Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

Completed

• Conditions: Fabry Disease

• Registration Number: NCT05186324
• Lead Sponsor: Chiesi Farmaceutici S.p.A.

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).

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 Timeline

• Study First Submitted: 2021-12-21
• Study First Submitted QC: 2022-01-10
• Study First Posted: 2022-01-11
• Study Start: 2022-01-26
• Primary Completion: 2022-08-31
• Study Completion: 2022-08-31
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-20
• Last Update Posted: 2023-03-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in symptoms experienced	2 years	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
Impacts of Fabry disease on patient's life	2 years	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
Change in the ability to perform daily activities	2 years	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
Symptoms experience while on treatment with pegunigalsidase alfa	2 years	Description of the symptoms experienced by patients treated with pegunigalsidase alfa for more than 2 years
Patients' perceptions of the advantages and disadvantages associated with the every 4 weeks infusion schedule	2 years	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

Trial Locations

Locations (12)

#06; 🇺🇸 Dallas, Texas, United States

#22; 🇧🇪 Antwerp, Belgium

#03; 🇺🇸 Atlanta, Georgia, United States

#04; 🇺🇸 Iowa City, Iowa, United States

#11; 🇺🇸 Grand Rapids, Michigan, United States

#01; 🇺🇸 Fairfax, Virginia, United States

#50; 🇩🇰 Copenhagen, Denmark

#56; 🇮🇹 Napoli, Italy

#28; 🇬🇧 Cambridge, United Kingdom

#07; 🇬🇧 London, United Kingdom

#02; 🇺🇸 Birmingham, Alabama, United States

#05; 🇺🇸 Salt Lake City, Utah, United States