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Medication Adherence and Non-adherence in Adults With Rare Disease

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ClinicalTrials.gov Identifier: NCT04541875
Recruitment Status : Not yet recruiting
First Posted : September 9, 2020
Last Update Posted : December 16, 2020
Sponsor:
Collaborator:
The Touro College and University System
Information provided by (Responsible Party):
Xperiome

Tracking Information
First Submitted Date August 26, 2020
First Posted Date September 9, 2020
Last Update Posted Date December 16, 2020
Estimated Study Start Date January 2021
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 1, 2020)
  • To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. [ Time Frame: Baseline ]
    Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
  • To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. [ Time Frame: 3 months ]
    Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
  • To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. [ Time Frame: 6 months ]
    Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
  • To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. [ Time Frame: 9 months ]
    Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
  • To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. [ Time Frame: 1 year ]
    Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
  • To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. [ Time Frame: Baseline ]
    Tally of reasons for non-adherence.
  • To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. [ Time Frame: 3 months ]
    Tally of reasons for non-adherence.
  • To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. [ Time Frame: 6 months ]
    Tally of reasons for non-adherence.
  • To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. [ Time Frame: 9 months ]
    Tally of reasons for non-adherence.
  • To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. [ Time Frame: 1 year ]
    Tally of reasons for non-adherence.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: September 1, 2020)
To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients. [ Time Frame: Baseline ]
Use the 200 responses collected from each condition to determine the Cronbach's alpha of the scale for each condition.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Medication Adherence and Non-adherence in Adults With Rare Disease
Official Title A Longitudinal, Digital Study Using the Medication Adherence Reasons Scale (MAR-Scale) to Identify the Reasons for Non-adherence to Medications in Rare Disease
Brief Summary The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease, and to identify the top patient-reported reasons for non-adherence. Internal reliability of the MAR-Scale will also be assessed in each condition.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Anyone who fits the eligibility criteria and gives their consent to take part in this study.
Condition
  • Cystic Fibrosis
  • Hemophilia A
  • Hemophilia B
  • Idiopathic Pulmonary Fibrosis
  • Myasthenia Gravis
  • Sickle Cell Disease
Intervention Other: MAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.
Study Groups/Cohorts
  • Cystic fibrosis

    Patients aged 18+ and diagnosed with cystic fibrosis.

    Patients will answer the MAR-Scale once every three months for a year.

    Intervention: Other: MAR-Scale
  • Hemophilia A or B

    Patients aged 18+ and diagnosed with hemophilia A or B.

    Patients will answer the MAR-Scale once every three months for a year.

    Intervention: Other: MAR-Scale
  • Idiopathic pulmonary fibrosis

    Patients aged 18+ and diagnosed with idiopathic pulmonary fibrosis.

    Patients will answer the MAR-Scale once every three months for a year.

    Intervention: Other: MAR-Scale
  • Myasthenia gravis

    Patients aged 18+ and diagnosed with myasthenia gravis.

    Patients will answer the MAR-Scale once every three months for a year.

    Intervention: Other: MAR-Scale
  • Sickle cell disease

    Patients aged 18+ and diagnosed with sickle cell disease.

    Patients will answer the MAR-Scale once every three months for a year.

    Intervention: Other: MAR-Scale
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Not yet recruiting
Estimated Enrollment
 (submitted: September 1, 2020)
1000
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 2022
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Have a confirmed (self-reported) diagnosis by a doctor or other licensed healthcare professional of one of the following conditions:
  • Cystic fibrosis
  • Hemophilia (A or B)
  • Idiopathic pulmonary fibrosis
  • Myasthenia gravis
  • Sickle cell disease
  • Have access to the internet
  • Are aged 18 years or above
  • Are comfortable reading and answering questions in English
  • Have an active prescription for at least one medication indicated to treat the relevant condition (self-reported)
  • NB: Individuals will be eligible even if the prescription has not been dispensed (filled) yet, and also if they have the medication(s), but are not actually taking it/them Are able and willing to provide consent electronically through the my.raremark.com platform

Exclusion Criteria:

  • There are no exclusion criteria. Any member of a Raremark community will be able to take part in this study if they meet the inclusion criteria and can provide their informed consent.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Pete Chan, BSc +44 (0) 20 3920 9880 pete.chan@raremark.com
Listed Location Countries Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number NCT04541875
Other Study ID Numbers RM-RP005
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Undecided
Responsible Party Xperiome
Study Sponsor Xperiome
Collaborators The Touro College and University System
Investigators
Principal Investigator: Elizabeth J Unni, BpharmMBAPhD Touro College of Pharmacy
PRS Account Xperiome
Verification Date December 2020