Fabry DiseasePhase 3NCT03566017
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
A Phase 3 study testing pegunigalsidase alfa in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 30 sites in 13 countries.
Trial summary
The objective of CLI-06657AA1-04 (formerly PB-102-F60) was to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who had successfully completed studies PB-102-F20, PB-102-F30, or at least 48 months in… Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- pegunigalsidase alfa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 97 participants
- Where
- 30 sitesin 13 countries
- Study dates
- 16 Sep 2018to 21 Jan 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Completion of study PB-102-F20, study PB-102-F30, or at least 48 months in study PB-102-F03.
- The participant signed informed consent.
- Female participants and male participants whose co-partners were of child-bearing potential agreed to use a medically acceptable method of contraception. These included combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or sexual abstinence. Contraception had to be used for 2 weeks after treatment termination.
Who cannot join
- Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.
Trial locations and contacts
30 sites in 13 countries.
30 sites in 13 countries
United States15 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
UAB Medicine
Birmingham, Alabama, 35233
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Renal Disease Research Institute, LLC - Dallas
Dallas, Texas, 75235
Institute of Metabolic Disease
Dallas, Texas, 75246
O+O Alpan LLC
Fairfax, Virginia, 22030
University of Florida
Gainesville, Florida, 32610
Infusion Associates
Grand Rapids, Michigan, 49525
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of California San Diego
La Jolla, California, 92037
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of California Irvine Center
Orange, California, 92868
Phoenix Children's Hospital
Phoenix, Arizona, 85016
University of Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of Utah Hospitals & Clinics
Salt Lake City, Utah, 84108
United Kingdom4 sites
Addenbrooke's Hospital
Cambridge, CB2 0QQ
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
Edgbaston, Birmingham, B152TH
The Royal Free Hospital
London, NW3 2QG
Salford Royal
Salford, Greater Manchester, M6 8HD
Czechia1 site
Vseobecna fakultni nemocnice v Praze
Prague, Czech Republic, 12808
Italy1 site
Azienda Ospedaliera Universitaria "Federico II"
Naples, Via Pansini, 80131
About this study
Official title: Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
The objective of CLI-06657AA1-04 (formerly PB-102-F60) was to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who had successfully completed studies PB-102-F20, PB-102-F30, or at least 48 months in study PB-102-F03.
Read the detailed description
This was an open-label study. Participants were enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment was until pegunigalsidase alfa was commercially available to the participant, or at the discretion of the Sponsor. Efficacy and safety analyses were summarized using descriptive statistics for continuous variables and counts and percentages for categorical variables. Data from the parent studies were also included in the analyses.
- Conditions
- Fabry Disease
- Being tested
- pegunigalsidase alfa
- Keywords
- Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease
- Medical subject headings
- Fabry Disease, Proteinuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pegunigalsidase alfaDrug
Also called PRX-102
Recombinant human alpha galactosidase A
| Study group | Role | What they receive |
|---|---|---|
| Experimental open label | Experimental | pegunigalsidase alfa Receives: Drug: pegunigalsidase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-related Adverse Events
No primary or secondary endpoints were specified for this trial. Evaluation of safety was a main objective.
A treatment-emergent adverse event (TEAE) was defined as any adverse event (AE) occurring after the start of study treatment and within the time of residual drug effect (20 days after last administration of study treatment) or a pre-treatment AE or medical condition that worsened in severity after the start of study treatment and within the time of residual drug effect. Each TEAE was classified for severity based on the Common Terminology Criteria for Adverse Events (CTCAE) v4.03 and for causality in the categories unrelated, unlikely, possible, probable and definitely. Treatment-related AEs were TEAEs with causality assessed as possible, probable or definitely related to study treatment. TEAEs were coded using the Medical Dictionary for Regulatory Activities (MedDRA) version (v)19.0. Related TEAEs reported in ≥2 percent of participants by Preferred Term are reported.
Measured: From first ever infusion of pegunigalsidase alfa, which could have been in the parent study (PB-102-F01/F20/F30) or this extension study, CLI-06657AA1-04, until 90 days after the final dose visit for each participant. Mean individual exposure: 5.5 years
Secondary outcomes (17)
Change From Baseline to the Last Observation for the eGFR
The eGFR, calculated based on serum creatinine values, according to the Chronic Kidney Disease - Epidemiology Collaboration (CKD-EPI) formula (2009).
Measured: From baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Annualized eGFR Slope
The annualized eGFR slope was added in the statistical analysis plan. Individual eGFR slopes were derived for each participant using a linear regression model and summarized descriptively.
Measured: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or this extension study, CLI-06657AA1-04), until the end of the extension study; mean individual exposure: 5.5 years
Shift From Baseline to the Last Scheduled Visit in UPCR Category
The UPCR was assessed by spot urine test. Participants were categorized according to UPCR at baseline and subsequent visits.
Measured: Shift from baseline (assessment before first exposure to study drug which could be in the parent study PB-102-F01/F02/F30 or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation in Plasma Lyso-Gb3 and Globotriaosylceramide (Gb3) Concentrations
Plasma concentrations of Lyso-Gb3 and Gb3 (biomarkers for Fabry disease) were assessed at baseline and subsequent time points and change from baseline was assessed.
Measured: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04 ) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation for Pain Severity Items of the Brief Pain Inventory (BPI)
The short form Brief Pain Inventory (BPI) consists of 4 pain severity items (pain at its worst in the last 24 hours, pain at its least in the last 24 hours, pain on average and current pain) and 7 pain interference items (general activity, mood, walking ability, normal work, relations with other people, sleep and enjoyment of life) assessed over a 24-hour recall period. Each item is rated on a scale from 0 (no pain/interference) to 10 (worst pain imaginable/complete interference). Participants report percentage of pain relief by analgesics over the past 24 hours as well. Assessment of pain (by the BPI) was performed at baseline and subsequent time points.
Measured: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation in Overall Score for the Mainz Severity Score Index (MSSI)
The MSSI represents patients with Fabry disease. Overall scores of <20, ≥20 and ≤40 and >40 indicate that patients are mildly, moderately or severely affected, respectively. An increase in the score indicates increased severity of the disease. Symptom assessment by the MSSI was performed at baseline and at subsequent time points.
The ranges for the MSSI general, neurological, cardiovascular and renal dysfunction domain scores are 0 to 18, 0 to 20, 0 to 20 and 0 to 18 respectively. The overall score considered for the study is the sum of the domain scores - min 0 max 76
Measured: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change in Use of Pain Medication During Treatment
At baseline, participants were categorized by the number of pain medications used (0, 1 and 2+ pain medications). Shifts from baseline to 72 months in the number of participants using pain medications (categorized by number of pain medications used) are presented.
Measured: Shift from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to 72 months, in number of participants using pain medications (categorized by 0, 1 and 2+ pain medications)
Change From Baseline to the Last Observation in the EuroQoL Visual Analog Scale (EQ VAS) Score
Quality of life was assessed by administering the EuroQoL-5 Dimensions-5 Levels (EQ-5D-5L) questionnaire at baseline and subsequent time points. The assessment includes the EuroQoL-5 Dimensions (EQ-5D) descriptive system and the EQ VAS. For the EQ VAS, participants rated their current health on a vertical VAS which has endpoints labelled 'the best health you can imagine' (score of 100) and 'the worst health you can imagine' (score of 0). An increase in score indicates improvement.
Measured: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation in Left Ventricular Mass Index
Cardiac assessment was performed by cardiac magnetic resonance imaging (MRI) at baseline and subsequent time points and various parameters including the left ventricular mass index (LVMI) (g/m^2) with and without hypertrophy were assessed.
Measured: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change in Infusion Premedication Use From Baseline to 12 Months
Infusion premedication use was not collected in studies PB-102-F01/F02/F03. Changes in use of infusion premedication were analyzed in a pooled sample of participants enrolled from studies PB-102-F20 and PB-102-F30. Participants were categorized at baseline by number of infusion premedications used (0, 1, 2 and 3 or more) and shifts from baseline to 12 months in the number of participants using infusion premedications, categorized by number of infusion premedications used, are presented.
Measured: Baseline (assessment before fist exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or this extension study, CLI-06657AA1-04) to 12 months
Change in Infusion Premedication Use From Baseline to 24 Months
Infusion premedication use was not collected in studies PB-102-F01/F02/F03. Changes in use of infusion premedication were analyzed in a pooled sample of participants enrolled from studies PB-102-F20 and PB-102-F30. Participants were categorized at baseline by number of infusion premedications used (0, 1, 2 and 3 or more) and shifts from baseline to 24 months in the number of participants using infusion premedications, categorized by number of infusion premedications used, are presented.
Measured: Baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30 or this extension study, CLI-06657AA1-04) to 24 months
Change in Infusion Premedication Use From Baseline to 48 Months
Infusion premedication use was not collected in studies PB-102-F01/F02/F03. Changes in use of infusion premedication were analyzed in a pooled sample of participants enrolled from studies PB-102-F20 and PB-102-F30. Participants were categorized at baseline by number of infusion premedications used (0, 1, 2 and 3 or more) and shifts from baseline to 48 months in the number of participants using infusion premedications, categorized by number of infusion premedications used, are presented.
Measured: Baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30 or this extension study, CLI-06657AA1-04) to 48 months
Change in Infusion Premedication Use From Baseline to 72 Months
Infusion premedication use was not collected in studies PB-102-F01/F02/F03. Changes in use of infusion premedication were analyzed in a pooled sample of participants enrolled from studies PB-102-F20 and PB-102-F30. Participants were categorized at baseline by number of infusion premedications used (0, 1, 2 and 3 or more) and shifts from baseline to 72 months in the number of participants using infusion premedications, categorized by number of infusion premedications used, are presented.
Measured: Baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30 or this extension study, CLI-06657AA1-04) to 72 months
Number of Participants Who Had ADA at Any Post-baseline Visit
The ADA status was assessed as per the schedule of assessments based on sequential evaluation. The participant was considered ADA-positive if IgG screening was presumptive positive and subsequent IgG immunodepletion was positive.
Measured: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Number of Participants With Treatment-emergent ADA Who Had Titer-boosted or Treatment-induced ADA
The ADA status at a visit was decided based on a sequential evaluation as per the schedule of assessments. If the IgG screening was negative, the participant was ADA-negative at that visit. If the IgG screening was presumptive positive, an IgG immunodepletion test was performed and the participant was considered ADA-positive or -negative at the visit based on whether the test was positive or negative. Baseline was the last assessment before the first infusion of pegunigalsidase alfa. Participants were considered to be treatment-emergent ADA-positive if they had titer-boosted or treatment-induced ADA. The ADA were titer-boosted if participants were ADA-positive at baseline and ADA titer had increased by at least 4-fold from baseline during treatment. The ADA were treatment-induced if participants were ADA-negative at baseline and ADA-positive in at least one timepoint post-first infusion.
Measured: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Infusion-related Reactions (IRRs) Occurring During the Infusion or Within 2 Hours After Its Completion (IRR-2H) in More Than One Participant Overall by MedDRA Preferred Term (PT)
An IRR was defined as a reaction to the infusion of pharmacological and/or biological substances; symptoms could appear within minutes to 2 hours following the end of the infusion and could include pruritus, flushing, swelling, dyspnea, bronchospasm and hypotension. IRRs could be evaluated up to 24 hours from occurrence. Two time frames were considered for IRR analysis: IRR-2H and IRRs occurring during the infusion or within 24 hours after its completion (IRR-24H). All IRR-2H were also classified as IRR-24H. The IRR-2H reported for more than one participant overall by MedDRA PT are presented.
Measured: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Infusion-related Reactions Occurring During the Infusion or Within 24H After Its Completion (IRR-24H) in More Than One Participant Overall by MedDRA PT
An IRR was defined as a reaction to the infusion of pharmacological and/or biological substances; symptoms could appear within minutes to 2 hours following the end of the infusion and could include pruritus, flushing, swelling, dyspnea, bronchospasm and hypotension. IRRs could be evaluated up to 24 hours from occurrence. Two time frames were considered for IRR analysis: IRR-2H and IRR-24H. All IRR-2H were also classified as IRR-24H. IRR-24H reported for more than one participant overall by MedDRA PT are presented.
Measured: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2018
- Study start16 Sep 2018
- Primary completion21 Jan 2025
- Study completion21 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label extension study
- Masking
- None (open label)
- Enrolment
- 97 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03566017
- Sponsor's protocol ID
- CLI-06657AA1-04
- Status last verified
- Mar 2026
- First submitted
- 24 May 2018
- Last updated
- 24 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT03566017
- Acronym / sponsor ID
- CLI-06657AA1-04
- Conditions
- Fabry Disease
- Keywords
- Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 97 participants (actual)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Study start
- 16 Sep 2018 (actual)
- Primary completion
- 21 Jan 2025 (actual)
- Study completion
- 21 Jan 2025 (actual)
- First posted
- 21 Jun 2018 (actual)
- Results
- Posted 24 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of CLI-06657AA1-04 (formerly PB-102-F60) was to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who had successfully completed studies PB-102-F20, PB-102-F30, or at least 48 months in study PB-102-F03.
Detailed description
This was an open-label study. Participants were enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment was until pegunigalsidase alfa was commercially available to the participant, or at the discretion of the Sponsor. Efficacy and safety analyses were summarized using descriptive statistics for continuous variables and counts and percentages for categorical variables. Data from the parent studies were also included in the analyses.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label extension study
| Arm | Type | Receives |
|---|---|---|
| Experimental open label | Experimental | Drug: pegunigalsidase alfapegunigalsidase alfa |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Completion of study PB-102-F20, study PB-102-F30, or at least 48 months in study PB-102-F03.
- The participant signed informed consent.
- Female participants and male participants whose co-partners were of child-bearing potential agreed to use a medically acceptable method of contraception. These included combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or sexual abstinence. Contraception had to be used for 2 weeks after treatment termination.
Key exclusion criteria (1 listed)
- Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.
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 |
|---|---|---|
| pegunigalsidase alfaAlso: PRX-102Recombinant human alpha galactosidase A | Drug | Experimental open label |
Primary outcome
Number of Participants With Treatment-related Adverse Events
Time frame: From first ever infusion of pegunigalsidase alfa, which could have been in the parent study (PB-102-F01/F20/F30) or this extension study, CLI-06657AA1-04, until 90 days after the final dose visit for each participant. Mean individual exposure: 5.5 years
No primary or secondary endpoints were specified for this trial. Evaluation of safety was a main objective. A treatment-emergent adverse event (TEAE) was defined as any adverse event (AE) occurring after the start of study treatment and within the time of residual drug effect (20 days after last administration of study treatment) or a pre-treatment AE or medical condition that worsened in severity after the start of study treatment and within the time of residual drug effect. Each TEAE was classified for severity based on the Common Terminology Criteria for Adverse Events (CTCAE) v4.03 and for causality in the categories unrelated, unlikely, possible, probable and definitely. Treatment-related AEs were TEAEs with causality assessed as possible, probable or definitely related to study treatment. TEAEs were coded using the Medical Dictionary for Regulatory Activities (MedDRA) version (v)19.0. Related TEAEs reported in ≥2 percent of participants by Preferred Term are reported.
17 secondary outcomes
Change From Baseline to the Last Observation for the eGFR
Time frame: From baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Annualized eGFR Slope
Time frame: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or this extension study, CLI-06657AA1-04), until the end of the extension study; mean individual exposure: 5.5 years
Shift From Baseline to the Last Scheduled Visit in UPCR Category
Time frame: Shift from baseline (assessment before first exposure to study drug which could be in the parent study PB-102-F01/F02/F30 or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation in Plasma Lyso-Gb3 and Globotriaosylceramide (Gb3) Concentrations
Time frame: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04 ) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation for Pain Severity Items of the Brief Pain Inventory (BPI)
Time frame: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation in Overall Score for the Mainz Severity Score Index (MSSI)
Time frame: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change in Use of Pain Medication During Treatment
Time frame: Shift from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to 72 months, in number of participants using pain medications (categorized by 0, 1 and 2+ pain medications)
Change From Baseline to the Last Observation in the EuroQoL Visual Analog Scale (EQ VAS) Score
Time frame: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change From Baseline to the Last Observation in Left Ventricular Mass Index
Time frame: Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)
Change in Infusion Premedication Use From Baseline to 12 Months
Time frame: Baseline (assessment before fist exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or this extension study, CLI-06657AA1-04) to 12 months
Change in Infusion Premedication Use From Baseline to 24 Months
Time frame: Baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30 or this extension study, CLI-06657AA1-04) to 24 months
Change in Infusion Premedication Use From Baseline to 48 Months
Time frame: Baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30 or this extension study, CLI-06657AA1-04) to 48 months
Change in Infusion Premedication Use From Baseline to 72 Months
Time frame: Baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30 or this extension study, CLI-06657AA1-04) to 72 months
Number of Participants Who Had ADA at Any Post-baseline Visit
Time frame: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Number of Participants With Treatment-emergent ADA Who Had Titer-boosted or Treatment-induced ADA
Time frame: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Infusion-related Reactions (IRRs) Occurring During the Infusion or Within 2 Hours After Its Completion (IRR-2H) in More Than One Participant Overall by MedDRA Preferred Term (PT)
Time frame: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Infusion-related Reactions Occurring During the Infusion or Within 24H After Its Completion (IRR-24H) in More Than One Participant Overall by MedDRA PT
Time frame: From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or in this extension study, CLI-06657AA1-04), until the end of the study. Mean individual exposure was 5.5 years.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
30 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing pegunigalsidase alfa in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 30 sites in 13 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 3 inclusion and 1 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 24 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, United Kingdom, Australia, Canada, Czechia and 8 more countries. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Treatment-related Adverse Events (time frame: From first ever infusion of pegunigalsidase alfa, which could have been in the parent study (PB-102-F01/F20/F30) or this extension study, CLI-06657AA1-04, until 90 days after the final dose visit for each participant. Mean individual exposure: 5.5 years). 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 24 Mar 2026.