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Table 2 Overview of the taliglucerase alfa clinical studies

From: Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease

Studya Design Patient population N b Taliglucerase alfa
treatment
Duration
(Months)
Key efficacy findings Key safety findings
PB-06-001
(NCT00376168)
Zimran, 2011 [15]
• Multicentre
• Double-blind
• Randomized
• Adults ≥18 years
• Treatment-naive
31 • Parallel dose
• 30 U/kg or 60 U/kg
9 • Significant improvements in visceral and haematologic parameters and chitotriosidase activity • TEAEs mild/moderate and transient
• No SAEs
PB-06-002
(NCT00712348)
Pastores, 2014 [16]
• Multicentre
• Open-label
• Switchover
• Adults (n = 26) and children (n = 5)  > 2 years
• Previously treated with imiglucerase
31 • Same dose as previously treated with imiglucerase (9–60 U/kg) 9 • Stability of visceral, haematologic, and biomarker parameters • TEAEs mild/moderate and transient
• No SAEs related to treatment
• No discontinuations due to drug-related AEs
PB-06-003
(NCT0070593)
Zimran, 2016 [18]; Pastores, 2016 [19]
• Multicentre
• Double-blind and open-label
• Extension
• Adults from PB-06-001 or PB-06-002
• Treatment-naïve (n = 26) or treatment-switched (n = 19)
45 • Same dose as in previous study 30 • Treatment-naïve: Visceral and biomarker parameters and platelets improved continuously; haemoglobin reached normal levels and stabilized
• Treatment-switched: Continued stability or improvement
• TEAEs mild/moderate and transient
• All SAEs not related to treatment
PB-06-005
(NCT01132690)
Zimran, 2015 [17]
• Multicentre
• Double-blind
• Randomized
• Children 2–< 18 years
• Treatment-naïve
11 • Parallel dose
• 30 U/kg or 60 U/kg
12 • Clinically significant improvements in visceral, haematologic, and biomarker parameters
• Trend toward improvement in exploratory parameters of growth and development
• Treatment well tolerated
• Most AEs mild/moderate, transient, and not treatment-related
• No deaths
• No clinically meaningful laboratory abnormalities
PB-06-006
(NCT01411228)
Zimran, 2016 [20]
• Multicentre
• Double-blind and open-label
• Extension
• Children from PB-06-005 (originally treatment-naïve; N = 10)
• Children from PB-06-002 (treatment-switched; N = 5)
15 • Continued parallel doses at 30 U/kg or 60 U/kg
• Continued at previous dose in PB-06-002
24 • Continued improvement (naïve) or stability (switched) in visceral, haematologic, and biomarker parameters
• Continued improvement in exploratory parameters of growth and development
• Treatment well tolerated
• All AEs mild/moderate
• No deaths
• No discontinuations due to AE
• No clinically meaningful laboratory abnormalities
PB-06-007
(NCT01422187)
Zimran, 2016 [21]
• Multicentre
• Open-label
• Extension
• Originally treatment-naïve adults from PB-06-003 19 • Continued parallel doses at 30 U/kg or 60 U/kg 21 • Visceral and biomarker parameters and platelets improved continuously; haemoglobin remained stable • Most AEs mild/moderate and not treatment-related
• Most laboratory values normal
  1. aStudy PB-06-004 was an open-label expanded access study; results will be reported separately
  2. bNumber of patients treated
  3. AEs, adverse events; SAEs, serious adverse events; TEAEs, treatment-emergent adverse events