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Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.
This is an extension trial to Study NCT00376168 and NCT00712348.
This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Naive 30 Units/kg | Experimental | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
|
| Naive 60 Units/kg | Experimental | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
|
| Switchover | Experimental | Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Taliglucerase alfa | Drug | Intravenous infusion every 2 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Spleen Volume | Spleen volume measured by MRI | Spleen Volume at Baseline and Months 12, 24, and 36 |
| Measure | Description | Time Frame |
|---|---|---|
| Liver Volume | Liver volume measured by MRI | Liver volume at Baseline and Months 12, 24 and 36 |
| Hemoglobin | Hemoglobin at Baseline and Months 12, 24 and 36 |
| Measure | Description | Time Frame |
|---|---|---|
| Spleen Volume Multiples of Normal (MN) | Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg) | Baseline and Months 12, 24, and 36 |
| Liver Volume Multiples of Normal (MN) | Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg). |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Department of Human Genetics, Emory University School of Medicine | Decatur | Georgia | 30033 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27174694 | Derived | Zimran A, Duran G, Mehta A, Giraldo P, Rosenbaum H, Giona F, Amato DJ, Petakov M, Munoz ET, Solorio-Meza SE, Cooper PA, Varughese S, Chertkoff R, Brill-Almon E. Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naive patients with Gaucher disease. Am J Hematol. 2016 Jul;91(7):656-60. doi: 10.1002/ajh.24369. Epub 2016 Apr 24. | |
| 27102949 |
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Patients completing Studies PB-06-001 (NCT00376168) or PB-06-002 (NCT00712348) were offered continued treatment in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Naive 30 Units/kg | Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg |
| FG001 | Naive 60 Units/kg | Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with taliglucerase alfa 60 units/kg |
| FG002 | Switchover | Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
All treated patients are included in the analysis
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| ID | Title | Description |
|---|---|---|
| BG000 | Naive 30 Units/kg | Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg |
| BG001 | Naive 60 Units/kg | Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with 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 | Intent to treat. In the Switchover group, two patients did not have MRI and one patient was splenectomized. | Posted | Mean | Standard Deviation | mL | Spleen Volume at Baseline and Months 12, 24, and 36 |
|
36 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 | Naive 30 Units/kg | Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment | Surgery: Left knee arthroscopy - Not Related |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Vice President Product Development | Protalix Ltd. | +(972) 4-9889488 | 137 | einata@protalix.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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| Platelet Count | Platelet count at Baseline and Months 12, 24 and 36 |
| Baseline and Months 12, 24 and 36 |
| 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 |
| Pontificia Universidad Catolica de Chile | Santiago | Chile |
| Rambam Medical Center | Haifa | 31096 | Israel |
| Shaare Zedek Medical Center | Jerusalem | Israel |
| Morningside Medi-Clinic | Morningside | 2196 | South Africa |
| 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 |
| Derived |
| Pastores GM, Shankar SP, Petakov M, Giraldo P, Rosenbaum H, Amato DJ, Szer J, Chertkoff R, Brill-Almon E, Zimran A. Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 18. |
| Physician Decision |
|
| BG002 | Switchover | Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose |
| 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 |
|
| OG002 |
| Switchover |
Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose. |
|
|
| Secondary | Liver Volume | Liver volume measured by MRI | Intent to treat. In the Switchover group, two patients did not have MRI . | Posted | Mean | Standard Deviation | mL | Liver volume at Baseline and Months 12, 24 and 36 |
|
|
|
| Other Pre-specified | Spleen Volume Multiples of Normal (MN) | Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg) | Intent to treat. In the Switchover group, two patients did not have MRI and one patient was splenectomized. | Posted | Mean | Standard Deviation | Multiples of Normal Spleen Volume | Baseline and Months 12, 24, and 36 |
|
|
|
| Other Pre-specified | Liver Volume Multiples of Normal (MN) | Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg). | Intent to treat. In the Switchover group, two patients did not have MRI. | Posted | Mean | Standard Deviation | Multiples of Normal Liver Volume | Baseline and Months 12, 24 and 36 |
|
|
|
| Secondary | Hemoglobin | Posted | Mean | Standard Deviation | mg/dL | Hemoglobin at Baseline and Months 12, 24 and 36 |
|
|
|
| Secondary | Platelet Count | Posted | Mean | Standard Deviation | Platelets per cubic millimeter | Platelet count at Baseline and Months 12, 24 and 36 |
|
|
|
| 2 |
| 12 |
| 12 |
| 12 |
| EG001 | Naive 60 Units/kg | Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with taliglucerase alfa 60 units/kg | 2 | 14 | 12 | 14 |
| EG002 | Switchover | Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose | 3 | 18 | 17 | 18 |
|
| AUTOIMMUNE THROMBOCYTOPENIA | Blood and lymphatic system disorders | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| HAEMANGIOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment | pain due to hemangioma left knee - Not Related |
|
| HEAD INJURY | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment | Head trauma - Not Related |
|
| OSTEONECROSIS | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment | Right acetabular necrosis - Not Related |
|
| PNEUMOTHORAX TRAUMATIC | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| PULMONARY EMBOLISM | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| RENAL STONE REMOVAL | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| TONSILLECTOMY | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| TOOTH EXTRACTION | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| VOCAL CORD POLYPECTOMY | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment | Not Related |
|
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| DIARRHOEA | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| ENDODONTIC PROCEDURE | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment |
|
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| ERYTHEMA | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| FATIGUE | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| GASTROENTERITIS | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| HEADACHE | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| HYPERTENSION | Vascular disorders | MedDRA 15.0 | Systematic Assessment |
|
| LIMB INJURY | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| MUSCULOSKELETAL PAIN | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| NASOPHARYNGITIS | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| OROPHARYNGEAL PAIN | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| PAIN | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| PHARYNGITIS | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| PRURITUS | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| PYREXIA | General disorders | 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 |
|
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| VOMITING | Gastrointestinal 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 |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
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| Month 24 |
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| Month 36 |
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| Month 24 |
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| Month 36 |
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| Month 24 |
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| Month 36 |
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| Month 24 |
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| Month 36 |
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| Month 24 |
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| Month 36 |
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