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A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 60 Units/kg | Experimental |
| |
| 30 Units/kg | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Taliglucerase alfa | Drug | Taliglucerase alfa for infusion every two weeks for 24 months |
|
| Measure | Description | Time Frame |
|---|---|---|
| Hemoglobin | Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 9, 12 and 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Chitotriosidase | Chitotriosidase. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 9, 12 and 24 |
| Spleen Volume |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Shaare Zedek Medical Center | Jerusalem | Israel | ||||
| Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C) |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27839981 | Derived | Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Cooper PA, Varughese S, Giraldo P, Petakov M, Tan ES, Chertkoff R. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naive or previously treated with imiglucerase. Blood Cells Mol Dis. 2018 Feb;68:163-172. doi: 10.1016/j.bcmd.2016.10.005. Epub 2016 Oct 20. | |
| 26053270 |
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| ID | Title | Description |
|---|---|---|
| FG000 | 60 Units/kg | Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months |
| FG001 | 30 Units/kg | Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months |
| FG002 | Switchover | Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 60 Units/kg | Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months |
| BG001 | 30 Units/kg | Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months |
| 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 | Hemoglobin | Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program | Posted | Median | Inter-Quartile Range | g/dL | Baseline, months 9, 12 and 24 |
|
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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 | 60 Units/kg | Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dengue Fever | Infections and infestations | MedDRA 15.0 | Systematic Assessment | (Grade 2 Dengue Fever), which required hospitalization, resolved in 5 weeks and was assessed as not related to study drug |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| LYMPHADENOPATHY | Blood and lymphatic system disorders | MedDRA 15.0 | 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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Spleen volume measured by MRI (or ultrasound). Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
| Baseline, months 12 and 24 |
| Platelet Count | Platelet count. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 9, 12, 24 and 33-36 |
| Liver Volume | Liver volume by MRI or Ultrasound. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 12 and 24 |
| Barrio Asunción |
| Paraguay |
| Morningside Medi-Clinic | Morningside | South Africa |
| Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015. |
| BG002 | Dose Adjusted | Taliglucerase alfa: Dose increased from 30 Units/kg to 45 or 60 Units/kg |
| 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 | Taliglucerase alfa: Maintained dose from PB-06-002 study |
|
|
| Secondary | Chitotriosidase | Chitotriosidase. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Chitotriosidase was not analyzed for the subjects in the Switchover group | Posted | Mean | Standard Deviation | nmol/mL*h | Baseline, months 9, 12 and 24 |
|
|
|
| Secondary | Spleen Volume | Spleen volume measured by MRI (or ultrasound). Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program | Posted | Mean | Standard Error | Milliliters | Baseline, months 12 and 24 |
|
|
|
| Secondary | Platelet Count | Platelet count. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program | Posted | Mean | Standard Error | Platelets/mm^3 | Baseline, months 9, 12, 24 and 33-36 |
|
|
|
| Secondary | Liver Volume | Liver volume by MRI or Ultrasound. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program | Posted | Mean | Standard Error | Milliliters | Baseline, months 12 and 24 |
|
|
|
| 1 |
| 5 |
| 5 |
| 5 |
| EG001 | 30 Units/kg | Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months | 0 | 5 | 4 | 5 |
| EG002 | Switchover | Taliglucerase alfa: Maintained dose from PB-06-002 study | 0 | 5 | 5 | 5 |
|
| EAR PAIN | Ear and labyrinth disorders | MedDRA 15.0 | Systematic Assessment |
|
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| DIARRHOEA | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| DENGUE FEVER | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| NASOPHARYNGITIS | Infections and infestations | 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 |
|
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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 |
| Month 12 |
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| Month 24 |
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| 24 Months |
|
| 33-36 Months |
|
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| Month 12 |
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| Month 24 |
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| Month 33-36 |
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| Month 24 |
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| Month 33-36 |
|