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

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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

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.
    


Study Type

Interventional


Primary Outcome

Feasibility

Secondary Outcome

 Patient Characteristics

Condition

Neurofibromatosis 1

Intervention

Medication Event Monitoring System (MEMS)

Study Arms / Comparison Groups

 1/Intervention
Description:  Questionnaires and use of the medication event monitoring system (MEMS)

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Behavioral

Estimated Enrollment

30

Start Date

October 23, 2018

Completion Date

September 30, 2022

Primary Completion Date

September 30, 2022

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
      

Gender

All

Ages

3 Years - N/A

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Staci M Peron, Ph.D., (240) 858-3853, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03531814

Organization ID

180093

Secondary IDs

18-C-0093

Responsible Party

Sponsor

Study Sponsor

National Cancer Institute (NCI)


Study Sponsor

Staci M Peron, Ph.D., Principal Investigator, National Cancer Institute (NCI)


Verification Date

August 31, 2021