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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

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.

Condition or disease Intervention/treatment
Cystic Fibrosis Hemophilia A Hemophilia B Idiopathic Pulmonary Fibrosis Myasthenia Gravis Sickle Cell Disease Other: MAR-Scale

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Study Type : Observational
Estimated Enrollment : 1000 participants
Observational Model: Cohort
Time Perspective: Prospective
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
Estimated Study Start Date : January 2021
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : September 2022


Group/Cohort Intervention/treatment
Cystic fibrosis

Patients aged 18+ and diagnosed with cystic fibrosis.

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

Other: MAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.

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.

Other: MAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.

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.

Other: MAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.

Myasthenia gravis

Patients aged 18+ and diagnosed with myasthenia gravis.

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

Other: MAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.

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.

Other: MAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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.


Secondary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Anyone who fits the eligibility criteria and gives their consent to take part in this study.
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.

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): NCT04541875


Contacts
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Contact: Pete Chan, BSc +44 (0) 20 3920 9880 pete.chan@raremark.com

Sponsors and Collaborators
Xperiome
The Touro College and University System
Investigators
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Principal Investigator: Elizabeth J Unni, BpharmMBAPhD Touro College of Pharmacy
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Responsible Party: Xperiome
ClinicalTrials.gov Identifier: NCT04541875    
Other Study ID Numbers: RM-RP005
First Posted: September 9, 2020    Key Record Dates
Last Update Posted: December 16, 2020
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Xperiome:
Adherence
MAR-Scale
Rare disease
Observational
Additional relevant MeSH terms:
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Cystic Fibrosis
Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis
Myasthenia Gravis
Hemophilia A
Anemia, Sickle Cell
Hemophilia B
Fibrosis
Rare Diseases
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hemoglobinopathies
Idiopathic Interstitial Pneumonias
Lung Diseases, Interstitial
Disease Attributes
Genetic Diseases, X-Linked
Autoimmune Diseases of the Nervous System