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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-004233-40 | EudraCT Number |
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| Name | Class |
|---|---|
| Takeda | INDUSTRY |
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The main aim of this study is to learn more about the safety profile of Replagal.
Participants will receive Replagal every 2 weeks at the clinic for about 1 year.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Replagal | Experimental | Participants with fabry disease will receive Replagal 0.2 milligram per kilogram (mg/kg) intravenous infusion on Day 1 and every 2 weeks up to Week 51. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Replagal | Biological | Participants will receive Replagal 0.2 mg/kg, intravenous infusion at Day 1 and every 2 weeks. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event. | From the start of study up to 53 weeks |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | AE is any unfavorable and unintended sign, symptom, or disease temporally associated with study or use of investigational drug product (IP), whether or not the AE is considered related to IP. TEAEs: AEs occurring or worsening at or after first dose of IP or ongoing at time of enrollment. SAE :untoward medical occurrence that at any dose met one, more of the following criteria: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event. Severity: Mild: event that does not generally interfere with usual activities of daily living; Moderate: event that interferes with usual activities of daily living, causing discomfort, permanent risk of harm; Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. | From the study drug administration up to Week 53 |
| Number of Participants With Adverse Drug Reactions (ADRs) Related to Replagal | An ADR is defined as a response to a drug which is noxious and unintended, and which occurs at doses normally used in humans for prophylaxis, diagnosis, or therapy of disease, or for the modification of physiological function. Number of participants with ADRs will be reported. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) | Change in eGRF levels will be assessed from baseline up to week 53. | Baseline and at Weeks 13, 27, 39, and 53 |
| Number of Participants With Change in Frequency and Regimen of Analgesic use of Replagal for Neuropathic Pain |
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Inclusion Criteria:
Note: Female participants not of childbearing potential defined as those who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (example, defined as at least 1 year since last regular menses with an appropriate clinical profile [that is, age appropriate, history of vasomotor symptoms]).
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Takeda Contact | Contact | +1 866 842 5335 | ClinicalTransparency@takeda.com |
| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Institute of Child Health | Not yet recruiting | Kolkata | 700017 | India |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
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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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| ID | Term |
|---|---|
| C000627036 | agalsidase alfa |
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| From the study drug administration up to Week 53 |
| Number of Participants With Infusion-related Reactions of Replagal | Number of participants with infusion-related reactions of Replagal will be reported. | From the study drug administration up to Week 53 |
Participants who are already taking analgesics, any change in frequency and regimen of analgesic use will be monitored throughout the study. Number of participants with change in frequency and regimen of analgesic use of Replagal for neuropathic pain will be reported. |
| Baseline up to Week 53 |
| Change From Baseline in Urine Concentration of Globotriaosylceramide (Gb3) | Change from baseline in urine concentration of Gb3 will be assessed. | Baseline and at Weeks 13, 27, 39, and 53 |
| Change From Baseline in Urine Protein Creatinine Ratio | Urine protein creatinine ratio will be assessed. | Baseline and at Weeks 13, 27, 39, and 53 |
| Percent Change From Baseline in Left Ventricular Mass Index (LVMI) | The 2-dimensional (2D) echocardiogram will be performed within one month of participant enrolment or at the time of screening, Week 27, and Week 53 and the percent change values from baseline in LVMI (gram per meter square [g/m^2]) will be measured and reported. | Baseline and at Weeks 27 and 53 |
| Percent Change From Baseline in Left Ventricular Wall Thickness | The 2D echocardiogram will be performed within one month of participant enrolment or at the time of screening, Week 27, and Week 53 and the percent change values from baseline in left ventricular muscular thickness will be measured and reported. | Baseline and at Weeks 27 and 53 |
| Percent Change From Baseline in Ejection Fraction | The 2D echocardiogram will be performed within one month of participant enrolment or at the time of screening, Week 27, and Week 53 and the percent change values from baseline in ejection fraction will be measured and reported. | Baseline and at Weeks 27 and 53 |
| Percent Change From Baseline in Quality of Life Based on Questionnaire 36-itme Form Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) | SF-36 is a generic quality-of-life instrument that has been widely used to assess health-related quality of life (HRQL) of participants. SF-36 consist of 36 items that are aggregated into 8 multi-item scales (physical functioning [1=yes, limited a lot to 3=no, not limited at all], role-physical [1=all of the time to 5=none of the time], bodily pain [1=very severe to 6=none], general health [1=poor to 5=excellent], vitality [1=none of the time to 5=all of the time], social functioning [1=all of the time: to 5=none of the time], role emotional [1=all of the time to 5=none of the time] and mental health [1=all of the time to 5=none of the time]). Four domains comprised physical component summary (PCS) score (physical functioning, role-physical, bodily pain, general health) and remaining 4 domains comprised mental component summary (MCS) score (vitality, social functioning, role-emotional, mental health). The scores ranges from 0 to 100. Higher scores indicating better HRQL. | Baseline and at Weeks 27 and 53 |
| All India Institute of Medical Sciences (AIIMS) | Recruiting | New Delhi | 110029 | India |
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| Sir Gangaram Hospital | Recruiting | New Delhi | 110060 | India |
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| 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 |