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

Primary Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03702361
• Other study ID #: 307-17-SZMC
• Status: Completed
• Phase: Phase 4
• Start date: September 4, 2018
• Est. completion date: March 18, 2021

Study information

• Verified date: March 2021
• Source: Shaare Zedek Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: March 18, 2021
• Est. primary completion date: September 30, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Gaucher Disease
• Primary Disease

Intervention

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

Locations

Country	Name	City	State
Israel	Michal Becker- Cohen	Jerusalem	Please Select...

Sponsors (1)

Lead Sponsor	Collaborator
Shaare Zedek Medical Center

Country where clinical trial is conducted

Israel, 

References & 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change from baseline in spleen volume measured by MRI	Percent change from baseline	12 months.
Secondary	Change from baseline in Hemoglobin	Percent change from baseline	12 months
Secondary	Change from baseline in platelet count	Percent change from baseline	12 months
Secondary	Change from baseline in Lyso-GB1	Percent change from baseline	12 months
Secondary	Change from baseline in liver volume	Percent change from baseline	12 months
Secondary	Change from baseline 10% reduction in spleen volume	Percent change from baseline	6 months