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

NCT04120506 | Completed Phase 4 DMC FDA Drug

• 1 other identifier: 0075-15-SZMC
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

Brief Summary

Background: In order to allow our satisfied patients, who have successfully completed 24 months of rapid intravenous infusion of Velaglucerase alfa (VPRIV), to continue with the 10 minutes IV therapy, the clinical trial framework must be extended; and this extension is important for the assessment of long term benefit (up to 5 years) of this regimen of administration of Velaglucerase alfa.. Suggested trial: An additional 36 months home therapy follow up of safety and efficacy of rapid intravenous infusion of Velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease. Patients must have completed the prior 4 parts / 24 months of the protocol before enrolling into this extension phase ("Part 5") and have provided a new consent before entering PART 5 of the study. Patients must not have experienced clinically significant AEs, including allergic reactions, in any of the prior study parts of this protocol to be eligible to participate, and have maintained stability in the key disease features. All infusions of 10' will be given in the context of home therapy. "Clinically significant" AEs will be determined by the PI using standard description of AEs as previously described at phase 3, and if necessary will support withdrawal of the patient from the study.

Conditions

Gaucher Disease, Type 1

Outcome Measures

Primary Outcomes (3)

• Incidence of change from baseline in blood pressure for rapid infusion-1

  measured by blood pressure pre and post infusion

  9 months

• Incidence of change from baseline in heart rate for rapid infusion-1

  measured by heart rate pre and post infusion

  9 months

• Incidence of change from baseline in temperature for rapid infusion-1

  measured by temperature pre and post infusion

  9 months

Secondary Outcomes (5)

• Non deterioration in Gaucher manifestations- stability in platelet counts

  9 months

• Non deterioration in Gaucher manifestations- stability in hemaglobin count

  9 months

• Non deterioration in Gaucher manifestations- measured by liver volume

  9 months

• Non deterioration in Gaucher manifestations- measured by spleen volumes

  9 months

• Non deterioration in Gaucher manifestations- measured by lack of elevated biomarker- Lyso-GB1

  9 months

Study Arms (1)

Rapid infusion of Vpriv

EXPERIMENTAL

: Infusions at Baseline and during step-wise rate increases and End-of-study will be performed in the Shaare Zedek Medical Center (SZMC) by the Study Nurse who will monitor vital signs (see below) for a total of 8 visits at SZMC. Home therapy will be approved if the patient so desires for 5 infusions in Phase 1 and for the first 5 infusions in Phase 3. All routine hematological and biochemical tests will be performed in the SZMC clinical labs. Abdominal quantitative MR Imaging (MRI) for spleen and liver volumes will be performed at SZMC

Drug: VPRIV

Interventions

VPRIV DRUG

Safety, pharmacokinetics, and efficacy of rapid intravenous infusion of velaglucerase alfa (VPRIV) in adult 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:

• Aged 18-75 years, non-splenectomized Enzymatic diagnosis \& molecular analysis indicative of type 1 Gaucher disease Receiving VPRIV for at least 6 infusions (3 months) prior to Baseline at a constant dose and frequency and without clinically significant AEs including allergic reactions

You may not qualify if:

• Experience of a clinically significant AE to VPRIV at any time in the past Existence of a clinically significant co-morbidity

Sponsors & Collaborators

• Shaare Zedek Medical Center: lead
• Shire: collaborator

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

Conditions

Gaucher Disease

Interventions

Glucosylceramidase

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

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

Study Officials

• Ari Zimran

  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: Objective: To show non-inferiority of rapid (defined as 10') intravenous (IV) infusion versus standard IV infusion rate (60') of VPRIV with regard to safety, pharmacokinetic(PK) profile, and efficacy in patients who have been receiving VPRIV for a minimum of 3 months at a constant dosage.

Study Record Dates

• First Submitted: August 10, 2018
• First Posted: October 9, 2019
• Study Start: January 10, 2016
• Primary Completion: January 20, 2017
• Study Completion: January 1, 2022
• Last Updated: October 28, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share