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

• ClinicalTrials.gov Identifier: NCT01411228
• Recruitment Status: Completed
• First Posted: August 8, 2011
• Results First Posted: January 18, 2016
• Last Update Posted: September 7, 2018
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Study Description

Brief 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).

Condition or disease	Intervention/treatment	Phase
Gaucher Disease	Drug: Taliglucerase alfa	Phase 3

Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial.   More info ...

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 15 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• Official Title: A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
• Study Start Date: September 2011
• Actual Primary Completion Date: July 2014
• Actual Study Completion Date: August 2014

Arms and Interventions

Arm	Intervention/treatment
Experimental: 60 Units/kg	Drug: Taliglucerase alfa; Taliglucerase alfa for infusion every two weeks for 24 months; Other Name: prGCD, plant cell expressed glucocerebrosidase
Experimental: 30 Units/kg	Drug: Taliglucerase alfa; Taliglucerase alfa for infusion every two weeks for 24 months; Other Name: prGCD, plant cell expressed glucocerebrosidase

Outcome Measures

Primary Outcome Measures :

1. Hemoglobin [ Time Frame: Baseline, months 9, 12 and 24 ]
   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.

Secondary Outcome Measures :

1. Chitotriosidase [ Time Frame: Baseline, months 9, 12 and 24 ]
   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.
2. Spleen Volume [ Time Frame: Baseline, months 12 and 24 ]
   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.
3. Platelet Count [ Time Frame: Baseline, months 9, 12, 24 and 33-36 ]
   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.
4. Liver Volume [ Time Frame: Baseline, months 12 and 24 ]
   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.

Eligibility Criteria

• Ages Eligible for Study: 2 Years to 18 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Locations

Israel

Shaare Zedek Medical Center

Jerusalem, Israel

Paraguay

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)

Barrio Asuncion, Paraguay

South Africa

Morningside Medi-Clinic

Morningside, South Africa

Sponsors and Collaborators

Pfizer

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Cooper PA, Varughese S, Giraldo P, Petakov M, Tan ES, Chertkoff R. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase. Blood Cells Mol Dis. 2018 Feb;68:163-172. doi: 10.1016/j.bcmd.2016.10.005. Epub 2016 Oct 20.

Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT01411228
• Other Study ID Numbers: PB-06-006
• First Posted: August 8, 2011
• Results First Posted: January 18, 2016
• Last Update Posted: September 7, 2018
• Last Verified: September 2018

Keywords provided by Pfizer:

Gaucher disease; pediatrics

Additional relevant MeSH terms:

Gaucher Disease; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Lipid Metabolism Disorders