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

Tracking Information

• First Submitted Date: August 4, 2011
• First Posted Date: August 8, 2011
• Results First Submitted Date: November 6, 2015
• Results First Posted Date: January 18, 2016
• Last Update Posted Date: September 7, 2018
• Study Start Date: September 2011
• Actual Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 11, 2015)

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.

Original Primary Outcome Measures (submitted: August 4, 2011)

Hemoglobin [ Time Frame: Every 3 months for 2 years ]

median percentage and the interquartile range for change from baseline in haemoglobin

Current Secondary Outcome Measures (submitted: December 11, 2015)

• 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.
• 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.
• 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.
• 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.

Original Secondary Outcome Measures (submitted: August 4, 2011)

• Chitotriosidase [ Time Frame: Every 3 months for 2 years ]
  Percent change from baseline in chitotriosidase
• Spleen and liver volume [ Time Frame: at 1 and 2 years ]
  Percent change in spleen and liver volume measured by MRI (or ultrasound)
• Platelet Count [ Time Frame: Every 3 months for 2 years ]
  Percent change from baseline in platelet count
• Anti taliglucerase alfa antibodies [ Time Frame: Every 3 months for 2 years ]
  Occurrence of positive antibody response

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
• Official Title: A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
• 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).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
• Condition: Gaucher Disease

Intervention

Drug: Taliglucerase alfa

Taliglucerase alfa for infusion every two weeks for 24 months

Other Name: prGCD, plant cell expressed glucocerebrosidase

Study Arms

• Experimental: 60 Units/kg
  Intervention: Drug: Taliglucerase alfa
• Experimental: 30 Units/kg
  Intervention: Drug: Taliglucerase alfa

Publications *

• 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 4, 2011): 15
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: August 2014
• Actual Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 18 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Israel, Paraguay, South Africa

Administrative Information

• NCT Number: NCT01411228
• Other Study ID Numbers: PB-06-006
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Pfizer
• Study Sponsor: Pfizer
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Pfizer
• Verification Date: September 2018