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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-09-04

Study Completion Date

2021-03-18

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

NCT04120506

Gaucher Disease, Type 1

COMPLETED PHASE4

A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

NCT04721366

Gaucher Disease

COMPLETED

Home Therapy With VPRIV in Gaucher's Disease

NCT01356537

Gaucher Disease

COMPLETED

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

NCT00635427

Gaucher Disease, Type 1

COMPLETED PHASE3

A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

NCT04718779

Gaucher Disease

COMPLETED PHASE4

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Primary Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

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.

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Rapid infusion of Vpriv

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

Group Type EXPERIMENTAL

VPRIV

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VPRIV

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

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Velaglucerase Alfa

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Minimum Eligible Age

6 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No