You are here

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

Last updated on November 17, 2019

FOR MORE INFORMATION
Study Location
Shaare Zedek Medical Center
Jerusalem, , Israel
Contact
1-800-718-1021
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

NEED INFO?

Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative

Pfizer Clinical Trials Contact Center

1-800-718-1021

[email protected]

call now

Try a new search

Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your city or zip code to find studies near you.
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 SummaryThis 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 DescriptionNot 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 DateMay 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 ?-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 CountriesUnited States
 
Administrative Information
NCT Number  ICMJE NCT01132690
Other Study ID Numbers  ICMJE PB-06-005
Has Data Monitoring CommitteeYes
U.S. FDA-regulated ProductNot Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible PartyPfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS AccountPfizer
Verification DateSeptember 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 the study website. Please see the references below:

BY PHONE

Pfizer Clinical Trials Contact Center

1-800-718-1021

BY EMAIL

Contact

[email protected]

Call Now