Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

23 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-01-26

Study Completion Date

2022-08-31

Brief Summary

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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).

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Detailed Description

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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

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Fabry Disease

Study Design

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Observational Model Type

OTHER

Study Time Perspective

OTHER

Interventions

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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.

Intervention Type OTHER

Eligibility Criteria

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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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No