Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion (PEOPLE)

Chiesi

Status: Completed

Conditions: Fabry Disease

Treatments: Other: Interview

Study type: Observational

Funder types: Industry

Identifiers

NCT05186324

CLI-06657AA1-05

Details and patient eligibility

About

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).

Full 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).

Enrollment: 23 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• The patient is participating in study PB-102-F51
• The patient is willing and able to participate in a 60-minute recorded interview
• The patient is able to read, understand, and speak sufficiently to participate in the interviews
• The patient signs informed consent to participate in the study

Exclusion criteria

• At investigators discretion, patient is considered to be unable to participate in a 60- minute telephone interview.
• Patient has any clinically relevant medical or psychiatric condition that, in the opinion of the investigator would interfere with the completion of the study activities. This includes but is not limited to language, speech, hearing or cognitive disorders that could impact a patient's ability to participate in an interview-based discussion.