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The participants' perspective on facioscapulohumeral muscular dystrophy trials in The Netherlands - A qualitative
Lizan Stinissen1, Joost Kools1, Sietse Bouma1
1Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, P.O. Box 9101, 6500 HB, Nijmegen, The Netherlands.
Background:
Facioscapulohumeral 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.
Objective:
To explore the motivation, expectations, concerns, and experiences of FSHD patients in the first clinical trials in the Netherlands resulting in recommendations for future trials.
Methods:
Semi-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.
Results:
Thirteen 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.
Conclusions:
This study presents the participants' perspective on FSHD trials, providing important key findings for future clinical trial design, study site practices and patient education.
Insights
Patients in facioscapulohumeral muscular dystrophy (FSHD) clinical trials were motivated by altruism and health improvement. While generally positive, phase III participants experienced a higher psychological burden, highlighting needs for better communication and updates in future FSHD research.
Area of Science:
- Neurology
- Clinical Trials
- Patient Experience Research
Background:
- Facioscapulohumeral muscular dystrophy (FSHD) is a hereditary muscle disease with no current cure.
- Early-stage clinical trials for disease-modifying therapies, including losmapimod, are underway.
- Understanding patient experiences is crucial for optimizing future FSHD clinical trial design and recruitment.
Purpose of the Study:
- To explore the motivations, expectations, concerns, and experiences of FSHD patients participating in clinical trials.
- To gather recommendations for improving future FSHD clinical trial design and execution.
Main Methods:
- Semi-structured interviews were conducted with 13 participants from Phase II and III losmapimod trials.
- Interviews explored motivation, expectations, trial experience, and recommendations.
- Data were transcribed, anonymized, and analyzed using a deductive approach with Atlas.ti.
Main Results:
- Primary motivations included altruism, scientific contribution, and personal health improvement.
- Participants generally had realistic expectations and a positive trial experience, valuing transparent communication.
- Phase III participants reported a significant psychological burden due to the placebo-controlled nature of the trial.
Conclusions:
- Patient perspectives offer vital insights for enhancing future FSHD clinical trial design.
- Findings can inform best practices for study sites and patient education initiatives.
- Improved communication and frequent updates are recommended for future trial participants.
