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This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.
This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT. Eligible patients will enter a 12-week Stability Evaluation Period to establish the stability of their disease. Patients with stable disease will then be switched from their imiglucerase treatment to receive intravenous (IV) infusions of taliglucerase alfa every two weeks for a total of 20 IV infusions. The dose of taliglucerase alfa will be equal to each patient's previous imiglucerase dose. The infusions will be administered at the selected investigational site (clinic/hospital), infusion center, or at home. At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Taliglucerase alfa | Experimental | Open label taliglucerase alfa treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Taliglucerase alfa | Drug | Intravenous infusion every 2 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Hemoglobin | Every 3 months from Baseline to Month 9 |
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| Measure | Description | Time Frame |
|---|---|---|
| Platelet Count | Every 3 months from Baseline to Month 9 | |
| Spleen Volume | Spleen volume measured by MRI in mL | Baseline and 9 Months |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Research Foundation for Lysosomal Storage Diseases, Inc. | Coral Springs | Florida | 33065 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24950666 | Derived | Pastores GM, Petakov M, Giraldo P, Rosenbaum H, Szer J, Deegan PB, Amato DJ, Mengel E, Tan ES, Chertkoff R, Brill-Almon E, Zimran A. A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase. Blood Cells Mol Dis. 2014 Dec;53(4):253-60. doi: 10.1016/j.bcmd.2014.05.004. Epub 2014 Jun 18. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Taliglucerase Alfa | Open label taliglucerase alfa treatment Taliglucerase alfa: Intravenous infusion every 2 weeks |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Taliglucerase Alfa | Open label taliglucerase alfa treatment Taliglucerase alfa: Intravenous infusion every 2 weeks |
| 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 | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Other Pre-specified | Platelet Count | Posted | Mean | Standard Deviation | platelets/mm^3 | Every 3 months from Baseline to Month 9 |
|
|
9 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 | Taliglucerase Alfa | Open label taliglucerase alfa treatment Taliglucerase alfa: Intravenous infusion every 2 weeks |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment | Right nostril due to perforated septum - Not Related |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| DIARRHOEA | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| VP Medical Affairs | Protalix Ltd. | +972-4-9028100 | raul@protalix.com |
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| D016464 | Lysosomal Storage Diseases |
| 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 |
|---|---|
| C582473 | taliglucerase alfa |
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| Liver Volume |
Liver volume measured by MRI |
| Baseline and 9 months |
| Department of Human Genetics, Emory University School of Medicine |
| Decatur |
| Georgia |
| 30033 |
| United States |
| Neurogenetics, NYU at Rivergate | New York | New York | 10016 | United States |
| Bone Marrow Transplant Service, The Royal Melbourne Hospital | Parkville | Victoria | Australia |
| Mount Sinai Hospital | Toronto | Ontario | M5G 1X5 | Canada |
| Rambam Medical Center | Haifa | 31096 | Israel |
| Shaare Zedek Medical Center | Jerusalem | Israel |
| Sala de Hematologia, Hospital Universitario Miguel Servet | Zaragoza | 50009 | Spain |
| Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust | Cambridge | United Kingdom |
| Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Religion | Number | participants |
|
|
| Other Pre-specified | Spleen Volume | Spleen volume measured by MRI in mL | 25 patients had spleen volume measured by MRI, 2 were MRI phobic and 3 were splenectomized | Posted | Mean | Standard Deviation | mL | Baseline and 9 Months |
|
|
|
| Other Pre-specified | Liver Volume | Liver volume measured by MRI | 2 patients were MRI phobic | Posted | Mean | Standard Deviation | mL | Baseline and 9 months |
|
|
|
| Primary | Hemoglobin | Posted | Mean | Standard Deviation | g/dL | Every 3 months from Baseline to Month 9 |
|
|
|
| 3 |
| 31 |
| 29 |
| 31 |
|
| NEPHROLITHIASIS | Renal and urinary disorders | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| PELVIC PROLAPSE | Reproductive system and breast disorders | MedDRA 15.0 | Systematic Assessment | Prolapsed rectum, cervix and bladder - Not Related |
|
| PAIN | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| NASOPHARYNGITIS | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| INFUSION RELATED REACTION | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| HEADACHE | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| PRURITUS | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
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| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |