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

  • STATUS
    Recruiting
  • End date
    Aug 26, 2022
  • participants needed
    20
  • sponsor
    Takeda
Updated on 22 May 2021
replacement therapy
type 1 gaucher disease
velaglucerase alfa
substrate reduction therapy
vpriv

Summary

The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means the participant will be treated according to the clinic's standard practice. The study sponsor will not be involved in how participants are treated with VPRIV, will provide instructions on how the clinic will record what happens during the study.

VPRIV is a type of enzyme replacement therapy (also known as ERT). Before starting the study, participants must either have switched from substrate reduction therapies (SRT) to VPRIV or switched from other enzyme replacement therapies to SRT then finally to VPRIV. During this time, medical data will be collected from the participants' medical records.

During the study, participants will be treated with VPRIV according to their clinic's standard practice. VPRIV is given by a slow injection into the vein, also known as an infusion. This will happen in the clinic or at home.

The study will record if blood levels of specific substances remain stable or improve during the switch to treatment with VPRIV. Some of these substances will show if organs such as the liver or spleen are working well. Others are blood cells that help blood to clot, known as platelets. Another is a substance in a red blood cell used to carry oxygen around the body, known as hemoglobin.

Participants will use a digital tool so they can be more involved in decision making in their treatment. The digital tool is a mobile phone app, in which each participant can log their daily activities, their general health and wellbeing, and other key information.

Medical data will also be collected from the participants' charts during this time.

Health problems of the participants will be recorded during the study to check if there were any side effects from VPRIV treatment.

Participants will be in this study for up to 12 months.

Details
Condition Gaucher Disease, gaucher's disease
Treatment Velaglucerase alfa
Clinical Study IdentifierNCT04718779
SponsorTakeda
Last Modified on22 May 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Participant eligibility is determined according to the following criteria
prior to entry into the study
In the opinion of the investigator, the participant is capable of understanding and complying with protocol requirements
Participant either signs and dates a written, informed consent form or completes an e-consent process prior to the initiation of any study procedures
Participant has been diagnosed with GD type I, diagnosis was confirmed biochemically and/or genetically
Participant has been treated with an SRT for at least 3 months prior to switch to VPRIV
Participant has been treated with VPRIV at least 3 months prior to enrollment (Baseline [Day 0])
Participant is aged 18 or older
Participant is able to use mobile application based on clinician's judgment, (e.g., owns an iPhone version 5 or later or smartphones with Android operating systems, have an active data plan or regular Wi-Fi access)
Participants primary language is English

Exclusion Criteria

Any participant who meets any of the following criteria will not qualify for
entry into the
study
Participant is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (e.g., spouse, parent, child, sibling) or may consent under duress
Participant is judged by the investigator as being ineligible for any other reason
Participant has L444P/L444P GBA1 genotype (c.1448T greater than [>] C)
Participant has Parkinson's disease, a history of central nervous system [CNS] manifestations, or any other neurological disorder (e.g. Lewy Body Disease, Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Multiple sclerosis)
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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