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Completed

NCT Number: NCT04120506

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

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.

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Key information

Conditions

Gaucher Disease, Type 1 Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Gaucher Disease Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

6 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

About this study

Every 6 months, patients will be required to come for routine checkups at SZMC, where the following tests will be performed:

  • Complete Blood Count (CBC)
  • Routine serum biochemistry including liver function tests (LFTs)
  • Plasma biomarker lyso Gb-1
  • Height & weight & calculation of BMI
  • Physical examination and medical history elicited including concomitant medications
  • Ultrasound for spleen and liver volumes

In addition, the following tests will be performed at 12, 24 and 36 months:

  • Echocardiography
  • Electrocardiogram (ECG)
  • Urinalysis
  • HRQoL questionnaire (TBD)

At each home visit, the following assessments will be performed by the study nurse:

Queries regarding AEs and changes in clinically relevant Gaucher parameters as described by the patient (e.g., bone pain), inter-current illnesses, etc.

Patients will be required to complete the End-of-study visit, including the final infusion at 10', at SZMC. This final visit will include in addition to the usual safety and efficacy assessments and routine tests, (mentioned above) also, DEXA and anti-drug antibodies.

In addition, we would perform a 4th PK measurement at end of the extension period.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

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

Treatment and study plan

VPRIV

Drug

Safety, pharmacokinetics, and efficacy of rapid intravenous infusion of velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease

Other names: Velaglucerase Alfa

Primary outcomes

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

    Time frame: 9 months

    measured by blood pressure pre and post infusion

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

    Time frame: 9 months

    measured by heart rate pre and post infusion

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

    Time frame: 9 months

    measured by temperature pre and post infusion

Secondary outcomes

  1. Non deterioration in Gaucher manifestations- stability in platelet counts

    Time frame: 9 months

    Efficacy secondary endpoints are non-deterioration (defined as stability) in platelet count

  2. Non deterioration in Gaucher manifestations- stability in hemaglobin count

    Time frame: 9 months

    Efficacy secondary endpoints are non-deterioration (defined as stability) in hemaglobin count

  3. Non deterioration in Gaucher manifestations- measured by liver volume

    Time frame: 9 months

    Efficacy secondary endpoints are non-deterioration (defined as stability) in organ volumes of liver volume (as previously defined in the TKT034 clinical trial)

  4. Non deterioration in Gaucher manifestations- measured by spleen volumes

    Time frame: 9 months

    Efficacy secondary endpoints are non-deterioration (defined as stability) in organ volumes of spleen volume (as previously defined in the TKT034 clinical trial)

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

    Time frame: 9 months

    Efficacy secondary endpoints are non-deterioration (defined as stability) by lack of sustained increases in the biomarkers.

Sponsors and collaborators

Lead sponsor

Shaare Zedek Medical Center

Other

Collaborators

  • Shire

Registry information

Official study title

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study

Important dates

Study start
2016
Primary completion
2017
Study completion
2022
First posted
Oct 9, 2019
Registry last updated
Oct 28, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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