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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01411228
Other study ID # PB-06-006
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date September 2011
Est. completion date August 2014

Study information

Verified date September 2018
Source Pfizer
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa).


Recruitment information / eligibility

Status Completed
Enrollment 15
Est. completion date August 2014
Est. primary completion date July 2014
Accepts healthy volunteers No
Gender All
Age group 2 Years to 18 Years
Eligibility Inclusion Criteria:

- Successful completion of Protocol PB-06-002 or PB-06-005

- The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent

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 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months

Locations

Country Name City State
Israel Shaare Zedek Medical Center Jerusalem
Paraguay Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C) Barrio Asuncion
South Africa Morningside Medi-Clinic Morningside

Sponsors (1)

Lead Sponsor Collaborator
Pfizer

Countries where clinical trial is conducted

Israel,  Paraguay,  South Africa, 

Outcome

Type Measure Description Time frame Safety issue
Primary Hemoglobin Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. Baseline, months 9, 12 and 24
Secondary Chitotriosidase Chitotriosidase. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. Baseline, months 9, 12 and 24
Secondary Spleen Volume Spleen volume measured by MRI (or ultrasound). Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. Baseline, months 12 and 24
Secondary Platelet Count Platelet count. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. Baseline, months 9, 12, 24 and 33-36
Secondary Liver Volume Liver volume by MRI or Ultrasound. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. Baseline, months 12 and 24
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