A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
This study has been completed.
Sponsor:
Protalix
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01411228
First received: August 4, 2011
Last updated: September 9, 2014
Last verified: September 2014
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Purpose
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).
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease |
Drug: Taliglucerase alfa |
Phase 3 |
Access to an investigational treatment associated with this study is no longer available outside the clinical trial. More info ...
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment |
| Official Title: | A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Gaucher disease
Schindler disease
succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics:
Gaucher's Disease
Drug Information available for:
Taliglucerase alfa
U.S. FDA Resources
Further study details as provided by Protalix:
Primary Outcome Measures:
- Hemoglobin [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: No ]median percentage and the interquartile range for change from baseline in haemoglobin
Secondary Outcome Measures:
- Chitotriosidase [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: No ]Percent change from baseline in chitotriosidase
- Spleen and liver volume [ Time Frame: at 1 and 2 years ] [ Designated as safety issue: No ]Percent change in spleen and liver volume measured by MRI (or ultrasound)
- Platelet count [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: No ]Percent change from baseline in platelet count
- Anti taliglucerase alfa antibodies [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: Yes ]Occurrence of positive antibody response
| Enrollment: | 15 |
| Study Start Date: | September 2011 |
| Study Completion Date: | August 2014 |
| Primary Completion Date: | July 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 60 Units/kg |
Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months
Other Name: prGCD, plant cell expressed glucocerebrosidase
|
| Experimental: 30 Units/kg |
Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months
Other Name: prGCD, plant cell expressed glucocerebrosidase
|
Eligibility| Ages Eligible for Study: | 2 Years to 18 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Successful completion of Protocol PB-06-002 or PB-06-005
- The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent
Exclusion Criteria:
- Currently taking another investigational drug for any condition.
- Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01411228
Please refer to this study by its ClinicalTrials.gov identifier: NCT01411228
Locations
| Israel | |
| Shaare Zedek Medical Center | |
| Jerusalem, Israel | |
| Paraguay | |
| Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C) | |
| Barrio Asuncion, Paraguay | |
| South Africa | |
| Morningside Medi-Clinic | |
| Morningside, South Africa | |
Sponsors and Collaborators
Protalix
Investigators
| Principal Investigator: | Ari Zimran, MD | Shaare Zedek Medical Center |
More Information
No publications provided
| Responsible Party: | Protalix |
| ClinicalTrials.gov Identifier: | NCT01411228 History of Changes |
| Other Study ID Numbers: | PB-06-006 |
| Study First Received: | August 4, 2011 |
| Last Updated: | September 9, 2014 |
| Health Authority: | United States: Food and Drug Administration Israel: Ministry of Health |
Keywords provided by Protalix:
|
Gaucher disease pediatrics |
Additional relevant MeSH terms:
|
Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors |
Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Sphingolipidoses |
ClinicalTrials.gov processed this record on March 03, 2015