A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
1 other identifier
interventional
19
0 countries
N/A
Brief Summary
This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Aug 2011
Typical duration for phase_3
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2011
CompletedFirst Submitted
Initial submission to the registry
August 22, 2011
CompletedFirst Posted
Study publicly available on registry
August 23, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2014
CompletedResults Posted
Study results publicly available
December 14, 2015
CompletedApril 19, 2023
April 1, 2023
2.8 years
August 22, 2011
November 6, 2015
April 18, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Spleen Volume
Spleen volume measured by MRI
60 months
Secondary Outcomes (3)
Liver Volume
60 months
Platelet Count
60 months
Hemoglobin
60 months
Study Arms (2)
Taliglucerase alfa 30 units/kg
EXPERIMENTALSubjects randomized to receive 30 units/kg
Taliglucerase alfa 60 units/kg
EXPERIMENTALSubjects randomized to 60 units/kg
Interventions
Taliglucerase infusion every two weeks for 21 months
Eligibility Criteria
You may qualify if:
- Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003
- The subject signs an informed consent
You may not qualify if:
- Currently taking another investigational drug for any condition.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Pfizerlead
Related Publications (1)
Zimran A, Duran G, Giraldo P, Rosenbaum H, Giona F, Petakov M, Terreros Munoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R. Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naive patients with Gaucher disease. Blood Cells Mol Dis. 2019 Sep;78:14-21. doi: 10.1016/j.bcmd.2016.07.002. Epub 2016 Jul 18.
PMID: 27499018DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Glen Park
- Organization
- Target Health Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
August 22, 2011
First Posted
August 23, 2011
Study Start
August 1, 2011
Primary Completion
May 1, 2014
Study Completion
September 1, 2014
Last Updated
April 19, 2023
Results First Posted
December 14, 2015
Record last verified: 2023-04