Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fabry patients | Adult patients whit genetically confirmed diagnosis of Fabry disease being treated or planning to start treatment with Pegunigalsidase alfa according to clinical practice. Participants will be required to meet the inclusion and exclusion criteria and sign informed consent to be enrolled in the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| pegunigalsidase alfa | Drug | Pegunigalsidase alfa is 2 mg/mL concentrate for solution and is administered via intravenous infusion every two weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of structural abnormalities of the left ventricle | Changes from baseline in structural abnormalities of the left ventricle assessed by echocardiography and defined by the presence of Left Ventricular Hypertrophy. | 24 months |
| Assessment of left ventricular diastolic function | Changes from baseline in left ventricular diastolic function by echocardiography. Diastolic dysfunction will be assessed using early to late diastolic trans-mitral flow velocity. | 12 and 24 months |
| Assessment of renal function | Changes from baseline in renal function by estimated Glomerular Filtration Rate. | 6, 12, 18 and 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of peak oxygen uptake (pVO2) | Changes from baseline in peak oxygen uptake (pVO2) using cardiopulmonary exercise testing (CPET). | 24 months |
| Assessment of carbon dioxide production (VCO2) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Adults patients who have a genetically confirmed diagnosis of Fabry disease and are being treated or plan to initiate treatment with pegunigalsidase alfa as per clinical practice.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Chiesi Clinical Trial info | Contact | +3905212791 | clinicaltrials_info@chiesi.com |
| Name | Affiliation | Role |
|---|---|---|
| Antonio Pisani, MD | AOU Federico II, Dipartimento di Nefrologia, Napoli, Italy | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| AUSL Toscana Sud-Est - Ospedale San Donato | Not yet recruiting | Arezzo | Italy |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
Not provided
Not provided
Not provided
Not provided
Not provided
Changes from baseline in carbon dioxide production (VCO2) using cardiopulmonary exercise testing (CPET).
| 24 months |
| Assessment of NT-pro-BNP and high sensitivity cardiac troponin levels | Changes from baseline in NT-pro-BNP and high sensitivity cardiac troponin levels. | 6, 12,18 and 24 months |
| Assessment of Cardiovascular Magnetic Resonance (CMR) | Changes from baseline in CMR assessed using T1 mapping to achieve myocardial tissue characterization. | 12 and 24 months |
| Assessment of proteinuria and microalbuminuria | Changes from baseline in proteinuria and microalbuminuria assessed on 24-hour urine sample. | 6, 12, 18 and 24 months |
| Assessment of Globotriaosylsphingosine (lyso-Gb3) | Changes from baseline in blood concentration of lyso-Gb3 | 3, 6, 12 and 24 months |
| Assessment of QoL outcomes using the Short Form Health Survey 36 (SF-36) | Changes from baseline in SF-36 score. It is a disease-specific questionnaire consisting of eight domains, each represented by a scale calculated as the weighted sum of responses in the respective sections. Each domain score ranges from 0 to 100, with all questions given equal weight. A score of 0 indicates the worst possible health status, while a score of 100 represents the best possible health status. | 12 and 24 months |
| Assessment of QoL outcomes using the Kansas City Cardiomyopathy Questionnaire (KCCQ) | Changes from baseline in KCCQ scores. It measures patient's perception of their health status, focusing on heart failure symptoms, the impact on physical and social functioning, and the overall effect of heart failure on their QoL within a 2-week recall period. It evaluates seven domains, with scores ranging from 0 to 100, where lower scores indicate more severe symptoms or limitations, while a score of 100 represents no symptoms, no limitations, and excellent quality of life. | 12 and 24 months |
| Assessment of severity of neuropathic pain using Chronic Pain Grade (CPG) | Changes from baseline in CPG score. It is a 7-item questionnaire that evaluates chronic pain severity experienced over the past six months. Pain intensity is rated from 0 (no pain) to 10 ("pain as bad as it can be"). Scores for pain intensity and disability are combined to classify the overall severity of chronic pain into four grades, ranging from Grade 0 (no pain) to Grade IV (high disability, severely limiting). | 12 and 24 months |
| Assessment of severity of neuropathic pain using Short Form of the Brief Pain Inventory (BPI-SF) | Changes from baseline in BPI-SF score. It is a 9-item questionnaire assessing Pain Intensity and Pain Interference, as its impact on daily functions, over the past 24 hours. Pain intensity is rated on a 0 (no pain) to 10 (pain as bad as you can imagine) scale, across four measures: worst, least, average, and current pain. Pain interference with daily activities is also scored from 0 (no interference) to 10 (completely interferes). | 12 and 24 months |
| Assessment of gastrointestinal symptoms using the Gastrointestinal Symptoms Rating Scale (GSRS) | Changes from baseline in GSRS score. It is a 15-item questionnaire designed to assess changes in gastrointestinal symptoms across five clusters: reflux, abdominal pain, indigestion, diarrhea, and constipation. The GSRS uses a seven-point Likert-type scale, with scores ranging from 1 (no troublesome symptoms) to 7 (very troublesome symptoms). | 12 and 24 months |
| Assessment of specific Fabry disease ocular findings | Number of patients with presence of specific Fabry disease ocular findings (i.e. cornea verticillate) by slit lamp | Baseline, 12 and 24 months |
| Adverse events | Number of AEs | 24 months |
| Adverse drug reactions to pegunigalsidase alfa | Number of ADRs | 24 months |
| Anti-drug antibodies (ADAs) testing | Number of patients with ADAs | 24 months |
| Infusion-Related Reactions (IRRs) | Number of patients and occurrence of IRRs | 24 months |
| Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari | Recruiting | Bari | Italy |
|
| Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola | Recruiting | Bologna | Italy |
|
| A.O. Sant'Anna e San Sebastiano | Not yet recruiting | Caserta | Italy |
|
| U.O.C. Nefrologia ASST Monza - Ospedale San Gerardo | Not yet recruiting | Monza | Italy |
|
| AOU Federico II, Dipartimento di Nefrologia | Not yet recruiting | Naples | Italy |
|
| A.O.U. Policlinico "Giaccone" | Not yet recruiting | Palermo | Italy |
|
| 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 |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |