Phase 3
Completed N=19
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Source: ClinicalTrials.gov NCT01422187 ↗Enrolled (actual)
19
Serious AEs
10.5%
Results posted
Dec 2015
Primary outcomePrimary: Spleen Volume — 2032.0; 1960.0; 2321.7; 1522.6 Milliliters
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Spleen Volume |
2032.0; 1960.0; 2321.7; 1522.6; 1063.7; 1677.9 | — |
| SECONDARY Liver Volume |
3103.4; 2536.1; 2488.6; 2619.3; 2155.2; 2048.0 | — |
| SECONDARY Platelet Count |
70975; 70067; 69500; 86613; 137556; 88000 | — |
| SECONDARY Hemoglobin |
12.3; 11.6; 12.5; 13.9; 14.2; 14.3 | — |
Eligibility Criteria
Inclusion Criteria
- Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003
- The subject signs an informed consent
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT01422187). 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.