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

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

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).

Study Overview

• Status: Completed
• Conditions: Fabry Disease
• Intervention / Treatment: Other: Interview

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).

• Study Type: Observational
• Enrollment (Actual): 23

Contacts and Locations

Study Locations

• Belgium
  • Antwerp, Belgium, 2650
    • #22
• Denmark
  • Copenhagen, Denmark, 2100
    • #50
• Italy
  • Napoli, Italy, 80131
    • #56
• United Kingdom
  • Cambridge, United Kingdom, CB2 2QQ
    • #28
  • London, United Kingdom, NW3 2QG
    • #07
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • #02
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • #03
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • #04
  • Michigan
    • Grand Rapids, Michigan, United States, 49525
      • #11
  • Texas
    • Dallas, Texas, United States, 75246
      • #06
  • Utah
    • Salt Lake City, Utah, United States, 84132
      • #05
  • Virginia
    • Fairfax, Virginia, United States, 22030
      • #01

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

The PEOPLE study will recruit patients who have taken part in the BRIGHT (F50) clinical trial and are participating in the BRIGHT-F51 extension clinical trial (regardless of their infusion schedule and dosing regimen). Based on the total number of patients (n=29) enrolled in the BRIGHT-F51 clinical trial extension, up to 29 patients could be recruited.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Symptoms experience while on treatment with pegunigalsidase alfa	Description of the symptoms experienced by patients treated with pegunigalsidase alfa for more than 2 years	2 years
Change in symptoms experienced	Description of any worsening or relapse in Fabry disease symptoms during the 4 weeks between two consecutive infusions of pegunigalsidase alfa administered every 4 weeks in patients treated for more than 2 years	2 years
Impacts of Fabry disease on patient's life	Description of the impacts of Fabry disease on patient's lives i.e., activities of daily living, school/work, ability to take holidays/vacation) in patients treated with pegunigalsidase alfa for more than 2 years	2 years
Change in the ability to perform daily activities	Description of any worsening or relapse in the ability to perform daily activities during the 4 weeks between two consecutive infusions of pegunigalsidase alfa in patients treated every 4 weeks for more than 2 years	2 years
Patients' perceptions of the advantages and disadvantages associated with the every 4 weeks infusion schedule	Summary of patients' perceptions of the advantages and disadvantages associated with the every 4 weeks infusion schedule (compared to the 2-week infusion schedule) in patients treated with pegunigalsidase alfa for more than 2 years	2 years

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Perception of change in symptoms and impacts with infusion schedules	Description of patients' perception of change in symptoms and impacts with the with the every 4 weeks infusion schedule compared to the 2-week infusion schedule in patients treated with pegunigalsidase alfa for more than 2 years	2 years

Collaborators and Investigators

• Sponsor: Chiesi Farmaceutici S.p.A.
• Collaborators: IQVIA Pty Ltd; Protalix

Study record dates

Study Major Dates

• Study Start (Actual): January 26, 2022
• Primary Completion (Actual): August 31, 2022
• Study Completion (Actual): August 31, 2022

Study Registration Dates

• First Submitted: December 21, 2021
• First Submitted That Met QC Criteria: January 10, 2022
• First Posted (Actual): January 11, 2022

Study Record Updates

• Last Update Posted (Actual): March 21, 2023
• Last Update Submitted That Met QC Criteria: March 20, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Metabolic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Fabry Disease
• Other Study ID Numbers: CLI-06657AA1-05

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No