Fabry DiseasePEOPLENCT05186324
Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion
An observational study of Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 12 sites in 5 countries.
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Interview
- Type of treatment
- Injection or infusion
- How many people
- 23 participants
- Where
- 12 sitesin 5 countries
- Study dates
- 26 Jan 2022to 31 Aug 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
12 sites in 5 countries.
12 sites in 5 countries
United States7 sites
#03
Atlanta, Georgia, 30322
#02
Birmingham, Alabama, 35233
#06
Dallas, Texas, 75246
#01
Fairfax, Virginia, 22030
#11
Grand Rapids, Michigan, 49525
#04
Iowa City, Iowa, 52242
#05
Salt Lake City, Utah, 84132
About this study
Official title: 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).
Read the 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).
- Conditions
- Fabry Disease
- Being tested
- Interview
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InterviewOther
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
Symptoms experience while on treatment with pegunigalsidase alfa
Description of the symptoms experienced by patients treated with pegunigalsidase alfa for more than 2 years
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2022
- Study start26 Jan 2022
- Primary completion31 Aug 2022
- Study completion31 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Protalix, IQVIA Pty Ltd
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05186324
- Sponsor's protocol ID
- CLI-06657AA1-05
- Acronym
- PEOPLE
- Status last verified
- Mar 2023
- First submitted
- 21 Dec 2021
- Last updated
- 21 Mar 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion - PEOPLE Study
- ClinicalTrials.gov ID
- NCT05186324
- Acronym / sponsor ID
- PEOPLE / CLI-06657AA1-05
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Collaborators
- Protalix, IQVIA Pty Ltd
- Study start
- 26 Jan 2022 (actual)
- Primary completion
- 31 Aug 2022 (actual)
- Study completion
- 31 Aug 2022 (actual)
- First posted
- 11 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Other
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InterviewDuring 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. | Other | – |
Primary outcome
Symptoms experience while on treatment with pegunigalsidase alfa
Time frame: 2 years
Description of the symptoms experienced by patients treated with pegunigalsidase alfa for more than 2 years
Change in symptoms experienced
Time frame: 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
Time frame: 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
Time frame: 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
Patients' perceptions of the advantages and disadvantages associated with the every 4 weeks infusion schedule
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 12 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, United Kingdom, Belgium, Denmark, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Symptoms experience while on treatment with pegunigalsidase alfa (time frame: 2 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2023.