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NCT04120506

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

Completed Phase 4 Last updated 28 October 2022
What this trial tests

Phase 4 trial testing VPRIV in Gaucher Disease, Type 1 in 15 participants. Completed in 1 January 2022.

Timeline
10 January 2016
Primary endpoint
20 January 2017
1 January 2022

Quick facts

Lead sponsorShaare Zedek Medical Center
PhasePhase 4
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment15
Start date10 January 2016
Primary completion20 January 2017
Estimated completion1 January 2022

Drugs / interventions tested

Conditions studied

Sponsor

Shaare Zedek Medical Center

Who can join

Adults 6 to 75, any sex, with Gaucher Disease, Type 1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

Verify or expand the search:

Other trials of VPRIV

Trials testing the same drug.

Other recruiting trials for Gaucher Disease, Type 1

Currently open trials in the same condition.

Other Shaare Zedek Medical Center trials

Trials by the same sponsor.

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Data sources for this page

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