Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

NCT05186324 | Completed

• 1 other identifier: CLI-06657AA1-05
• Study Type: observational
• Target: 23
• Locations: 5 countries
• Sites: 12

Brief 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

Fabry Disease

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (1)

• Perception of change in symptoms and impacts with infusion schedules

  2 years

Interventions

Interview OTHER

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chiesi Farmaceutici S.p.A.: lead
• Protalix: collaborator
• Iqvia Pty Ltd: collaborator

Study Sites (12)

#02

Birmingham, Alabama, 35233, United States

Location

#03

Atlanta, Georgia, 30322, United States

Location

#04

Iowa City, Iowa, 52242, United States

Location

#11

Grand Rapids, Michigan, 49525, United States

Location

#06

Dallas, Texas, 75246, United States

Location

#05

Salt Lake City, Utah, 84132, United States

Location

#01

Fairfax, Virginia, 22030, United States

Location

#22

Antwerp, 2650, Belgium

Location

#50

Copenhagen, 2100, Denmark

Location

#56

Napoli, 80131, Italy

Location

#28

Cambridge, CB2 2QQ, United Kingdom

Location

#07

London, NW3 2QG, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

Interviews as Topic

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2021
• First Posted: January 11, 2022
• Study Start: January 26, 2022
• Primary Completion: August 31, 2022
• Study Completion: August 31, 2022
• Last Updated: March 21, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1); US (7); GB (2); IT (1); BE (1)