A Phase 4 interventional study of VPRIV in Gaucher Disease, Type 1, sponsored by Shaare Zedek Medical Center. Completed. Open to participants aged 6 Years to 75 Years. Per ClinicalTrials.gov, last updated 2022-10-28.
Sponsored by Shaare Zedek Medical Center · Phase 4, Interventional, and Treatment
Background: In order to allow our satisfied patients, who have successfully completed 24 months of rapid intravenous infusion of Velaglucerase alfa (VPRIV), to continue with the 10 minutes IV therapy, the clinical trial framework must be extended; and this extension is important for the assessment of long term benefit (up to 5 years) of this regimen of administration of Velaglucerase alfa..
Suggested trial: An additional 36 months home therapy follow up of safety and efficacy of rapid intravenous infusion of Velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease.
Patients must have completed the prior 4 parts / 24 months of the protocol before enrolling into this extension phase ("Part 5") and have provided a new consent before entering PART 5 of the study.
Patients must not have experienced clinically significant AEs, including allergic reactions, in any of the prior study parts of this protocol to be eligible to participate, and have maintained stability in the key disease features.
All infusions of 10' will be given in the context of home therapy. "Clinically significant" AEs will be determined by the PI using standard description of AEs as previously described at phase 3, and if necessary will support withdrawal of the patient from the study.
Every 6 months, patients will be required to come for routine checkups at SZMC, where the following tests will be performed:
In addition, the following tests will be performed at 12, 24 and 36 months:
At each home visit, the following assessments will be performed by the study nurse:
Queries regarding AEs and changes in clinically relevant Gaucher parameters as described by the patient (e.g., bone pain), inter-current illnesses, etc.
Patients will be required to complete the End-of-study visit, including the final infusion at 10', at SZMC. This final visit will include in addition to the usual safety and efficacy assessments and routine tests, (mentioned above) also, DEXA and anti-drug antibodies.
In addition, we would perform a 4th PK measurement at end of the extension period.
171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.
This study's enrollment of 15 is below the median of 20 across 98 interventional studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →Shaare Zedek Medical Center is the lead sponsor of 191 studies on the registry; 17 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
: Infusions at Baseline and during step-wise rate increases and End-of-study will be performed in the Shaare Zedek Medical Center (SZMC) by the Study Nurse who will monitor vital signs (see below) for a total of 8 visits at SZMC. Home therapy will be approved if the patient so desires for 5 infusions in Phase 1 and for the first 5 infusions in Phase 3. All routine hematological and biochemical tests will be performed in the SZMC clinical labs. Abdominal quantitative MR Imaging (MRI) for spleen and liver volumes will be performed at SZMC
Drug: VPRIV
Safety, pharmacokinetics, and efficacy of rapid intravenous infusion of velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease
Also known as: Velaglucerase Alfa
Incidence of change from baseline in blood pressure for rapid infusion-1
measured by blood pressure pre and post infusion
Time frame: 9 months
Incidence of change from baseline in heart rate for rapid infusion-1
measured by heart rate pre and post infusion
Time frame: 9 months
Incidence of change from baseline in temperature for rapid infusion-1
measured by temperature pre and post infusion
Time frame: 9 months
Non deterioration in Gaucher manifestations- stability in platelet counts
Efficacy secondary endpoints are non-deterioration (defined as stability) in platelet count
Time frame: 9 months
Non deterioration in Gaucher manifestations- stability in hemaglobin count
Efficacy secondary endpoints are non-deterioration (defined as stability) in hemaglobin count
Time frame: 9 months
Non deterioration in Gaucher manifestations- measured by liver volume
Efficacy secondary endpoints are non-deterioration (defined as stability) in organ volumes of liver volume (as previously defined in the TKT034 clinical trial)
Time frame: 9 months
Non deterioration in Gaucher manifestations- measured by spleen volumes
Efficacy secondary endpoints are non-deterioration (defined as stability) in organ volumes of spleen volume (as previously defined in the TKT034 clinical trial)
Time frame: 9 months
Non deterioration in Gaucher manifestations- measured by lack of elevated biomarker- Lyso-GB1
Efficacy secondary endpoints are non-deterioration (defined as stability) by lack of sustained increases in the biomarkers.
Time frame: 9 months
No study locations are listed for this record.
Plan to share: No
This study is completed, as verified in Oct 2022. You cannot join it, but the record below documents what was studied.
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Shaare Zedek Medical Center