Back to resultsA Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Primary Purpose
Gaucher Disease
Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Taliglucerase alfa
Sponsored by
About this trial
This is an interventional treatment trial for Gaucher Disease focused on measuring gaucher disease, pediatric
Eligibility Criteria
Inclusion Criteria:
- Males and females 2 to <18 years old.
- Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
- Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
- Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
- Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).
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 unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
- Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
- History of allergy to carrots.
- Presence of HIV, HBsAg or hepatitis C infections.
- Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
- 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.
Sites / Locations
- Shaare Zedek Medical Center
- Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
- Morningside Medi-Clinic
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
30 units/kg
60 units/kg
Arm Description
Outcomes
Primary Outcome Measures
Hemoglobin
median and interquartile range for change from baseline in haemoglobin
Secondary Outcome Measures
Chitotriosidase
Percent change from baseline in chitotriosidase
Spleen Volume
Spleen volume measured by MRI
Platelet Count
Mean and standard deviation of platelet count per cubic mm
Chemokine (C-C Motif) Ligand 18 (CCL18)
Percent change from baseline in CCL18
Liver Volume
Liver volume measured by MRI
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01132690
Brief Title
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Official Title
A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Study Type
Interventional
2. Study Status
Record Verification Date
September 2018
Overall Recruitment Status
Completed
Study Start Date
August 2010 (undefined)
Primary Completion Date
May 2012 (Actual)
Study Completion Date
July 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease
Keywords
gaucher disease, pediatric
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
30 units/kg
Arm Type
Experimental
Arm Title
60 units/kg
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Taliglucerase alfa
Other Intervention Name(s)
prGCD, plant cell expressed glucocerebrosidase
Intervention Description
Taliglucerase alfa for infusion every two weeks for 12 months
Primary Outcome Measure Information:
Title
Hemoglobin
Description
median and interquartile range for change from baseline in haemoglobin
Time Frame
Every 3 months for 12 months
Secondary Outcome Measure Information:
Title
Chitotriosidase
Description
Percent change from baseline in chitotriosidase
Time Frame
Every 3 months for 12 months
Title
Spleen Volume
Description
Spleen volume measured by MRI
Time Frame
Baseline and Month 12
Title
Platelet Count
Description
Mean and standard deviation of platelet count per cubic mm
Time Frame
Baseline and 12 months
Title
Chemokine (C-C Motif) Ligand 18 (CCL18)
Description
Percent change from baseline in CCL18
Time Frame
Every 3 months for 12 months
Title
Liver Volume
Description
Liver volume measured by MRI
Time Frame
Baseline and Month 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Males and females 2 to <18 years old.
Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).
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 unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
History of allergy to carrots.
Presence of HIV, HBsAg or hepatitis C infections.
Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
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.
Facility Information:
Facility Name
Shaare Zedek Medical Center
City
Jerusalem
Country
Israel
Facility Name
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
City
Barrio Sajonia Asunción
Country
Paraguay
Facility Name
Morningside Medi-Clinic
City
Morningside
ZIP/Postal Code
2196
Country
South Africa
12. IPD Sharing Statement
Citations:
PubMed Identifier
25453586
Citation
Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Elstein D, Paz A, Brill-Almon E, Chertkoff R. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease. Blood Cells Mol Dis. 2015 Jan;54(1):9-16. doi: 10.1016/j.bcmd.2014.10.002. Epub 2014 Nov 7.
Results Reference
derived
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A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
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