Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

Fabry Disease Clinical Trial

— PEOPLE  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05186324
• Other study ID #: CLI-06657AA1-05
• Status: Completed
• Start date: January 26, 2022
• Est. completion date: August 31, 2022

Study information

• Verified date: March 2023
• Source: Chiesi Farmaceutici S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 23
• Est. completion date: August 31, 2022
• Est. primary completion date: August 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

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.

Locations

Country	Name	City	State
Belgium	#22	Antwerp
Denmark	#50	Copenhagen
Italy	#56	Napoli
United Kingdom	#28	Cambridge
United Kingdom	#07	London
United States	#03	Atlanta	Georgia
United States	#02	Birmingham	Alabama
United States	#06	Dallas	Texas
United States	#01	Fairfax	Virginia
United States	#11	Grand Rapids	Michigan
United States	#04	Iowa City	Iowa
United States	#05	Salt Lake City	Utah

Sponsors (3)

Lead Sponsor	Collaborator
Chiesi Farmaceutici S.p.A.	Iqvia Pty Ltd, Protalix

Countries where clinical trial is conducted

United States,  Belgium,  Denmark,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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