Clinical trial NCT05186324: Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion
Official title: Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion - PEOPLE Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Fabry Disease
Lead sponsor: Chiesi Farmaceutici S.p.A.
Sponsor class: INDUSTRY
Collaborators: name: Protalix
name: Iqvia Pty Ltd
First posted: 2022-01-11
Enrollment: 23
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
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.

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Symptoms experience while on treatment with pegunigalsidase alfa
timeFrame: 2 years
measure: Change in symptoms experienced
timeFrame: 2 years
measure: Impacts of Fabry disease on patient's life
timeFrame: 2 years
measure: Change in the ability to perform daily activities
timeFrame: 2 years
measure: Patients' perceptions of the advantages and disadvantages associated with the every 4 weeks infusion schedule
timeFrame: 2 years
Secondary outcomes:
Other outcomes:
measure: Perception of change in symptoms and impacts with infusion schedules
timeFrame: 2 years
Locations:
city: Birmingham
country: United States
facility: #02
state: Alabama
city: Atlanta
country: United States
facility: #03
state: Georgia
city: Iowa City
country: United States
facility: #04
state: Iowa
city: Grand Rapids
country: United States
facility: #11
state: Michigan
city: Dallas
country: United States
facility: #06
state: Texas
city: Salt Lake City
country: United States
facility: #05
state: Utah
city: Fairfax
country: United States
facility: #01
state: Virginia
city: Antwerp
country: Belgium
facility: #22
city: Copenhagen
country: Denmark
facility: #50
city: Napoli
country: Italy
facility: #56
city: Cambridge
country: United Kingdom
facility: #28
city: London
country: United Kingdom
facility: #07
Study officials:
References: