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Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease

NCT03702361 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2021-03-19

No results posted yet for this study

Summary

During the past two years, the investigator has performed succsefully an IIR wherein patients with GD, previously treated with velaglucerase alfa ERT were gradually switched to a 10 minutes (rapid) administration of the same ERT. The success was expressed as safety (no clinically meaningful AEs, no antibodies detected, home therapy), efficacy ("lack of deterioration") and patients' satisfaction. The latter was based not just on specific questionnaires and analog scales, but particularly by the patients' sharing the experience with other patients and consequently repeated requests by many to switch to a rapid administration of their ERT.

Therefore, the investigator is hereby proposing to investigate the safety and efficacy of a 10 minutes administration of velaglucerase alfa in a cohort of treatment-naive patients.

The current VPRIV label is restricted to a dosage of 60 units/kg body weight every other week (60 units/kg EOW) - this dose will be used throughout the study period. The enzyme will be provided by Shire, which will also provide a research grant for the conduction of the trial.

Conditions

  • Primary Disease

Interventions

DRUG

VPRIV

VPRIV (Velaglucerase alfa) Long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

Sponsors & Collaborators

  • Shaare Zedek Medical Center

    lead OTHER

Principal Investigators

  • Ari Zimran, M.D. · 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
2018-09-04
Primary Completion
2019-09-30
Completion
2021-03-18

Countries

  • Israel

Study Locations

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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 NCT03702361 on ClinicalTrials.gov