Gaucher DiseasePhase 4SWITCHNCT04718779
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
A Phase 4 study testing Digital Engagement Application (GD App) in people with Gaucher Disease, run by Takeda at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- Digital Engagement Application (GD App)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Apr 2021to 16 Feb 2023
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 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]).
Show all 9Show fewer
- Participant is aged 18 or older.
- Arm A: 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).
- Arm A: The participant's primary language is English.
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
The Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030
About this study
Official title: A Multicenter, Interventional, Retrospective and Prospective Study of Enzyme Replacement Therapy (VPRIV) Clinical Outcomes and Safety in Gaucher Disease Type 1 Patients Previously Treated With Substrate Reduction Therapy
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.
Read more
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.
- Conditions
- Gaucher Disease
- Being tested
- Digital Engagement Application (GD App), No Intervention
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Digital Engagement Application (GD App)Other
This is a chart review (prospective) data analysis study to describe the effect of the treatment change on the clinical parameters and patient reported outcomes (PROs) with the use of a digital engagement application (GD app) (to evaluate participants clinical engagement, shared decision making, emotional wellbeing, activities of daily living, goal attainment and Gaucher Disease specific measures).
No InterventionOther
This is a chart review (retrospective) data analysis study to describe the effect of the treatment change on the clinical parameters.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: Prospective | Experimental | Participants with Gaucher's Disease 1 (GD1) transitioning from SRTs to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) in a real-world setting among adults 18 and older will be studied by observing standard patient care and by reviewing the results of tests and assessments that would be performed as part of their routine treatment. Receives: Other: Digital Engagement Application (GD App) |
| Arm B: Retrospective | Experimental | Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) will be assessed retrospectively after switch to ERT for up to 12 months. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Clinically Stable Hemoglobin (Hb) Concentration From Baseline up to Month 12
Hemoglobin concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of hemoglobin concentration level less than (<) 1.5 gram per deciliter (g/dl) decrease level. Number of participants with clinically stable hemoglobin from baseline up to Month 12 were reported.
Measured: From Baseline up to Month 12
Number of Participants With Clinically Stable Platelet Count From Baseline in Platelet Count up to Month 12
Platelet count concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of platelet count < 25 percent (%) decrease levels. Number of participants with clinically stable platelet count from baseline up to Month 12 were reported.
Measured: From Baseline up to Month 12
Number of Participants With Clinically Stable Liver Volume From Baseline up to Month 12
Clinical stability from baseline was calculated using prespecified threshold of liver volume < 20 % increase, was assessed using ultrasound or Magnetic Resonance Image (MRI). Number of participants with clinically stable liver volume from baseline up to Month 12 were reported.
Measured: From Baseline up to Month 12
Number of Participants With Clinically Stable Spleen Volume From Baseline up to Month 12
Clinical stability from baseline was calculated using prespecified threshold of spleen volume < 25 % increase was assessed using ultrasound or MRI. Number of participants with clinically stable spleen volume from baseline up to Month 12 were reported.
Measured: From Baseline up to Month 12
Secondary outcomes (1)
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE was defined as any untoward medical occurrence in a clinical investigation participant administered with a drug; it does not necessarily have to have a causal relationship with the treatment. An SAE was any event that resulted in: death; was life-threatening; required inpatient hospitalization or resulted in prolongation of existing hospitalization; persistent or significant disability/incapacity; was a congenital anomaly/birth defect or a medically important event. AEs included both SAEs, and non-serious AEs.
Measured: From start of the study up to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2021
- Study start22 Apr 2021
- Primary completion16 Feb 2023
- Study completion16 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04718779
- Sponsor's protocol ID
- TAK-669-4017
- Acronym
- SWITCH
- Status last verified
- Dec 2023
- First submitted
- 20 Jan 2021
- Last updated
- 21 Dec 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Interventional, Retrospective and Prospective Study of Enzyme Replacement Therapy (VPRIV) Clinical Outcomes and Safety in Gaucher Disease Type 1 Patients Previously Treated With Substrate Reduction Therapy
- ClinicalTrials.gov ID
- NCT04718779
- Acronym / sponsor ID
- SWITCH / TAK-669-4017
- Conditions
- Gaucher Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 22 Apr 2021 (actual)
- Primary completion
- 16 Feb 2023 (actual)
- Study completion
- 16 Feb 2023 (actual)
- First posted
- 22 Jan 2021 (actual)
- Results
- Posted 21 Dec 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A: Prospective | Experimental | Other: Digital Engagement Application (GD App)Participants with Gaucher's Disease 1 (GD1) transitioning from SRTs to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) in a real-world setting among adults 18 and older will be studied by observing standard patient care and by reviewing the results of tests and assessments that would be performed as part of their routine treatment. |
| Arm B: Retrospective | Experimental | Other: No InterventionParticipants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) will be assessed retrospectively after switch to ERT for up to 12 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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 SRT for at least 3 months prior to switch to VPRIV.
Key exclusion criteria (5 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Digital Engagement Application (GD App)This is a chart review (prospective) data analysis study to describe the effect of the treatment change on the clinical parameters and patient reported outcomes (PROs) with the use of a digital engagement application (GD app) (to evaluate participants clinical engagement, shared decision making, emotional wellbeing, activities of daily living, goal attainment and Gaucher Disease specific measures). | Other | Arm A: Prospective |
| No InterventionThis is a chart review (retrospective) data analysis study to describe the effect of the treatment change on the clinical parameters. | Other | Arm B: Retrospective |
Primary outcome
Number of Participants With Clinically Stable Hemoglobin (Hb) Concentration From Baseline up to Month 12
Time frame: From Baseline up to Month 12
Hemoglobin concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of hemoglobin concentration level less than (<) 1.5 gram per deciliter (g/dl) decrease level. Number of participants with clinically stable hemoglobin from baseline up to Month 12 were reported.
Number of Participants With Clinically Stable Platelet Count From Baseline in Platelet Count up to Month 12
Time frame: From Baseline up to Month 12
Platelet count concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of platelet count < 25 percent (%) decrease levels. Number of participants with clinically stable platelet count from baseline up to Month 12 were reported.
Number of Participants With Clinically Stable Liver Volume From Baseline up to Month 12
Time frame: From Baseline up to Month 12
Clinical stability from baseline was calculated using prespecified threshold of liver volume < 20 % increase, was assessed using ultrasound or Magnetic Resonance Image (MRI). Number of participants with clinically stable liver volume from baseline up to Month 12 were reported.
Number of Participants With Clinically Stable Spleen Volume From Baseline up to Month 12
Time frame: From Baseline up to Month 12
Clinical stability from baseline was calculated using prespecified threshold of spleen volume < 25 % increase was assessed using ultrasound or MRI. Number of participants with clinically stable spleen volume from baseline up to Month 12 were reported.
1 secondary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From start of the study up to Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: No. De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Digital Engagement Application (GD App) in people with Gaucher Disease, run by Takeda at 1 site in United States.
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 9 inclusion and 5 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 21 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Yes. Results are posted in the registry (first posted 21 Dec 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Clinically Stable Hemoglobin (Hb) Concentration From Baseline up to Month 12 (time frame: From Baseline up to Month 12). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Dec 2023.