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

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study

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.

Study Overview

• Status: Completed
• Conditions: Primary Disease
• Intervention / Treatment: Drug: VPRIV

Detailed Description

Protocol:

Study Design: This will be a single-center, open-label trial to assess the safety and efficacy of rapid administration of velaglucerase alfa (VPRIV) to treatment-naive patients with type I Gaucher disease. The first six infusions will be administered in the hospital: the first three infusions within 60, 30 and 20 minutes each, with the beginning of a 10 minutes administration from infusion #4. Following the three uneventful administration of the 10 minutes in the hospital, the bi-weekly ERT will continue as home therapy, as outlined in the protocol. The duration of the study will be 12 months, with an extension pending positive results.

Number of Patients: 15. The first ten patients will be adults, and then children will be allowed to enroll in the trial.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 4

Contacts and Locations

Study Locations

• Israel
  • Please Select...
    • Jerusalem, Please Select..., Israel, 9103102
      • Michal Becker- Cohen

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Males and females, 18 years or older for the first ten adult patients; 6 years or older for the last five patients.

  • Confirmed enzymatic diagnosis of Gaucher disease with a defined genotype and elevated biomarker LysoGb1, performed at CentoGene using DBS methodology.
  • Indications for ERT will be guided by fulfilling the MOH criteria.
  • Female patients of child-bearing potential who agree to use a medically acceptable method of contraception.
  • Patients who have not received ERT or SRT in the past or Patients who have not received ERT or SRT in the past 12 months and have a negative anti-glucocerebrosidase antibody

Exclusion Criteria:

• Currently taking another experimental drug for any condition

  • Presence of neurologic signs and symptoms characteristic of Type 2 or Type 3 Gaucher disease
  • Pregnant or nursing
  • Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Rapid infusion of Vpriv; Rapid intravenous infusion of velaglucerase alfa (VPRIV) in treatment-naive patients with type 1 Gaucher disease	Drug: VPRIV; VPRIV (Velaglucerase alfa) Long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.; Other Names: Velaglucerase Alfa

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent change from baseline in spleen volume measured by MRI	Percent change from baseline	12 months.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in Hemoglobin	Percent change from baseline	12 months
Change from baseline in platelet count	Percent change from baseline	12 months
Change from baseline in Lyso-GB1	Percent change from baseline	12 months
Change from baseline in liver volume	Percent change from baseline	12 months
Change from baseline 10% reduction in spleen volume	Percent change from baseline	6 months

Collaborators and Investigators

• Sponsor: Shaare Zedek Medical Center
• Investigators: Principal Investigator: Ari Zimran, M.D., Ari Zimran - Shaare Zedek

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): September 4, 2018
• Primary Completion (Actual): September 30, 2019
• Study Completion (Actual): March 18, 2021

Study Registration Dates

• First Submitted: August 6, 2018
• First Submitted That Met QC Criteria: October 9, 2018
• First Posted (Actual): October 11, 2018

Study Record Updates

• Last Update Posted (Actual): March 19, 2021
• Last Update Submitted That Met QC Criteria: March 18, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: 307-17-SZMC

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No