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

NCT03702361 | Completed Phase 4

• 1 other identifier: 307-17-SZMC
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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

Outcome Measures

Primary Outcomes (1)

• Percent change from baseline in spleen volume measured by MRI

  Percent change from baseline

  12 months.

Secondary Outcomes (5)

• Change from baseline in Hemoglobin

  12 months

• Change from baseline in platelet count

  12 months

• Change from baseline in Lyso-GB1

  12 months

• Change from baseline in liver volume

  12 months

• Change from baseline 10% reduction in spleen volume

  6 months

Study Arms (1)

Rapid infusion of Vpriv

EXPERIMENTAL

Rapid intravenous infusion of velaglucerase alfa (VPRIV) in treatment-naive patients with type 1 Gaucher disease

Drug: VPRIV

Interventions

VPRIV DRUG

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

Also known as: Velaglucerase Alfa

Rapid infusion of Vpriv

Eligibility Criteria

• Age: 6 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shaare Zedek Medical Center: lead

Study Sites (1)

Michal Becker- Cohen

Jerusalem, Please Select..., 9103102, Israel

Location

Related Publications (1)

• 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.

  PMID: 29989200 RESULT

MeSH Terms

Interventions

Glucosylceramidase

Intervention Hierarchy (Ancestors)

Glucosidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Study Officials

• Ari Zimran, M.D.

  Ari Zimran - Shaare Zedek

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: 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.

Study Record Dates

• First Submitted: August 6, 2018
• First Posted: October 11, 2018
• Study Start: September 4, 2018
• Primary Completion: September 30, 2019
• Study Completion: March 18, 2021
• Last Updated: March 19, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1)