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
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|
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 (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)|
|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|
Experimental: Rapid infusion of Vpriv
Rapid intravenous infusion of velaglucerase alfa (VPRIV) in treatment-naive patients with type 1 Gaucher disease
VPRIV (Velaglucerase alfa) Long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.
Other Name: Velaglucerase Alfa
- Percent change from baseline in spleen volume measured by MRI [ Time Frame: 12 months. ]Percent change from baseline
- Change from baseline in Hemoglobin [ Time Frame: 12 months ]Percent change from baseline
- Change from baseline in platelet count [ Time Frame: 12 months ]Percent change from baseline
- Change from baseline in Lyso-GB1 [ Time Frame: 12 months ]Percent change from baseline
- Change from baseline in liver volume [ Time Frame: 12 months ]Percent change from baseline
- Change from baseline 10% reduction in spleen volume [ Time Frame: 6 months ]Percent change from baseline
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
|Contact: Ari Zimran, MDemail@example.com|
|Contact: Shoshana Revel- Vilk, MDfirstname.lastname@example.org|
|Michal Becker- Cohen||Recruiting|
|Jerusalem, Please Select..., Israel, 9103102|
|Contact: Ari Zimran, M.D. +97226555143 email@example.com|
|Contact: Michal Becker- Cohen, M.Sc. +97226555143 firstname.lastname@example.org|
|Principal Investigator: Ari Zimran, Prof.|
|Principal Investigator:||Ari Zimran, M.D.||Ari Zimran - Shaare Zedek|