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



Status:Recruiting
Conditions:Cancer, Other Indications
Therapuetic Areas:Oncology, Other
Healthy:No
Age Range:3 - 99
Updated:11/2/2018
Start Date:October 23, 2018
End Date:September 30, 2019
Contact:Andrea M Gross, M.D.
Email:andrea.gross@nih.gov
Phone:(301) 443-5393

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

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.

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 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.

- 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
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