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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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ClinicalTrials.gov Identifier: NCT01132690
Recruitment Status : Completed
First Posted : May 28, 2010
Results First Posted : November 11, 2014
Last Update Posted : October 5, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE May 26, 2010
First Posted Date  ICMJE May 28, 2010
Results First Submitted Date  ICMJE October 28, 2014
Results First Posted Date  ICMJE November 11, 2014
Last Update Posted Date October 5, 2018
Study Start Date  ICMJE August 2010
Actual Primary Completion Date May 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 10, 2014)
Hemoglobin [ Time Frame: Every 3 months for 12 months ]
median and interquartile range for change from baseline in haemoglobin
Original Primary Outcome Measures  ICMJE
 (submitted: May 26, 2010)
Hemoglobin [ Time Frame: Every 3 months for 12 months ]
median percentage and the interquartile range for change from baseline in haemoglobin
Change History Complete list of historical versions of study NCT01132690 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 10, 2014)
  • Chitotriosidase [ Time Frame: Every 3 months for 12 months ]
    Percent change from baseline in chitotriosidase
  • Spleen Volume [ Time Frame: Baseline and Month 12 ]
    Spleen volume measured by MRI
  • Platelet Count [ Time Frame: Baseline and 12 months ]
    Mean and standard deviation of platelet count per cubic mm
  • Chemokine (C-C Motif) Ligand 18 (CCL18) [ Time Frame: Every 3 months for 12 months ]
    Percent change from baseline in CCL18
  • Liver Volume [ Time Frame: Baseline and Month 12 ]
    Liver volume measured by MRI
Original Secondary Outcome Measures  ICMJE
 (submitted: May 26, 2010)
  • Chitotriosidase or CCL18 [ Time Frame: Every 3 months for 12 months ]
    Percent change from baseline in chitotriosidase or CCL18
  • Spleen and liver volume [ Time Frame: Baseline and Month 12 ]
    Percent change in spleen and liver volume measured by MRI (or ultrasound)
  • Platelet count [ Time Frame: Every 3 months for 12 months ]
    Percent change from baseline in platelet count
  • Anti-taliglucerase alfa antibodies [ Time Frame: Every 3 months for 12 months ]
    Occurrence of positive antibody response
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Official Title  ICMJE A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Gaucher Disease
Intervention  ICMJE Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 12 months
Other Name: prGCD, plant cell expressed glucocerebrosidase
Study Arms  ICMJE
  • Experimental: 30 units/kg
    Intervention: Drug: Taliglucerase alfa
  • Experimental: 60 units/kg
    Intervention: Drug: Taliglucerase alfa
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 10, 2013)
11
Original Estimated Enrollment  ICMJE
 (submitted: May 26, 2010)
10
Actual Study Completion Date  ICMJE July 2012
Actual Primary Completion Date May 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Israel,   Paraguay,   South Africa
Removed Location Countries United States
 
Administrative Information
NCT Number  ICMJE NCT01132690
Other Study ID Numbers  ICMJE PB-06-005
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Pfizer
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP