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Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

NCT05186324 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 23

Last updated 2023-03-21

No results posted yet for this study

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).

Conditions

Interventions

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.

Sponsors & Collaborators

  • Protalix

    collaborator INDUSTRY

  • Iqvia Pty Ltd

    collaborator INDUSTRY

  • Chiesi Farmaceutici S.p.A.

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-26
Primary Completion
2022-08-31
Completion
2022-08-31

Countries

  • United States
  • Belgium
  • Denmark
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05186324 on ClinicalTrials.gov