ArticleJournal of neuromuscular diseases2025
The participants' perspective on facioscapulohumeral muscular dystrophy trials in The Netherlands - A qualitative study.
Article in Journal of neuromuscular diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- An international patient survey of RYR1-related diseases: The patient perspective on clinical trial participation.Journal of neuromuscular diseases · 2026Article
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundFacioscapulohumeral muscular dystrophy (FSHD) is a hereditary muscle disease without an available cure. The first trials with potentially disease-modifying therapies have started, including a phase ll open-label study and a phase lll double-blind randomized placebo-controlled trial assessing the safety and efficacy of losmapimod. Having a more in-depth understanding of the patient's experience of these trials will further enhance the design and recruitment of future trials.
objectiveTo explore the motivation, expectations, concerns, and experiences of FSHD patients in the first clinical trials in the Netherlands resulting in recommendations for future trials.
methodsSemi-structured interviews with participants of phase II and III losmapimod trials were conducted. The interview guide was based on previous conducted literature reviews and consultation of a patient representative. Participants were selected through convenience sampling. Four main themes were discussed: motivation for participation, expectations regarding study drug and trial visits, trial participation experience, and recommendations for future trials. The interviews were transcribed, anonymized, and analyzed using Atlas.ti version 23.1.1 using a deductive approach.
resultsThirteen participants were interviewed; six phase II participants and seven phase III participants. The primary motivations to participate concerned altruistic motives, contribute to science or improve their own health status. The participants had realistic expectations of the effect of the study drug before trial participation. Overall, participants were positive about their trial participation. Specifically, the personal and transparent communication within a trusting and dedicated trial team was appreciated. The phase III participants reported a higher than expected psychological burden on participating in a placebo-controlled trial. Recommendations consisted of more frequent updates on the overall progress and results of the trials.
conclusionsThis study presents the participants' perspective on FSHD trials, providing important key findings for future clinical trial design, study site practices and patient education.
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Registered trials
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