Phase 3
N=95
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
Gaucher Disease, Type 1
Bottom Line
View on ClinicalTrials.gov: NCT00635427 ↗Enrolled (actual)
95
Serious AEs
16.8%
Results posted
Jan 2014
Primary outcome: Primary: Overall Summary of Treatment Emergent Adverse Events — 3; 1; 3; 38 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- VPRIV® (Biological)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Dec 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Summary of Treatment Emergent Adverse Events |
3; 1; 3; 38; 15; 35 | — |
| SECONDARY Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group |
2.75; 2.00; -0.05 | — |
| SECONDARY Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group |
87.85; 160.94; 9.03 | — |
| SECONDARY Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group |
-1.206; -1.688; -0.026 | — |
| SECONDARY Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group |
-64.49; -63.82; -8.04 | — |
Summary
The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.
Eligibility Criteria
Inclusion Criteria
- The patient has completed study TKT032 or TKT034, or study HGT-GCB-039.
- Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study.
- Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the investigator.
- The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
- The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator
Exclusion Criteria
- The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted.
- The patient is pregnant or lactating.
- The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.
- The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.).
- The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator
Data sourced from ClinicalTrials.gov (NCT00635427). 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.