Gaucher DiseasePEDSNCT04721366
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
An observational study of Gaucher Disease, run by Takeda at 1 site in United States.
Trial summary
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests. Read more about this study
- Who is it for?
- Ages Up to 5 years
- Conditions the study is for
- Gaucher Disease
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Jan 2021to 17 Apr 2023
- Phase
- Observational
- 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 Up to 5 years
- All sexes
- No healthy volunteers
- Child
Study population: Neonatal and pediatric participants included have a diagnosis of GD type I and III and are currently being treated with total ERT (VPRIV) for less than or equal to (<=) 36 months from the time of treatment initiation.
Who may be able to join
- The participant's caregiver is able and willing to provide informed consent.
- The participant is male or female younger than or equal to 4 years of age at treatment initiation.
- The participant has received and confirmed a current diagnosis of GD type 1 or type 3 (biochemically and/or genetically).
- The participant has been receiving intravenous (IV) Velaglucerase alfa treatment for GD.
- In the opinion of the investigator, the participant's caregiver is capable of understanding and complying with protocol requirements.
- The participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.
Who cannot join
- The 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., child, sibling) or may consent under duress.
- The participant is judged by the investigator as being ineligible for any other reason.
Trial locations and contacts
1 site in 1 country.
United States1 site
Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, 22030
About this study
Official title: Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
This study is about collecting data available in the participant's medical record as well as data from each participant's ongoing treatment. No study medicines will be provided to participants in this study. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.
When the participants start the study, they will visit the study clinic every 6 months after their first visit.
- Conditions
- Gaucher Disease
- Being tested
- Standard of Care
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard of CareOther
Also called SOC
Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.
| Study group | Role | What they receive |
|---|---|---|
| Standard of Care (SoC) | Neonatal and pediatric participants who has been on ERT (VPRIV) will be followed up for 36 months from the time of treatment initiation as per SOC. Receives: Other: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Hemoglobin (Hb) Level
Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed.
Measured: From start of ERT initiation up to 5 years of age
Percent Change From Baseline in Platelet Count Increase
Percent change from baseline for platelet count increase will be assessed.
Measured: From start of ERT initiation up to 5 years of age
Percent Change From Baseline in Liver Volume
Percent change from baseline in liver volume will be assessed.
Measured: From start of ERT initiation up to 5 years of age
Percent Change From Baseline for Spleen Volume
Percent change from baseline for spleen volume will be assessed.
Measured: From start of ERT initiation up to 5 years of age
Percentage of Participants With Growth Normalization
Percentage of participants with growth normalization will be assessed.
Measured: From start of ERT initiation up to 5 years of age
Percentage of Participants With Improvement in Bone Disease
Percentage of participants with improvement in bone disease will be assessed.
Measured: From start of ERT initiation up to 5 years of age
Percentage of Participants With Improvement in Thrombocytopenia
Percentage of participants with improvement in thrombocytopenia will be assessed.
Measured: From start of ERT initiation up to 5 years of age
Secondary outcomes (1)
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. AEs include SAEs, non-serious AEs.
Measured: From start of ERT initiation up to 5 years of age
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 start8 Jan 2021
- Primary completion17 Apr 2023
- Study completion17 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 11 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
- NCT04721366
- Sponsor's protocol ID
- MACS-2020-052801
- Other IDs
- TAK-669-4019
- Acronym
- PEDS
- Status last verified
- Mar 2024
- First submitted
- 21 Jan 2021
- Last updated
- 29 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- 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 29 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT04721366
- Acronym / sponsor ID
- PEDS / MACS-2020-052801
- Conditions
- Gaucher Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 8 Jan 2021 (actual)
- Primary completion
- 17 Apr 2023 (actual)
- Study completion
- 17 Apr 2023 (actual)
- First posted
- 22 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
This study is about collecting data available in the participant's medical record as well as data from each participant's ongoing treatment. No study medicines will be provided to participants in this study. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.
When the participants start the study, they will visit the study clinic every 6 months after their first visit.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Standard of Care (SoC) | – | Other: Standard of CareNeonatal and pediatric participants who has been on ERT (VPRIV) will be followed up for 36 months from the time of treatment initiation as per SOC. |
Who may qualify
- Ages
- Up to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Neonatal and pediatric participants included have a diagnosis of GD type I and III and are currently being treated with total ERT (VPRIV) for less than or equal to (<=) 36 months from the time of treatment initiation.
Key inclusion criteria (6 listed)
- The participant's caregiver is able and willing to provide informed consent.
- The participant is male or female younger than or equal to 4 years of age at treatment initiation.
- The participant has received and confirmed a current diagnosis of GD type 1 or type 3 (biochemically and/or genetically).
- The participant has been receiving intravenous (IV) Velaglucerase alfa treatment for GD.
- In the opinion of the investigator, the participant's caregiver is capable of understanding and complying with protocol requirements.
Key exclusion criteria (2 listed)
- The 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., child, sibling) or may consent under duress.
- The participant is judged by the investigator as being ineligible for any other reason.
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 |
|---|---|---|
| Standard of CareAlso: SOCNeonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC. | Other | Standard of Care (SoC) |
Primary outcome
Change From Baseline in Hemoglobin (Hb) Level
Time frame: From start of ERT initiation up to 5 years of age
Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed.
Percent Change From Baseline in Platelet Count Increase
Time frame: From start of ERT initiation up to 5 years of age
Percent change from baseline for platelet count increase will be assessed.
Percent Change From Baseline in Liver Volume
Time frame: From start of ERT initiation up to 5 years of age
Percent change from baseline in liver volume will be assessed.
Percent Change From Baseline for Spleen Volume
Time frame: From start of ERT initiation up to 5 years of age
Percent change from baseline for spleen volume will be assessed.
Percentage of Participants With Growth Normalization
Time frame: From start of ERT initiation up to 5 years of age
Percentage of participants with growth normalization will be assessed.
Percentage of Participants With Improvement in Bone Disease
Time frame: From start of ERT initiation up to 5 years of age
Percentage of participants with improvement in bone disease will be assessed.
Percentage of Participants With Improvement in Thrombocytopenia
Time frame: From start of ERT initiation up to 5 years of age
Percentage of participants with improvement in thrombocytopenia will be assessed.
1 secondary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From start of ERT initiation up to 5 years of age
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2024.
- 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).
Related trials for Gaucher Disease
RecruitingObservational study
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Neurological Regression, Myoclonus
RecruitingObservational study
Parkinson's Disease in Type 1 Gaucher Disease
Parkinson Disease (PD), Type 1 Gaucher Disease
RecruitingObservational study
Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
Gaucher Disease Type I, Oxidative Stress
Not yet recruitingPhase 1/2
4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
Neuroblastoma
Enrolling by invitationObservational study
Genetic Studies of Lysosomal Storage Disorders
Lysosomal Storage Disorders, Gaucher Disease
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?
An observational study of 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 6 inclusion and 2 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 29 Mar 2024. 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.
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?
Change From Baseline in Hemoglobin (Hb) Level (time frame: From start of ERT initiation up to 5 years of age). The study lists 7 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 29 Mar 2024.