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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental open label | Experimental | pegunigalsidase alfa |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| pegunigalsidase alfa | Drug | Recombinant human alpha galactosidase A |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 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) |
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Inclusion Criteria:
Exclusion Criteria:
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.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UAB Medicine | Birmingham | Alabama | 35233 | United States | ||
| Phoenix Children's Hospital |
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Study CLI-06657AA1-04 was an extension study of studies PB-102-F03, PB-102-F20 and PB-102-F30. Participants who completed studies PB-102-F20 or PB-102-F30 or at least 48 months in study PB-102-F03 and opted to enroll in the extension study were included.
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| ID | Title | Description |
|---|---|---|
| FG000 | Experimental Open Label | pegunigalsidase alfa pegunigalsidase alfa: Recombinant human alpha galactosidase A |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 25, 2022 | Jan 28, 2026 |
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Open-label extension study
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| 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. | 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. | 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. | 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. | 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 | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. |
| Phoenix |
| Arizona |
| 85016 |
| United States |
| University of California San Diego | La Jolla | California | 92037 | United States |
| University of California Irvine Center | Orange | California | 92868 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| Emory University School of Medicine | Atlanta | Georgia | 30322 | United States |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| Infusion Associates | Grand Rapids | Michigan | 49525 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Renal Disease Research Institute, LLC - Dallas | Dallas | Texas | 75235 | United States |
| Institute of Metabolic Disease | Dallas | Texas | 75246 | United States |
| University of Utah Hospitals & Clinics | Salt Lake City | Utah | 84108 | United States |
| O+O Alpan LLC | Fairfax | Virginia | 22030 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Royal Melbourne Hospital | Parkville | Victoria | 3050 | Australia |
| Capital District Health Authority | Halifax | Nova Scotia | B3H 1V8 | Canada |
| Vseobecna fakultni nemocnice v Praze | Prague | Czech Republic | 12808 | Czechia |
| Turku University Central Hospital | Turku | FI-20521 | Finland |
| Hospital Raymond-Poincaré | Garches | 92380 | France |
| Semmelweis Egyetem | Budapest | 1083 | Hungary |
| Azienda Ospedaliera Universitaria "Federico II" | Naples | Via Pansini | 80131 | Italy |
| Academisch Medisch Centrum | Amsterdam | 1105 AZ | Netherlands |
| Haukeland University Hospital Klinisk Forskningspost | Bergen | 5021 | Norway |
| General Hospital Slovenj Gradec | Slovenj Gradec | 2380 | Slovenia |
| Hospital de Dia Quiron Zaragoza | Zaragoza | 50012 | Spain |
| University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital | Edgbaston | Birmingham | B152TH | United Kingdom |
| Salford Royal | Salford | Greater Manchester | M6 8HD | United Kingdom |
| Addenbrooke's Hospital | Cambridge | CB2 0QQ | United Kingdom |
| The Royal Free Hospital | London | NW3 2QG | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Experimental Open Label | pegunigalsidase alfa pegunigalsidase alfa: Recombinant human alpha galactosidase A |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Fabry disease classification | Count of Participants | Participants |
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| Estimated glomerular filtration rate (eGFR) | Mean | Standard Deviation | mL/min/1.73m^2 |
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| Plasma globotriaosylsphingosine (Lyso-Gb3) concentration | Participants in Cohort F03 (those enrolled from study PB-102-F03) were excluded as the plasma Lyso-Gb3 was assessed in different laboratories in studies PB-102-F01/F02/F03 and in studies PB-102-F20, PB-102-F30 and CLI-06657AA1-04. Baseline data were available for 86/87 participants in the Intention-to-Treat (ITT) Population excluding Cohort F03. | Mean | Standard Deviation | nM |
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| Status of immunoglobulin G (IgG) anti-drug antibodies to pegunigalsidase alfa (ADA) | The ADA status was assessed based on sequential evaluation. The participant was considered ADA-positive if IgG screening was presumptive positive and subsequent IgG immunodepletion was positive. The participant was considered ADA-negative if the IgG screening was negative, or if the IgG screening was presumptive positive and subsequent IgG immunodepletion was negative. | Count of Participants | Participants |
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| Urine protein to creatinine ratio (UPCR) > 0.5 g/g | Count of Participants | Participants |
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| Previous treatment with enzyme replacement therapy | Count of Participants | Participants |
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| 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. 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 | Mean | Standard Deviation | Score on a scale |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
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| Primary | 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. | Safety Population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose (including a partial dose) of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Count of Participants | Participants | 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 |
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| Secondary | 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). | Intent-to-treat (ITT) Population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose, including a partial dose of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Mean | Standard Deviation | mL/min/1.73 m^2 | 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) |
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| Secondary | 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. | The ITT population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose, including a partial dose of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Mean | Standard Deviation | mL/min/1.73 m^2/year | 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 |
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| Secondary | 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. | Shift table was generated. The number of participants in the ITT Population per UPCR category at baseline is presented by row with shifts in UPCR category at the last scheduled visit for each row. | Posted | Count of Participants | Participants | 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) |
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| Secondary | 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. | Plasma Lyso-Gb3 was assessed in different laboratories in studies PB-102-F01/F02/F03 and studies PB-102-F20/F30 and CLI-06657AA1-04. Plasma Gb3 was assessed in a different unit in studies PB-102-F01/F02/F03 and and studies PB-102-F20/F30 and CLI-06657AA1-04 and conversion between the units was not possible. Therefore, analyses of changes from baseline were performed in the pooled sample of participants enrolled from studies PB-102-F20 and PB-102-F30 (87 participants). | Posted | Mean | Standard Deviation | nM | 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) |
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| Secondary | 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. | The ITT population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose, including a partial dose of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Mean | Standard Deviation | score on a scale | 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) |
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| Secondary | 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 | The ITT population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose, including a partial dose of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Mean | Standard Deviation | score on a scale | 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) |
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| Secondary | 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. | Participants in the ITT population with data available for pain medication use at baseline and at 72 months | Posted | Count of Participants | Participants | 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) |
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| Secondary | 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. | The EQ-5D-5L was not evaluated during study PB-102-F03. Therefore, analysis of change from baseline was performed in a pooled sample of participants from studies PB-102-F20 and PB-102-F30 (ITT Population excluding Cohort F03). | Posted | Mean | Standard Deviation | score on a scale | 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) |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | 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. | The ITT population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose, including a partial dose of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Mean | Standard Deviation | g/m^2 | 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) |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Number of participants in the Safety Population (excluding Cohort F03) who had data available for infusion premedication use at baseline and at 12 months. | Posted | Count of Participants | Participants | 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 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Number of participants in the Safety Population (excluding Cohort F03) who had data available for infusion premedication use at baseline and at 24 months. | Posted | Count of Participants | Participants | 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 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Number of participants in the Safety Population (excluding Cohort F03) who had data available for infusion premedication use at baseline and at 48 months | Posted | Count of Participants | Participants | 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 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Number of participants in the Safety Population (excluding Cohort F03) who had data available for infusion premedication use at baseline and at 72 months | Posted | Count of Participants | Participants | 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 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Safety Population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose (including a partial dose) of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Count of Participants | Participants | 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. |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Participants in the Safety Population with treatment-emergent ADA | Posted | Count of Participants | Participants | 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. |
| ||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Safety Population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose (including a partial dose) of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Count of Participants | Participants | 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. |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Safety Population, which included all participants who provided informed consent for study CLI-06657AA1-04 and received any dose (including a partial dose) of pegunigalsidase alfa in study CLI-06657AA1-04. | Posted | Count of Participants | Participants | 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. |
|
|
From first infusion until 90 days following the final visit dose for each participant. Mean (SD) individual exposure to study treatment was 5.5 (1.96) years.
Each participant was questioned about AEs. The Investigator reported all AEs (directly observed or spontaneously reported by the participant).
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Experimental Open Label | pegunigalsidase alfa pegunigalsidase alfa: Recombinant human alpha galactosidase A | 4 | 97 | 45 | 97 | 97 | 97 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute myocardial infarction | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Atrial flutter | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Myocardial ischaemia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Acute coronary syndrome | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Atrioventricular block complete | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Atrioventricular block second degree | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Left ventricular dysfunction | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Sinus arrest | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Ventricular tachycardia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Left ventricular outflow tract obstruction | Congenital, familial and genetic disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Vertigo positional | Ear and labyrinth disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pneumoperitoneum | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Protein-losing gastroenteropathy | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Sudden death | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chills | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypothermia | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Arthritis bacterial | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Clostridial infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Infectious mononucleosis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Meningitis aseptic | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Femur fracture | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Alcohol poisoning | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Pelvic fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Tendon rupture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Vaccination complication | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Troponin I increased | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Soft tissue mass | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Ischaemic stroke | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abortion spontaneous | Pregnancy, puerperium and perinatal conditions | MedDRA (19.0) | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| End stage renal disease | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Eosinophilic bronchitis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Obstructive airways disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Implantable defibrillator insertion | Surgical and medical procedures | MedDRA (19.0) | Systematic Assessment |
| |
| Medical device battery replacement | Surgical and medical procedures | MedDRA (19.0) | Systematic Assessment |
| |
| Nephrectomy | Surgical and medical procedures | MedDRA (19.0) | Systematic Assessment |
| |
| Postoperative care | Surgical and medical procedures | MedDRA (19.0) | Systematic Assessment |
| |
| Aortic stenosis | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Extremity necrosis | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Venous thrombosis limb | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Left ventricular hypertrophy | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Bundle branch block right | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Mitral valve incompetence | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Bundle branch block left | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Tricuspid valve incompetence | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Infusion site extravasation | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Vaccination complication | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Coronavirus test positive | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Urine protein/creatinine ratio | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
|
Any information relating to the study product or the study including any data and results from the study is the exclusive property of the Sponsor. The Investigator and any other persons involved in the study must protect the confidentiality of the proprietary information belonging to Chiesi Farmaceutici S.p.A. The Investigator should ensure that confidentiality of participants' data is preserved.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Transparency | Chiesi Farmaceutici S.p.A. | +3905212791 | clinicaltrials_info@chiesi.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 14, 2025 | Jan 28, 2026 | SAP_003.pdf |
Not provided
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| D011507 | Proteinuria |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
| D014555 | Urination Disorders |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D020924 | Urological Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
|
| Unknown or Not Reported |
|
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
| ADA-negative |
|
|
|
|
|
|
| None |
|
|
| Title | Measurements |
|---|---|
|
| Pruritus |
|
| Headache |
|
| Abdominal pain |
|
| Chills |
|
| Dizziness |
|
| Fatigue |
|
| Vomiting |
|
| Body temperature increased |
|
| Chest discomfort |
|
| Diarhoea |
|
| Hypersensitivity |
|
| Infusion site extravasation |
|
| Oedema peripheral |
|
| Paraesthesia |
|
| Peripheral swelling |
|
| Sneezing |
|
| Supraventricular extrasystoles |
|
| Weight increased |
|
|
|
|
|
| Participants |
|
|
|
|
|
|
|
|
|
|
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Participants |
|
|
|
|
| UPCR ≥1 g/g at last scheduled visit |
|
|
| UPCR ≥1 g/g at last scheduled visit |
|
|
| UPCR ≥1 g/g at last scheduled visit |
|
| Participants using 2 or more pain medications at 72 months |
|
| Participants using 2 or more pain medications at 72 months |
|
| Participants who used 2 infusion premedications at 12 months |
|
| Participants who used 3 or more infusion premedications at 12 months |
|
| Participants who used 2 infusion premedications at 12 months |
|
| Participants who used 3 or more infusion premedications at 12 months |
|
| Participants who used 2 infusion premedications at 12 months |
|
| Participants who used 3 or more infusion premedications at 12 months |
|
| Participants who used 2 infusion premedications at 24 months |
|
| Participants who used 3 or more infusion premedication at 24 months |
|
| Participants who used 2 infusion premedications at 24 months |
|
| Participants who used 3 or more infusion premedication at 24 months |
|
| Participants who used 2 infusion premedications at 24 months |
|
| Participants who used 3 or more infusion premedication at 24 months |
|
| Participants who used 2 infusion premedications at 48 months |
|
| Participants who used 3 or more infusion premedications at 48 months |
|
| Participants who used 2 infusion premedications at 48 months |
|
| Participants who used 3 or more infusion premedications at 48 months |
|
| Participants who used 2 infusion premedications at 48 months |
|
| Participants who used 3 or more infusion premedications at 48 months |
|
| Participants who used 2 infusion premedication at 72 months |
|
| Participants who used 3 or more infusion premedication at 72 months |
|
| Participants who used 2 infusion premedication at 72 months |
|
| Participants who used 3 or more infusion premedication at 72 months |
|
| Participants who used 2 infusion premedication at 72 months |
|
| Participants who used 3 or more infusion premedication at 72 months |
|