Medication Adherence and Non-adherence in Adults With Rare Disease

An observational study of Cystic Fibrosis and Hemophilia A, run by Xperiome.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Trial 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… Read more about this study

Who is it for?: Ages 18 years and olderHealthy volunteers can take part
Conditions the study is for: Cystic Fibrosis, Hemophilia A, Hemophilia B
What is being tested?: MAR-Scale
How many people: 0 participants
Where: No sites listed yet
Study dates: Jan 2021to Sep 2022
Phase: Observational
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
Healthy volunteers accepted
Adult, Older adult

Study population: Anyone who fits the eligibility criteria and gives their consent to take part in this study.

Who may be able to join

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

Show all 11Show fewer

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

Who cannot join

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.

About this study

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

Conditions: Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis, Myasthenia Gravis, Sickle Cell Disease
Being tested: MAR-Scale
Keywords: Adherence, MAR-Scale, Rare disease, Observational
Medical subject headings: Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis, Myasthenia Gravis, Anemia, Sickle Cell, Rare Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT04541875
Study group	Role	What they receive
Cystic fibrosis		Patients aged 18+ and diagnosed with cystic fibrosis. Patients will answer the MAR-Scale once every three months for a year. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: Other: MAR-Scale

What is being measured

The primary outcome is the main question the study is designed to answer.

To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
Measured: Baseline
To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
Measured: 3 months
To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
Measured: 6 months
To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
Measured: 9 months
To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
Percentage of respondents who indicated at least one reason for non-adherence in the past seven days.
Measured: 1 year
To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
Tally of reasons for non-adherence.
Measured: Baseline
To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
Tally of reasons for non-adherence.
Measured: 3 months
To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
Tally of reasons for non-adherence.
Measured: 6 months
To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
Tally of reasons for non-adherence.
Measured: 9 months
To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
Tally of reasons for non-adherence.
Measured: 1 year

Secondary outcomes (1)

To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients.
Use the 200 responses collected from each condition to determine the Cronbach's alpha of the scale for each condition.
Measured: Baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2021Sep 2022

First posted9 Sep 2020
Study startJan 2021 (estimated)
Primary completionSep 2022 (estimated)
Study completionSep 2022 (estimated)

Why it stopped: Liquidation of sponsor

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04541875
Sponsor's protocol ID: RM-RP005
Status last verified: May 2022
First submitted: 26 Aug 2020
Last updated: 12 May 2022
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Undecided

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 12 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04541875
Acronym / sponsor ID: RM-RP005
Conditions: Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis, Myasthenia Gravis, Sickle Cell Disease
Keywords: Adherence, MAR-Scale, Rare disease, Observational
Study type: Observational
Recruitment status: WithdrawnReason stopped: Liquidation of sponsor
Enrolment: 0 participants (actual)
Lead sponsor: XperiomeUniversity, hospital or other
Collaborators: The Touro College and University System
Study start: Jan 2021 (estimated)
Primary completion: Sep 2022 (estimated)
Study completion: Sep 2022 (estimated)
First posted: 9 Sep 2020 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model: Cohort
Time perspective: Prospective
Number of arms: 5

Arm	Type	Receives
Cystic fibrosis	–	Other: MAR-ScalePatients aged 18+ and diagnosed with cystic fibrosis. Patients will answer the MAR-Scale once every three months for a year.
Hemophilia A or B	–	Other: MAR-ScalePatients aged 18+ and diagnosed with hemophilia A or B. Patients will answer the MAR-Scale once every three months for a year.
Idiopathic pulmonary fibrosis	–	Other: MAR-ScalePatients aged 18+ and diagnosed with idiopathic pulmonary fibrosis. Patients will answer the MAR-Scale once every three months for a year.
Myasthenia gravis	–	Other: MAR-ScalePatients aged 18+ and diagnosed with myasthenia gravis. Patients will answer the MAR-Scale once every three months for a year.
Sickle cell disease	–	Other: MAR-ScalePatients aged 18+ and diagnosed with sickle cell disease. Patients will answer the MAR-Scale once every three months for a year.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult
Study population: Anyone who fits the eligibility criteria and gives their consent to take part in this study.

Key inclusion criteria (11 listed)

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

Key exclusion criteria (1 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
MAR-ScaleThe MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.	Other	Cystic fibrosis, Hemophilia A or B, Idiopathic pulmonary fibrosis, Myasthenia gravis, Sickle cell disease

Primary outcome

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.

1 secondary outcome

To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients.
Time frame: Baseline

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2022.
Individual participant dataSharing planned: Undecided

Related trials for Cystic Fibrosis

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Elizabeth J Unni, BpharmMBAPhD principal investigator

Touro College of Pharmacy

Questions about this trial

What is this clinical trial studying?

An observational study of Cystic Fibrosis and Hemophilia A, run by Xperiome.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 12 May 2022. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04541875.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 May 2022.