Objective: Describe the perspectives of people living with MSA (PwMSA) and their care partners on the design and operational characteristics of a phase 3 trial in MSA.

Background: The value of including the perspectives of people living with a disease in the design, conduct, and dissemination of research that affects them is increasingly recognized. This is especially the case when designing a global phase 3 trial in a rare disease, such as MSA.

Method: 39 interviewees (n=13 PwMSA and n=26 care partners) of whom 19 had participated in a prior phase 2 trial were interviewed to understand their perspectives on trial participation. Interview questions focused on understanding how to improve participant experience in MSA trials, covering the trial burden and other operational characteristics of the trial.

Results: There was consensus amongst interviewees that trial participation represents a significant commitment (time, resources, and emotional) for PwMSA. While the number of visits required to follow disease progression in the phase 2 trial was considered appropriate, respondents frequently expressed a wish for fewer and shorter visits, especially given the often-long distances to travel to trial sites. The phase 3 MASCOT study (NCT06706622) which will assess the efficacy and safety of the alpha-synuclein antibody, amlenetug in MSA was designed considering patient and care partner feedback. For example, the MASCOT protocol includes a rationalization of trial visits, including the combining of Screening and Baseline MRI scans so that participants will not have to undergo two MRI scans within a 3–6-week period. Efforts also have been made to ensure MRIs can be performed in-house or nearby the investigator clinic, and logistics assistance will be provided for participants. Furthermore, trial sites have been chosen based on their experience in treating people with MSA and for broad geographical coverage. Of note, care partner interviewees indicated that they preferred not to use digital diaries, which helped inform how patient (and care partner) reported outcomes will be collected in the phase 3 trial.

Conclusion: Incorporating feedback from PwMSA and their care partners on trial design is aimed to reduce trial burden, make trial participation easier, and improve the overall trial experience for PwMSA and their care partners.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/incorporating-patient-and-care-partner-feedback-on-the-protocol-for-a-clinical-trial-assessing-progression-in-msa/