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

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ClinicalTrials.gov Identifier: NCT03702361
Recruitment Status : Recruiting
First Posted : October 11, 2018
Last Update Posted : February 19, 2019
Sponsor:
Information provided by (Responsible Party):
Ari Zimran, Shaare Zedek Medical Center

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.


Condition or disease Intervention/treatment Phase
Primary Disease Drug: VPRIV Phase 4

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Intervention Model Description: 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.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study
Actual Study Start Date : September 4, 2018
Estimated Primary Completion Date : September 30, 2019
Estimated Study Completion Date : September 30, 2020


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 Name: Velaglucerase Alfa




Primary Outcome Measures :
  1. Percent change from baseline in spleen volume measured by MRI [ Time Frame: 12 months. ]
    Percent change from baseline


Secondary Outcome Measures :
  1. Change from baseline in Hemoglobin [ Time Frame: 12 months ]
    Percent change from baseline

  2. Change from baseline in platelet count [ Time Frame: 12 months ]
    Percent change from baseline

  3. Change from baseline in Lyso-GB1 [ Time Frame: 12 months ]
    Percent change from baseline

  4. Change from baseline in liver volume [ Time Frame: 12 months ]
    Percent change from baseline

  5. Change from baseline 10% reduction in spleen volume [ Time Frame: 6 months ]
    Percent change from baseline



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Ages Eligible for Study:   6 Years to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03702361


Contacts
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Contact: Ari Zimran, MD +972-2-6555143 azimran@gmail.com
Contact: Shoshana Revel- Vilk, MD +97226555143 svilk@szmc.org.il

Locations
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Israel
Michal Becker- Cohen Recruiting
Jerusalem, Please Select..., Israel, 9103102
Contact: Ari Zimran, M.D.    +97226555143    azimran@gmail.com   
Contact: Michal Becker- Cohen, M.Sc.    +97226555143    michalbec@gmail.com   
Principal Investigator: Ari Zimran, Prof.         
Sponsors and Collaborators
Shaare Zedek Medical Center
Investigators
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Principal Investigator: Ari Zimran, M.D. Ari Zimran - Shaare Zedek

Publications of Results:
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Responsible Party: Ari Zimran, Professor, Shaare Zedek Medical Center
ClinicalTrials.gov Identifier: NCT03702361     History of Changes
Other Study ID Numbers: 307-17-SZMC
First Posted: October 11, 2018    Key Record Dates
Last Update Posted: February 19, 2019
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders