WithdrawnNot Applicable

Medication Adherence and Non-adherence in Adults With Rare Disease

Stopped: Liquidation of sponsor

0Started 2021-01

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.

Timeframe: Baseline

To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.

Timeframe: 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.

Timeframe: 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.

Timeframe: 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.

Timeframe: 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.

Timeframe: Baseline

Trial details

NCT IDNCT04541875

SponsorXperiome

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2022-09

View on ClinicalTrials.gov More Cystic Fibrosis trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.

Timeframe: Baseline

To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.

Timeframe: 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.

Timeframe: 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.

Timeframe: 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.

Timeframe: 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.

Timeframe: Baseline