|
|United States, Pennsylvania|
|Childrens Hospital, Philadelphia||Recruiting|
|Philadelphia, Pennsylvania, United States, 19104|
|Contact: Michael Fisher, M.D. 215-590-5188 firstname.lastname@example.org|
|Principal Investigator:||Staci M Peron, Ph.D.||National Cancer Institute (NCI)|