Phase 3
Completed N=15
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Source: ClinicalTrials.gov NCT01411228 ↗Enrolled (actual)
15
Serious AEs
6.7%
Results posted
Jan 2016
Primary outcomePrimary: Hemoglobin — 10.6; 11.2; 13.5; 11.9 g/dL
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Hemoglobin |
10.6; 11.2; 13.5; 11.9; 12.5; 13.9 | — |
| SECONDARY Chitotriosidase |
34968; 30783; 17416; 13983; 14677; 13823 | — |
| SECONDARY Spleen Volume |
1023.1; 1244.7; 313.0; 524.0; 759.0; 274.1 | — |
| SECONDARY Platelet Count |
99600; 182000; 164587; 172600; 221400; 177400 | — |
| SECONDARY Liver Volume |
991.7; 1236.5; 1345.6; 849.1; 1140.6; 1460.7 | — |
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.
Data sourced from ClinicalTrials.gov (NCT01411228). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.