NCT05186324

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion - PEOPLE Study

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 23 (actual)
• Sponsor: Chiesi Farmaceutici S.p.A. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Pegunigalsidase alfa (PRX-102) is a long-term enzyme replacement therapy design for the treatment of patients with Fabry disease. Although in the clinical development program patient-reported outcomes and clinician-reported outcomes have been included, this may not allow for a sufficiently accurate assessment of the quality of life in patients with Fabry Disease treated with pegunigalsidase alfa. This study will collect the patient experience on the pegunigalsidase alfa treatment administered intravenously every 4 weeks in the BRIGHT-F51 clinical study (NCT03614234).

Detailed description

This is an additional qualitative concept elicitation interview-based study to further understand the patients' experience with Fabry disease and with the pegunigalsidase alfa administered intravenously every 4 weeks. Patients will be asked a set of open-ended questions with probes to describe their experiences with Fabry disease on treatment with pegunigalsidase alfa. Qualitative research methods will be used to obtain a deeper understanding of the patient experience by generating in-depth information about the experiences, perspectives, and feelings of patients and others, in their own words (FDA Patient-Focused Drug Development Guidance 2). The study will be offered to the 29 patients participating in the BRIGHT-F51 clinical trial (NCT03614234).

Conditions

• Fabry Disease

Interventions

Type	Name	Description
OTHER	Interview	During each interview, patients will be asked questions to collect demographic and clinical information, and asked a set of open-ended questions with probes to describe their experiences with Fabry disease (symptomology and impacts on patient's lives \[i.e., activities of daily living, school/work, ability to take holidays/vacation\]), and pegunigalsidase alfa treatment (experience of infusions and schedule) and their experience of change in symptoms and impacts over the BRIGHT-F51 clinical study. A semi-structured discussion guide will be used to conduct the approximately 60-minute interviews. The use of open-ended questions avoids bias and questions will not be read verbatim to allow for a free-flowing discussion.

Timeline

• Start date: 2022-01-26
• Primary completion: 2022-08-31
• Completion: 2022-08-31
• First posted: 2022-01-11
• Last updated: 2023-03-21

Locations

12 sites across 5 countries: United States, Belgium, Denmark, Italy, United Kingdom