A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01132690
First received: May 26, 2010
Last updated: April 25, 2014
Last verified: April 2014

May 26, 2010
April 25, 2014
August 2010
May 2012   (final data collection date for primary outcome measure)
Hemoglobin [ Time Frame: Every 3 months for 12 months ] [ Designated as safety issue: No ]
median percentage and the interquartile range for change from baseline in haemoglobin
Same as current
Complete list of historical versions of study NCT01132690 on ClinicalTrials.gov Archive Site
  • Chitotriosidase or CCL18 [ Time Frame: Every 3 months for 12 months ] [ Designated as safety issue: No ]
    Percent change from baseline in chitotriosidase or CCL18
  • Spleen and liver volume [ Time Frame: Baseline and Month 12 ] [ 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 12 months ] [ Designated as safety issue: No ]
    Percent change from baseline in platelet count
  • Anti-taliglucerase alfa antibodies [ Time Frame: Every 3 months for 12 months ] [ Designated as safety issue: Yes ]
    Occurrence of positive antibody response
Same as current
Not Provided
Not Provided
 
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

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.

Not Provided
Interventional
Phase 4
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Gaucher Disease
Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 12 months
Other Name: prGCD, plant cell expressed glucocerebrosidase
  • Experimental: 30 units/kg
    Intervention: Drug: Taliglucerase alfa
  • Experimental: 60 units/kg
    Intervention: Drug: Taliglucerase alfa
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
11
July 2012
May 2012   (final data collection date for primary outcome measure)

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.
Both
2 Years to 17 Years
No
Contact information is only displayed when the study is recruiting subjects
Paraguay,   South Africa,   Israel
 
NCT01132690
PB-06-005
Yes
Protalix
Protalix
Not Provided
Study Director: Ari Zimran, MD Shaare Zedek Medical Center
Protalix
April 2014

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