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Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease

RATIONALE & OBJECTIVE: The development of new therapies for autosomal dominant polycystic kidney disease requires clinical trials to be conducted efficiently. In this study, the factors affecting the recruitment and retention of participants enrolled in a 3-year randomized controlled trial in au...

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Autores principales: Amin, Sneha, Sangadi, Irene, Allman-Farinelli, Margaret, Badve, Sunil V., Boudville, Neil, Coolican, Helen, Coulshed, Susan, Foster, Sheryl, Fernando, Mangalee, Haloob, Imad, Harris, David C.H., Hawley, Carmel M., Holt, Jane, Howell, Martin, Kumar, Karthik, Johnson, David W., Lee, Vincent W., Mai, Jun, Rangan, Anna, Roger, Simon D., Sud, Kamal, Torres, Vicente, Vilayur, Eswari, Rangan, Gopala K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432794/
https://www.ncbi.nlm.nih.gov/pubmed/37602144
http://dx.doi.org/10.1016/j.xkme.2023.100691
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author Amin, Sneha
Sangadi, Irene
Allman-Farinelli, Margaret
Badve, Sunil V.
Boudville, Neil
Coolican, Helen
Coulshed, Susan
Foster, Sheryl
Fernando, Mangalee
Haloob, Imad
Harris, David C.H.
Hawley, Carmel M.
Holt, Jane
Howell, Martin
Kumar, Karthik
Johnson, David W.
Lee, Vincent W.
Mai, Jun
Rangan, Anna
Roger, Simon D.
Sud, Kamal
Torres, Vicente
Vilayur, Eswari
Rangan, Gopala K.
author_facet Amin, Sneha
Sangadi, Irene
Allman-Farinelli, Margaret
Badve, Sunil V.
Boudville, Neil
Coolican, Helen
Coulshed, Susan
Foster, Sheryl
Fernando, Mangalee
Haloob, Imad
Harris, David C.H.
Hawley, Carmel M.
Holt, Jane
Howell, Martin
Kumar, Karthik
Johnson, David W.
Lee, Vincent W.
Mai, Jun
Rangan, Anna
Roger, Simon D.
Sud, Kamal
Torres, Vicente
Vilayur, Eswari
Rangan, Gopala K.
author_sort Amin, Sneha
collection PubMed
description RATIONALE & OBJECTIVE: The development of new therapies for autosomal dominant polycystic kidney disease requires clinical trials to be conducted efficiently. In this study, the factors affecting the recruitment and retention of participants enrolled in a 3-year randomized controlled trial in autosomal dominant polycystic kidney disease were investigated. STUDY DESIGN: Qualitative study. SETTING & PARTICIPANTS: All participants (N=187) were invited to complete a 16-item questionnaire at the final study visit of the primary trial. Participants were recruited to complete a semistructured interview using purposeful sampling according to age, self-reported gender, and randomization group. ANALYTICAL APPROACH: Descriptive statistics were used for demographic data and questionnaires. The interview transcripts underwent inductive thematic coding. RESULTS: One hundred and forty-six of the 187 randomized participants (79%) completed the post-trial questionnaire, and 31 of the 187 participants (21%) completed the interview. Most participants (94%) rated their global satisfaction with the trial as high (a score of 8 or more out of 10). Altruism, knowledge gain, and access to new treatments were the main motivators for recruitment. The main reasons for considering leaving the study were concerns about the risk of intervention and family or work issues. Strategies that favored retention included flexibility in attending different study sites, schedule flexibility, staff interactions, and practical support with parking and reminders. The main burden was time away from work with lost wages, and burden associated with magnetic resonance imaging scans and 24-hour urine output collections. LIMITATIONS: The study population was restricted to participants in a single nondrug clinical trial, and the results could be influenced by selection and possible social desirability bias. CONCLUSIONS: Participants reported high levels of satisfaction that occurred as a function of the trial meeting participants’ expectations. Furthermore, retention was a balance between the perceived benefits and burden of participation. Consideration of these perspectives in the design of future clinical trials will improve their efficiency and conduct. PLAIN-LANGUAGE SUMMARY: Advances in the clinical practice of autosomal dominant polycystic kidney disease (ADPKD) require affected individuals to voluntarily participate in long-term multicenter randomized controlled trials (RCTs). In this qualitative post hoc study of a 3-year RCT of increased water intake in ADPKD, altruism, knowledge gain, and access to a nondrug treatment positively influenced the decision to volunteer. Ongoing participation was enabled by building flexibility into the study protocol and staff prioritizing a participant’s needs during study visits. Although participants completed the required tests, most were considered burdensome. This study highlights the importance of incorporating protocol flexibility into trial design; the preference for interventions with a low risk of adverse effects; and the urgent requirement for robust surrogate noninvasive biomarkers to enable shorter RCTs in ADPKD.
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spelling pubmed-104327942023-08-18 Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease Amin, Sneha Sangadi, Irene Allman-Farinelli, Margaret Badve, Sunil V. Boudville, Neil Coolican, Helen Coulshed, Susan Foster, Sheryl Fernando, Mangalee Haloob, Imad Harris, David C.H. Hawley, Carmel M. Holt, Jane Howell, Martin Kumar, Karthik Johnson, David W. Lee, Vincent W. Mai, Jun Rangan, Anna Roger, Simon D. Sud, Kamal Torres, Vicente Vilayur, Eswari Rangan, Gopala K. Kidney Med Original Research RATIONALE & OBJECTIVE: The development of new therapies for autosomal dominant polycystic kidney disease requires clinical trials to be conducted efficiently. In this study, the factors affecting the recruitment and retention of participants enrolled in a 3-year randomized controlled trial in autosomal dominant polycystic kidney disease were investigated. STUDY DESIGN: Qualitative study. SETTING & PARTICIPANTS: All participants (N=187) were invited to complete a 16-item questionnaire at the final study visit of the primary trial. Participants were recruited to complete a semistructured interview using purposeful sampling according to age, self-reported gender, and randomization group. ANALYTICAL APPROACH: Descriptive statistics were used for demographic data and questionnaires. The interview transcripts underwent inductive thematic coding. RESULTS: One hundred and forty-six of the 187 randomized participants (79%) completed the post-trial questionnaire, and 31 of the 187 participants (21%) completed the interview. Most participants (94%) rated their global satisfaction with the trial as high (a score of 8 or more out of 10). Altruism, knowledge gain, and access to new treatments were the main motivators for recruitment. The main reasons for considering leaving the study were concerns about the risk of intervention and family or work issues. Strategies that favored retention included flexibility in attending different study sites, schedule flexibility, staff interactions, and practical support with parking and reminders. The main burden was time away from work with lost wages, and burden associated with magnetic resonance imaging scans and 24-hour urine output collections. LIMITATIONS: The study population was restricted to participants in a single nondrug clinical trial, and the results could be influenced by selection and possible social desirability bias. CONCLUSIONS: Participants reported high levels of satisfaction that occurred as a function of the trial meeting participants’ expectations. Furthermore, retention was a balance between the perceived benefits and burden of participation. Consideration of these perspectives in the design of future clinical trials will improve their efficiency and conduct. PLAIN-LANGUAGE SUMMARY: Advances in the clinical practice of autosomal dominant polycystic kidney disease (ADPKD) require affected individuals to voluntarily participate in long-term multicenter randomized controlled trials (RCTs). In this qualitative post hoc study of a 3-year RCT of increased water intake in ADPKD, altruism, knowledge gain, and access to a nondrug treatment positively influenced the decision to volunteer. Ongoing participation was enabled by building flexibility into the study protocol and staff prioritizing a participant’s needs during study visits. Although participants completed the required tests, most were considered burdensome. This study highlights the importance of incorporating protocol flexibility into trial design; the preference for interventions with a low risk of adverse effects; and the urgent requirement for robust surrogate noninvasive biomarkers to enable shorter RCTs in ADPKD. Elsevier 2023-06-29 /pmc/articles/PMC10432794/ /pubmed/37602144 http://dx.doi.org/10.1016/j.xkme.2023.100691 Text en © 2023 The Authors https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Original Research
Amin, Sneha
Sangadi, Irene
Allman-Farinelli, Margaret
Badve, Sunil V.
Boudville, Neil
Coolican, Helen
Coulshed, Susan
Foster, Sheryl
Fernando, Mangalee
Haloob, Imad
Harris, David C.H.
Hawley, Carmel M.
Holt, Jane
Howell, Martin
Kumar, Karthik
Johnson, David W.
Lee, Vincent W.
Mai, Jun
Rangan, Anna
Roger, Simon D.
Sud, Kamal
Torres, Vicente
Vilayur, Eswari
Rangan, Gopala K.
Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease
title Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease
title_full Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease
title_fullStr Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease
title_full_unstemmed Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease
title_short Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease
title_sort participant perceptions in a long-term clinical trial of autosomal dominant polycystic kidney disease
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432794/
https://www.ncbi.nlm.nih.gov/pubmed/37602144
http://dx.doi.org/10.1016/j.xkme.2023.100691
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