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This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Taliglucerase alfa 30 units/kg | Experimental | Subjects randomized to receive 30 units/kg |
|
| Taliglucerase alfa 60 units/kg | Experimental | Subjects randomized to 60 units/kg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Taliglucerase alfa | Drug | Taliglucerase infusion every two weeks for 21 months |
|
| Measure | Description | Time Frame |
|---|---|---|
| Spleen Volume | Spleen volume measured by MRI | 60 months |
| Measure | Description | Time Frame |
|---|---|---|
| Liver Volume | Liver volume by MRI | 60 months |
| Platelet Count | Platelet count measure annually | 60 months |
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Inclusion Criteria:
Exclusion Criteria:
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27499018 | Derived | Zimran A, Duran G, Giraldo P, Rosenbaum H, Giona F, Petakov M, Terreros Munoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R. Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naive patients with Gaucher disease. Blood Cells Mol Dis. 2019 Sep;78:14-21. doi: 10.1016/j.bcmd.2016.07.002. Epub 2016 Jul 18. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Taliglucerase Alfa 30 Units/kg | Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months |
| FG001 | Taliglucerase Alfa 60 Units/kg | Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months |
| FG002 | Dose Adjusted | Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Taliglucerase Alfa 30 Units/kg | Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months |
| BG001 | Taliglucerase Alfa 60 Units/kg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Spleen Volume | Spleen volume measured by MRI | Posted | Mean | Standard Error | Milliliters | 60 months |
|
21 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 30 Units/kg | Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 10.1 | Systematic Assessment | right hip arthrosis which required artificial hip implantation |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| NASOPHARYNGITIS | Respiratory, thoracic and mediastinal disorders | MedDRA 10.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Glen Park | Target Health Inc. | 212-681-2100 | gpark@targethealth.com |
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| ID | Term |
|---|---|
| D005776 | Gaucher 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 |
|---|---|
| C582473 | taliglucerase alfa |
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| Hemoglobin | Hemoglobin measure yearly | 60 months |
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
| BG002 | Dose Adjusted | Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months |
| BG003 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|
| Secondary | Liver Volume | Liver volume by MRI | Posted | Mean | Standard Error | Milliliters | 60 months |
|
|
|
| Secondary | Platelet Count | Platelet count measure annually | Posted | Mean | Standard Error | platelets/mm^3 | 60 months |
|
|
|
| Secondary | Hemoglobin | Hemoglobin measure yearly | Posted | Mean | Standard Error | mg/dL | 60 months |
|
|
|
| 0 |
| 8 |
| 6 |
| 8 |
| EG001 | Taliglucerase Alfa 60 Units/kg | Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months | 2 | 9 | 8 | 9 |
| EG002 | Dose Adjusted | Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months | 0 | 2 | 1 | 2 |
|
| Thyroid Carcinoma | Metabolism and nutrition disorders | MedDRA 10.1 | Systematic Assessment | papillary thyroid carcinoma that required total thyroidectomy and left node resection |
|
| PHARYNGITIS | Respiratory, thoracic and mediastinal disorders | MedDRA 10.1 | Systematic Assessment |
|
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA 10.1 | Systematic Assessment |
|
| HYPOAESTHESIA | Nervous system disorders | MedDRA 10.1 | Systematic Assessment |
|
| BONE PAIN | Musculoskeletal and connective tissue disorders | MedDRA 10.1 | Systematic Assessment |
|
| CONTUSION | Injury, poisoning and procedural complications | MedDRA 10.1 | Systematic Assessment |
|
| DYSPEPSIA | Gastrointestinal disorders | MedDRA 10.1 | Systematic Assessment |
|
| TOOTHACHE | Gastrointestinal disorders | MedDRA 10.1 | 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 |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
|
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