A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Gaucher Disease Clinical Trial

Official title:

A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01411228
• Other study ID #: PB-06-006
• Status: Completed
• Phase: Phase 3
• Start date: September 2011
• Est. completion date: August 2014

Study information

• Verified date: September 2018
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• 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.

Related Conditions & MeSH terms

• Gaucher Disease

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