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| Name | Class |
|---|---|
| Chiesi Farmaceutici S.p.A. | INDUSTRY |
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This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients who had been treated for approximately 1 year with agalsidase beta and who had been on a stable dose of that product for at least 6 months were randomized in a 2:1 ratio to either switch to PRX-102 or to continue treatment with agalsidase beta. Both treatments were delivered by intravenous infusions every two weeks, at a dosage of 1 mg/kg.
This was a randomized, double-blind, active control study examining the safety and efficacy of pegunigalsidase alfa (PRX-102) in Fabry disease patients with impaired renal function. Participants had to have been taking the licensed ERT drug agalsidase beta (Fabrazyme®) for at least 1 year prior to study entry, and to have been on a stable dose of that product for at least the last 6 months. Since the disease expresses itself differently in males and females, gender could have an impact on the therapeutic effect; thus, there was additionally a requirement that no more than 50% of the patients could be female.
Following screening, eligible patients were randomized in a 2:1 ratio to either switch to PRX-102 or continue treatment with agalsidase beta, with randomization stratified according to whether the urine protein-to-creatinine ratio (UPCR), a measure of kidney function, was above or below a specified threshold. Both products were administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg, for up to 24 months. Both patients and study staff were blinded as to which treatment was being given.
Patients who completed the study were invited to continue in a long-term open-label extension study, PB-102-F60, in which all participants would receive PRX-102 1 mg/kg every 2 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PRX-102 (pegunigalsidase alfa) | Experimental | PRX-102 infusion every 2 weeks |
|
| agalsidase beta | Active Comparator | agalsidase beta infusion every 2 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PRX-102 (pegunigalsidase alfa) | Biological | PRX-102 1 mg/kg every 2 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) | The individual annualized mean change (slope) in eGFR (mL/min/1.73 m^2/year) is an estimate of the individual patient's annualized change in eGFR, which is derived from the eGFR assessments over time, for up to 24 months. The individual annualized mean change (slope) in eGFR is estimated for each patient with at least 4 eGFR observations. For patients with fewer than 4 eGFR observations, the slope will be missing. | 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Estimated Glomerular Filtration Rate (eGFR) | eGFR was calculated based on measured serum creatinine levels according to the CKD-EPI formula. The median values obtained at baseline and at Month 24 are reported. The change in eGFR from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. |
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Inclusion Criteria:
Symptomatic adult Fabry disease patients, age 18-60 years
Males: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels and one or more of the characteristic features of Fabry disease
i. neuropathic pain
ii. cornea verticillata
iii. clustered angiokeratoma
Females:
a. historical genetic test results consistent with Fabry pathogenic mutation and one or more of the described characteristic features of Fabry disease:
i. neuropathic pain
ii. cornea verticillata
iii. clustered angiokeratoma
b. or in the case of novel mutations a first degree male family member with Fabry disease with the same mutation, and one or more of the characteristic features of Fabry disease
i. neuropathic pain
ii. cornea verticillata
iii. clustered angiokeratoma
Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m²
Linear negative slope of eGFR based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit) of ≥ 2 mL/min/1.73 m²/year
Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months.
Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UAB Medicine | Birmingham | Alabama | 35233 | United States | ||
| Phoenix Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39847314 | Derived | Azimpour K, Dorling P, Koulinska I, Kunduri S, Lan Z, Poritz J, Tremblay G, Raad-Faherty A. Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials. Adv Ther. 2025 Mar;42(3):1421-1434. doi: 10.1007/s12325-024-03095-2. Epub 2025 Jan 23. | |
| 37940383 | Derived | Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tondel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445. |
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Of the 78 randomized patients, 53 were assigned to the PRX-102 arm and 25 to the agalsidase beta arm. One PRX-102 patient withdrew consent before receiving the study product; accordingly, 77 patients were treated, 52 in the PRX-102 arm and 25 in the agalsidase beta arm.
Symptomatic adult Fabry patients who had been taking agalsidase beta for at least 1 year and on a stable dose for at least 6 months. No more than 50% could be female. Screening eGFR (CKD-EPI) 40 to 120 mL/min/1.73 m^2; Screening linear eGFR slope more negative than -2 mL/min/1.73 m^2/year based on at least 3 values over ~1 year.
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| ID | Title | Description |
|---|---|---|
| FG000 | PRX-102 (Pegunigalsidase Alfa) | Pegunigalsidase alfa administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg |
| FG001 | Agalsidase Beta | Agalsidase beta administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg |
| 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 | Jul 14, 2017 | Mar 28, 2023 |
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| agalsidase beta | Biological | agalsidase beta 1 mg/kg every 2 weeks |
|
|
| Baseline and Month 24 |
| Plasma Lyso-Gb3 | Globotriaosylsphingosine (lyso-Gb3) is a Fabry disease-specific biomarker measured in the plasma. The median concentrations obtained at baseline and at Month 24, and the change from baseline to Month 24 in median concentration, are reported. The change in Lyso-Gb3 from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. | Baseline and Month 24 |
| Short Form Brief Pain Inventory (BPI) | The Short Form Brief Pain Inventory ( BPI) questioner is self-completed by patients regarding pain severity and interference. Descriptive statistics summarize the findings for the change from baseline at Week 104 for "Pain at Its Worst in Last 24 Hours". The severity of various aspects of pain scored on a scale of 0 to 10 ( no pain / pain as bad as you can imagine). The change in BPI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes. | Baseline and Month 24 |
| Mainz Severity Score Index (MSSI) | The Mainz Severity Score Index (MSSI) is an instrument that is specifically designed to measure the severity of Fabry disease signs/symptoms and to monitor the clinical course of the disease. The MSSI is administered by the investigator, and yields scores for general, neurological, cardiovascular, renal, and overall assessments. The overall score range from 0 to 76. An overall score of less than 20 points is considered mild signs and symptoms of Fabry disease, 20 to 40 is considered moderate, and greater than 40 is considered severe. The change in overall MSSI score from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes. | Baseline and Month 24 |
| Urine Protein/Creatinine Ratio (UPCR) | The UPCR provides an estimate of protein excretion in urine, and is used as an indicator of the extent of chronic kidney disease. It was classified into three categories: 1) UPCR ≤ 0.5 gr/gr, 2) 0.5 gr/gr < UPCR < 1 gr/gr, 3) 1 gr/gr ≤ UPCR. The results are presented as the percentage of patients (%) in each category at baseline and Month 24. There are no statistical analyses for this endpoint. | Baseline and Month 24 |
| Left Ventricular Mass Index With Hypertrophy at Baseline | Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients with hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2). The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. | Baseline and Month 24 |
| Left Ventricular Mass Index Without Hypertrophy at Baseline | Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients without hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2). The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. | Baseline and Month 24 |
| Phoenix |
| Arizona |
| 85016 |
| United States |
| University of California Irvine Center | Orange | California | 92868 | United States |
| University of California San Diego | San Diego | California | 92093 | United States |
| Emory University School of Medicine | Atlanta | Georgia | 30322 | United States |
| University of Iowa Hosptials and Clinics | Iowa City | Iowa | 52242 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Infusion Associates | Grand Rapids | Michigan | 49525 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Institute of Metabolic Disease, Baylor Healthcare | Dallas | Texas | 75226 | United States |
| Renal Disease Research Institute, LLC - Dallas | Dallas | Texas | 75235 | United States |
| Eccles Primary Children's Outpatient Services Building | Salt Lake City | Utah | 84132 | United States |
| O+O Alpan LLC | Fairfax | Virginia | 22030 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226-3596 | United States |
| Vseobecna fakultni nemocnice v Praze | Prague | Czech Republic | 12808 | Czechia |
| Turku University Central Hospital | Turku | 20520 | Finland |
| Hôpital Raymond Poincaré | Paris | 92380 | France |
| Semmelweis Egyetem | Budapest | 1083 | Hungary |
| Azienda Ospedaliera Universitaria "Federico II" | Naples | 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 |
| Klinik und Poliklinik für Innere Medizin UniversitätsSpital Zürich | Zurich | Switzerland |
| Institute of Metabolism and Systems Research | Edgbaston | Birmingham | United Kingdom |
| Addenbrooke's Hospital | Cambridge | CB2 0QQ | United Kingdom |
| The Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Salford Royal NHS Foundation Trust | Salford | M6 8HD | United Kingdom |
| Treated | One patient from PRX-102 arm withdrew consent before receiving the study product |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | PRX-102 (Pegunigalsidase Alfa) | Pegunigalsidase alfa administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg |
| BG001 | Agalsidase Beta | Agalsidase beta administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
|
| 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) | The individual annualized mean change (slope) in eGFR (mL/min/1.73 m^2/year) is an estimate of the individual patient's annualized change in eGFR, which is derived from the eGFR assessments over time, for up to 24 months. The individual annualized mean change (slope) in eGFR is estimated for each patient with at least 4 eGFR observations. For patients with fewer than 4 eGFR observations, the slope will be missing. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication with at least 4 eGFR observations. For 1 patient with fewer than 4 eGFR observations, the slope was missing. | Posted | Median | 95% Confidence Interval | mL/min/1.73 m^2/year | 24 months |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Estimated Glomerular Filtration Rate (eGFR) | eGFR was calculated based on measured serum creatinine levels according to the CKD-EPI formula. The median values obtained at baseline and at Month 24 are reported. The change in eGFR from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | Posted | Median | Full Range | mL/min/1.73 m^2 | Baseline and Month 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Plasma Lyso-Gb3 | Globotriaosylsphingosine (lyso-Gb3) is a Fabry disease-specific biomarker measured in the plasma. The median concentrations obtained at baseline and at Month 24, and the change from baseline to Month 24 in median concentration, are reported. The change in Lyso-Gb3 from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | Posted | Median | Full Range | nM | Baseline and Month 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Short Form Brief Pain Inventory (BPI) | The Short Form Brief Pain Inventory ( BPI) questioner is self-completed by patients regarding pain severity and interference. Descriptive statistics summarize the findings for the change from baseline at Week 104 for "Pain at Its Worst in Last 24 Hours". The severity of various aspects of pain scored on a scale of 0 to 10 ( no pain / pain as bad as you can imagine). The change in BPI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | Posted | Mean | Standard Error | score on a scale | Baseline and Month 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mainz Severity Score Index (MSSI) | The Mainz Severity Score Index (MSSI) is an instrument that is specifically designed to measure the severity of Fabry disease signs/symptoms and to monitor the clinical course of the disease. The MSSI is administered by the investigator, and yields scores for general, neurological, cardiovascular, renal, and overall assessments. The overall score range from 0 to 76. An overall score of less than 20 points is considered mild signs and symptoms of Fabry disease, 20 to 40 is considered moderate, and greater than 40 is considered severe. The change in overall MSSI score from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | Posted | Mean | Standard Error | score on a scale | Baseline and Month 24 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Urine Protein/Creatinine Ratio (UPCR) | The UPCR provides an estimate of protein excretion in urine, and is used as an indicator of the extent of chronic kidney disease. It was classified into three categories: 1) UPCR ≤ 0.5 gr/gr, 2) 0.5 gr/gr < UPCR < 1 gr/gr, 3) 1 gr/gr ≤ UPCR. The results are presented as the percentage of patients (%) in each category at baseline and Month 24. There are no statistical analyses for this endpoint. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | Posted | Number | percentage of participants | Baseline and Month 24 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Left Ventricular Mass Index With Hypertrophy at Baseline | Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients with hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2). The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. This analysis included the subset of patients in each treatment arm who had hypertrophy at baseline. | Posted | Median | Full Range | g/m^2 | Baseline and Month 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Left Ventricular Mass Index Without Hypertrophy at Baseline | Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients without hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2). The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the median (full range) of these changes. | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. This analysis included the subset of patients in each treatment arm who did not have hypertrophy at baseline. | Posted | Median | Full Range | g/m^2 | Baseline and Month 24 |
|
|
24 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PRX-102 (Pegunigalsidase Alfa) | Pegunigalsidase alfa administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg | 0 | 52 | 8 | 52 | 47 | 52 |
| EG001 | Agalsidase Beta | Agalsidase beta administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg | 0 | 25 | 6 | 25 | 24 | 25 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Aortic stenosis | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Venous thrombosis limb | Vascular disorders | 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 |
| |
| Hypothermia | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Atrioventricular block second degree | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Ventricular tachycardia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Altered state of consciousness | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Protein-losing gastroenteropathy | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Bronchitis | 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 |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypertension | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypotension | Vascular 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 |
| |
| Pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Device occlusion | Product Issues | MedDRA (19.0) | Systematic Assessment |
| |
| Urine protein/creatinine ratio increased | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Blood creatine increased | Investigations | MedDRA (19.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Vomiting | 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 |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue 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 |
| |
| 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 |
| |
| Muscle spasms | 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 |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sari Alon, MSc | Protalix Ltd. | +972-4-9028100 | 215 | sari@protalix.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 25, 2022 | Mar 28, 2023 | SAP_001.pdf |
| 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
| ID | Term |
|---|---|
| C459420 | agalsidase beta |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Hungary |
|
| United States |
|
| Czechia |
|
| Norway |
|
| Finland |
|
| Italy |
|
| United Kingdom |
|
| Slovenia |
|
| France |
|
| Spain |
|
|
|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
|