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Gaucher Disease, Type 1Phase 4NCT04120506

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

A Phase 4 study testing VPRIV in people with Gaucher Disease, Type 1, run by Shaare Zedek Medical Center.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 6 to 75 years
Conditions the study is for
Gaucher Disease, Type 1
What is being tested?
VPRIV
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
No sites listed yet
Study dates
10 Jan 2016to 1 Jan 2022
Phase
Phase 4
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 75 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

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

About this study

Official 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

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.

Read more

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.

Read the detailed description

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.

Being tested
VPRIV
Medical subject headings
Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VPRIVDrug

    Also called Velaglucerase Alfa

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

Study groups in NCT04120506
Study groupRoleWhat they receive
Rapid infusion of VprivExperimental: 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
Receives: Drug: VPRIV

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    measured by blood pressure pre and post infusion

    Measured: 9 months

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

    measured by heart rate pre and post infusion

    Measured: 9 months

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

    measured by temperature pre and post infusion

    Measured: 9 months

Secondary outcomes (5)

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

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

    Measured: 9 months

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

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

    Measured: 9 months

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

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

    Measured: 9 months

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

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

    Measured: 9 months

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

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

    Measured: 9 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Oct 2019
  2. Study start10 Jan 2016
  3. Primary completion20 Jan 2017
  4. Study completion1 Jan 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
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.
Masking
None (open label)
Enrolment
15 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04120506
Sponsor's protocol ID
0075-15-SZMC
Status last verified
Oct 2022
First submitted
10 Aug 2018
Last updated
28 Oct 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 29989200

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing VPRIV in people with Gaucher Disease, Type 1, run by Shaare Zedek Medical Center.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of change from baseline in blood pressure for rapid infusion-1 (time frame: 9 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04120506, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2022.

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