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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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NCT01132690

Last updated on October 9, 2018
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FOR MORE INFORMATION
Study Location
Shaare Zedek Medical Center
Jerusalem, , Israel
See other locations
Contact
1-800-718-1021
[email protected]
Related Links
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Gaucher Disease
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
2-17 years
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- Males and females 2 to

- 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details

- 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.

NCT01132690
Pfizer
Completed
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

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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
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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
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.
 
Completed
11
10
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 ?-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.
Sexes Eligible for Study: All
2 Years to 17 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Israel,   Paraguay,   South Africa
United States
 
NCT01132690
PB-06-005
Yes
Not Provided
Not Provided
Pfizer
Pfizer
Not Provided
Not Provided
Pfizer
September 2018

ICMJE     Data element required by the

International Committee of Medical Journal Editors and the
World Health Organization ICTRP

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting thte study website. To get updates and notications about this trail, sign up using the form below.

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1-800-718-1021

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[email protected]



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