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

NCT04120506 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2022-10-28

No results posted yet for this study

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.

Conditions

  • Gaucher Disease, Type 1

Interventions

DRUG

VPRIV

Safety, pharmacokinetics, and efficacy of rapid intravenous infusion of velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease

Sponsors & Collaborators

  • Shire

    collaborator INDUSTRY

  • Shaare Zedek Medical Center

    lead OTHER

Principal Investigators

  • Ari Zimran · Ari Zimran - Shaare Zedek

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-10
Primary Completion
2017-01-20
Completion
2022-01-01
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04120506 on ClinicalTrials.gov