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

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ClinicalTrials.gov Identifier: NCT03531814
Recruitment Status : Recruiting
First Posted : May 22, 2018
Last Update Posted : October 31, 2018
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

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.


Condition or disease Intervention/treatment Phase
Neurofibromatosis 1 Neurofibroma, Plexiform Behavioral: Medication Event Monitoring System (MEMS) Not Applicable

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 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


Arm Intervention/treatment
Experimental: 1/Intervention
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.




Primary Outcome Measures :
  1. 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%)


Secondary Outcome Measures :
  1. 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

  2. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 99 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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


Information from the National Library of Medicine

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


Contacts
Contact: Andrea M Gross, M.D. (301) 443-5393 andrea.gross@nih.gov

Locations
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21205
Contact: Jaishri Blakeley    410-955-6827    jblakel3@jhmi.edu   
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    nicole.ullrich@childens.harvard.edu   
United States, Pennsylvania
Childrens Hospital, Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Michael Fisher, M.D.    215-590-5188    fisherm@email.chop.edu   
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
Principal Investigator: Staci M Peron, Ph.D. National Cancer Institute (NCI)

Additional Information:
Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT03531814     History of Changes
Other Study ID Numbers: 180093
18-C-0093
First Posted: May 22, 2018    Key Record Dates
Last Update Posted: October 31, 2018
Last Verified: June 25, 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Plexiform Neurofibromas
Medication Event Monitoring Systems
Oral Medication

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms