Table 2 Overview of the taliglucerase alfa clinical studies
| 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 |
