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

NCT03531814 | Completed Results

• 2 other identifiers: 18009318-C-0093
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Neurofibromatosis 1; Neurofibroma, Plexiform

Keywords

Plexiform Neurofibromas; Medication Event Monitoring Systems; Oral Medication

Outcome Measures

Primary Outcomes (2)

• Proportion of Enrolled Participants for Which we Are Able to Collect Data From the Medication Event Monitoring Systems (MEMS^TM) System for Two or More Cycles of Treatment (Target = 75%)

  The proportion of enrolled participants are reported for individuals who had two full cycles of data recorded by the medication event monitoring systems (MEMS\^TM) system. To monitor medication adherence the medication event monitoring system (MEMSTM) used to track the dates and times a pill bottle was opened. At least 2 cycles of medication adherence are considered a success.

  Two cycles, approximately 56 days

• Median Number of Cycles Monitored for Participants From the Medication Event Monitoring System (MEMS^TM)

  To monitor medication adherence the medication event monitoring system (MEMSTM) was used to track the dates and times a pill bottle was opened. We calculated the median number of cycles monitored for all patients.

  Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Secondary Outcomes (11)

• Average Percent Medication Adherence Over Time Based on the Medication Event Monitoring Systems (MEMS^TM) Pill Cap Data

  Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

• ANOVA (Analysis of Variance) Comparing Average Adherence Measured by Medication Event Monitoring Systems (MEMS^TM), Medication Diary, and Pill Count

  Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), an range of approximately 112 days to 504 days

• Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Age

  Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

• Spearman Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap for Years of Education

  Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

• Spearman's Correlation of the Relationship Between Medication Event Monitoring Systems (MEMS^TM) Cap and Quality of Life (QOL) Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference T-scores

  Cycles 1-4, 5-8, 9-12 and 13-18 (1 cycle = 28 days), a range of approximately 112 days to 504 days

Other Outcomes (1)

• Number of Participants With Serious and/or Non-serious Adverse Events

  Baseline to cycle 18 (end of study), approximately 504 days (1.38 years)

Study Arms (2)

Adult Participants

EXPERIMENTAL

Questionnaires and use of the medication event monitoring system (MEMS\^TM)

Behavioral: Medication Event Monitoring System (MEMS^TM); Other: Questionnaires

Pediatric Participants 8+ Years

EXPERIMENTAL

Questionnaires and use of the medication event monitoring system (MEMS\^TM)

Behavioral: Medication Event Monitoring System (MEMS^TM); Other: Questionnaires

Interventions

Medication Event Monitoring System (MEMS^TM) BEHAVIORAL

A computerized method of tracking the dates and times of a pill bottle being opened.

Adult Participants; Pediatric Participants 8+ Years

Questionnaires OTHER

Adult and pediatric participants completed a series of questionnaires to assess medication adherence, demographics, life events, and barriers to adherence.

Adult Participants; Pediatric Participants 8+ Years

Eligibility Criteria

• Age: 3 Years - 59 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients must be between 3 and 59 years of age at the time of the baseline assessment.
• Patients must be enrolled on a neurofibromatosis Type 1 (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.
• 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.

You may not qualify if:

• In the opinion of the principal investigator (PI) or an associate investigator (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 taking routine would be significantly different from the general population of patients with NF1.
• Patients receiving the study drug in liquid form, since the use of medication event monitoring systems (MEMS\^TM) caps prohibits liquid dosing.
• None

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma, Plexiform

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; 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; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Results Point of Contact

• Title: Staci M Peron, Ph.D.
• Organization: National Cancer Institute

martins@mail.nih.gov

240-760-6025

Study Officials

• Staci M Peron, Ph.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: May 19, 2018
• First Posted: May 22, 2018
• Study Start: October 23, 2018
• Primary Completion: March 20, 2023
• Study Completion: March 20, 2023
• Last Updated: June 11, 2024
• Results First Posted: June 11, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Clinical data available during the study and indefinitely.
• Access Criteria: Clinical data will be made available via subscription to Biomedical Translational Research Information System (BTRIS) and with the permission of the study principal investigator (PI).

Locations

US (1)