Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
Condition: Gaucher Disease, Type 1
MeSH terms: Gaucher Disease
Study type: Interventional
Phase: Phase 4

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.
    

Intervention name(s): VPRIV
Intervention type(s): Drug
Intervention description(s): Safety, pharmacokinetics, and efficacy of rapid intravenous infusion of velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease
Other intervention name(s): Velaglucerase Alfa
Intervention model: Single Group Assignment
Intervention 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)
Primary purpose: Treatment