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Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Primary Purpose

Gaucher Disease, Type 1

Status
Completed
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
VPRIV
Sponsored by
Shaare Zedek Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease, Type 1

Eligibility Criteria

6 Years - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Aged 18-75 years, non-splenectomized Enzymatic diagnosis & molecular analysis indicative of type 1 Gaucher disease Receiving VPRIV for at least 6 infusions (3 months) prior to Baseline at a constant dose and frequency and without clinically significant AEs including allergic reactions

Exclusion Criteria:

  • Experience of a clinically significant AE to VPRIV at any time in the past Existence of a clinically significant co-morbidity

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Rapid infusion of Vpriv

    Arm Description

    : 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

    Outcomes

    Primary Outcome Measures

    Incidence of change from baseline in blood pressure for rapid infusion-1
    measured by blood pressure pre and post infusion
    Incidence of change from baseline in heart rate for rapid infusion-1
    measured by heart rate pre and post infusion
    Incidence of change from baseline in temperature for rapid infusion-1
    measured by temperature pre and post infusion

    Secondary Outcome Measures

    Non deterioration in Gaucher manifestations- stability in platelet counts
    Efficacy secondary endpoints are non-deterioration (defined as stability) in platelet count
    Non deterioration in Gaucher manifestations- stability in hemaglobin count
    Efficacy secondary endpoints are non-deterioration (defined as stability) in hemaglobin count
    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)
    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)
    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.

    Full Information

    First Posted
    August 10, 2018
    Last Updated
    October 27, 2022
    Sponsor
    Shaare Zedek Medical Center
    Collaborators
    Shire
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04120506
    Brief Title
    Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
    Official Title
    Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    October 2022
    Overall Recruitment Status
    Completed
    Study Start Date
    January 10, 2016 (Actual)
    Primary Completion Date
    January 20, 2017 (Actual)
    Study Completion Date
    January 1, 2022 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Shaare Zedek Medical Center
    Collaborators
    Shire

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Product Manufactured in and Exported from the U.S.
    Yes
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    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.
    Detailed Description
    Every 6 months, patients will be required to come for routine checkups at SZMC, where the following tests will be performed: Complete Blood Count (CBC) Routine serum biochemistry including liver function tests (LFTs) Plasma biomarker lyso Gb-1 Height & weight & calculation of BMI Physical examination and medical history elicited including concomitant medications Ultrasound for spleen and liver volumes In addition, the following tests will be performed at 12, 24 and 36 months: Echocardiography Electrocardiogram (ECG) Urinalysis HRQoL questionnaire (TBD) 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Gaucher Disease, Type 1

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Single Group Assignment
    Model Description
    Objective: To show non-inferiority of rapid (defined as 10') intravenous (IV) infusion versus standard IV infusion rate (60') of VPRIV with regard to safety, pharmacokinetic(PK) profile, and efficacy in patients who have been receiving VPRIV for a minimum of 3 months at a constant dosage.
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    15 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Rapid infusion of Vpriv
    Arm Type
    Experimental
    Arm Description
    : 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
    Intervention Type
    Drug
    Intervention Name(s)
    VPRIV
    Other Intervention Name(s)
    Velaglucerase Alfa
    Intervention Description
    Safety, pharmacokinetics, and efficacy of rapid intravenous infusion of velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease
    Primary Outcome Measure Information:
    Title
    Incidence of change from baseline in blood pressure for rapid infusion-1
    Description
    measured by blood pressure pre and post infusion
    Time Frame
    9 months
    Title
    Incidence of change from baseline in heart rate for rapid infusion-1
    Description
    measured by heart rate pre and post infusion
    Time Frame
    9 months
    Title
    Incidence of change from baseline in temperature for rapid infusion-1
    Description
    measured by temperature pre and post infusion
    Time Frame
    9 months
    Secondary Outcome Measure Information:
    Title
    Non deterioration in Gaucher manifestations- stability in platelet counts
    Description
    Efficacy secondary endpoints are non-deterioration (defined as stability) in platelet count
    Time Frame
    9 months
    Title
    Non deterioration in Gaucher manifestations- stability in hemaglobin count
    Description
    Efficacy secondary endpoints are non-deterioration (defined as stability) in hemaglobin count
    Time Frame
    9 months
    Title
    Non deterioration in Gaucher manifestations- measured by liver volume
    Description
    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
    Title
    Non deterioration in Gaucher manifestations- measured by spleen volumes
    Description
    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
    Title
    Non deterioration in Gaucher manifestations- measured by lack of elevated biomarker- Lyso-GB1
    Description
    Efficacy secondary endpoints are non-deterioration (defined as stability) by lack of sustained increases in the biomarkers.
    Time Frame
    9 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    6 Years
    Maximum Age & Unit of Time
    75 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Aged 18-75 years, non-splenectomized Enzymatic diagnosis & molecular analysis indicative of type 1 Gaucher disease Receiving VPRIV for at least 6 infusions (3 months) prior to Baseline at a constant dose and frequency and without clinically significant AEs including allergic reactions Exclusion Criteria: Experience of a clinically significant AE to VPRIV at any time in the past Existence of a clinically significant co-morbidity
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Ari Zimran
    Organizational Affiliation
    Ari Zimran - Shaare Zedek
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    Citations:
    PubMed Identifier
    29989200
    Citation
    Zimran A, Revel-Vilk S, Becker-Cohen M, Chicco G, Arbel N, Rolfs A, Szer J. Rapid intravenous infusion of velaglucerase-alfa in adults with type 1 Gaucher disease. Am J Hematol. 2018 Sep;93(9):E246-E248. doi: 10.1002/ajh.25205. Epub 2018 Aug 9. No abstract available.
    Results Reference
    result

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    Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

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