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| Name | Class |
|---|---|
| Chiesi Farmaceutici S.p.A. | INDUSTRY |
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To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
An open-label study to evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients (≥18 years of age). Patients enrolled received 1.0 mg/kg of PRX-102 as an intravenous infusion every 2 weeks (+/- 3 days) for up to 60 months and no less than 36 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PRX-102 (pegunigalsidase alfa) | Experimental | PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days) |
|
| 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 |
|---|---|---|
| Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03 | Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment. | Every two weeks up to 60 months |
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| Measure | Description | Time Frame |
|---|---|---|
| Plasma Lyso-Gb3 Concentration | Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker, which was measured at Baseline, every 3 months up to 24 months and then every 6 months up to the end of the study. Baseline and Month 60 and change from Baseline reported. | Baseline and month 60 |
| Estimated Glomerular Filtration Rate (eGFR) |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32611 | United States | ||
| University of Iowa Hospitals and Clinics |
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A total of 15 adult patients (8 male and 7 female) who completed study PB-102-F02 were enrolled in this study and included in both the Safety and Efficacy populations.
Adult Fabry disease patients who have successfully completed treatment with PRX-102 in previous studies PB-102-F01 and PB-102-F02
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| ID | Title | Description |
|---|---|---|
| FG000 | PRX-102 (Pegunigalsidase Alfa) | PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days) |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | PRX-102 (Pegunigalsidase Alfa) | PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days) |
| 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 |
| 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 | Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03 | Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment. | Safety population is defined as all patients who received any dose (partial or complete) of study treatment as part of study PB-102-F03. Efficacy population is defined as all patients who received at least one complete dose of the study treatment as part of study PB-102-F03 | Posted | Number | participants | Every two weeks up to 60 months |
|
The adverse events were collected during the period from PB-102-F03 baseline up to the end of study PB-102-F03 (up to 60 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) | PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anogenital warts | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (15.0) | Systematic Assessment |
Small sample size.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sari Alon MSc | Protalix Ltd. | +972-4-9028100 | 215 | sari@protalix.com |
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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 | Jan 5, 2016 | Jan 2, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 2, 2021 | Dec 20, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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eGFR was calculated based on the serum creatinine values according to the CKD-EPI equation. The absolute change in eGFR from baseline measurement at visit 1 to Month 60 was summarized using descriptive statistics. Baseline and month 60 reported. |
| Baseline and Month 60 |
| Iowa City |
| Iowa |
| 52242 |
| United States |
| Johns Hopkins University School of Medicine | Baltimore | Maryland | 21205 | United States |
| Institute of Metabolic Disease | Dallas | Texas | 75226 | United States |
| O & O Alpan | Fairfax | Virginia | 22030 | United States |
| Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C) | Asunción | Paraguay |
| Hospital de Dia Quiron Zaragoza | Zaragoza | 50012 | Spain |
| The Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Units | Counts |
|---|
| Participants |
|
|
| Other Pre-specified | Plasma Lyso-Gb3 Concentration | Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker, which was measured at Baseline, every 3 months up to 24 months and then every 6 months up to the end of the study. Baseline and Month 60 and change from Baseline reported. | Posted | Mean | Standard Error | ng/mL | Baseline and month 60 |
|
|
|
| Other Pre-specified | Estimated Glomerular Filtration Rate (eGFR) | eGFR was calculated based on the serum creatinine values according to the CKD-EPI equation. The absolute change in eGFR from baseline measurement at visit 1 to Month 60 was summarized using descriptive statistics. Baseline and month 60 reported. | Posted | Mean | Standard Error | ml/min/1.73 m^2 | Baseline and Month 60 |
|
|
|
| 1 |
| 15 |
| 3 |
| 15 |
| 15 |
| 15 |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (15.0) | Systematic Assessment |
|
| Drug hypersensitivity | Immune system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Seasonal allergy | Immune system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Feeling hot | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Infusion site bruising | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Oedema | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pain | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Peripheral swelling | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA (15.0) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA (15.0) | Systematic Assessment |
|
| Breast tenderness | Reproductive system and breast disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pruritis genital | Reproductive system and breast disorders | MedDRA (15.0) | Systematic Assessment |
|
| Animal bite | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Sunburn | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Vaccination complication | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA (15.0) | Systematic Assessment |
|
| Blood creatine phosphokinase increased | Investigations | MedDRA (15.0) | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA (15.0) | Systematic Assessment |
|
| Abnormal chest x-ray | Investigations | MedDRA (15.0) | Systematic Assessment |
|
| Electrocardiogram ST segment abnormal | Investigations | MedDRA (15.0) | Systematic Assessment |
|
| Electrocardiogram ST segment depression | Investigations | MedDRA (15.0) | Systematic Assessment |
|
| Nuclear magnetic resonance imaging abnormal | Investigations | MedDRA (15.0) | Systematic Assessment |
|
| Left ventricular dysfunction | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
|
| Left ventricular hypertrophy | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
|
| Palpitations | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
|
| Sinus arrythmia | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
|
| Tricuspid valve incompetence | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
|
| Ventricular hypertrophy | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
|
| Fabry's disease | Congenital, familial and genetic disorders | MedDRA (15.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Emphysema | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Balance disorder | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Burning sensation | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Neuralgia | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Visual field defect | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Conjunctival haemorrhage | Eye disorders | MedDRA (15.0) | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA (15.0) | Systematic Assessment |
|
| Vertigo | Ear and labyrinth disorders | MedDRA (15.0) | Systematic Assessment |
|
| Deafness bilateral | Ear and labyrinth disorders | MedDRA (15.0) | Systematic Assessment |
|
| Ear discomfort | Ear and labyrinth disorders | MedDRA (15.0) | Systematic Assessment |
|
| Hypoacusis | Ear and labyrinth disorders | MedDRA (15.0) | Systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Haemorrhoids | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Inguinal hernia | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Tooth disorder | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dysuria | Renal and urinary disorders | MedDRA (15.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Hypohidrosis | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Clubbing | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Muscle spasm | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Spondyloarthropathy | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Upper respiratory tract infections | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Herpes virus infection | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Onychomycosis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Subcutaneous abscess | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Tooth infection | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
The clinical trial agreement provided to the sites and Investigators contains a Publication paragraph that indicates the following: shall not, without the Sponsor's prior written consent, independently publish, present or otherwise disclose any results of or information pertaining to the trial until a multicenter publication is published, subject to certain limitations regarding timing and the confidentiality of unpublished data.
| 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 |
|
| Change from Baseline to Month 60 |
|
|
| Change from Baseline to Month 60 |
|