Gaucher Disease Type 1Phase 4NCT02528617
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
A Phase 4 study testing Velaglucerase alfa in people with Gaucher Disease Type 1 and Gaucher Disease Type 3, run by Baylor Research Institute at 1 site in United States.
Trial summary
The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied. Read more about this study
- Who is it for?
- Ages 4 to 14 years
- Conditions the study is for
- Gaucher Disease Type 1, Gaucher Disease Type 3
- What is being tested?
- Velaglucerase alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2015to Oct 2017
- 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 4 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Enzyme Replacement Therapy naive,
- confirmed diagnosis of Gaucher disease type 1 or 3,
- able to travel to Dallas, Texas 1x per year for baseline plus 3 consecutive years,
- able to tolerate all study procedures,
- skeleton not fully formed as confirmed by DXA and MRI),
- and willing to receive velaglucerase alfa infusions every other week for the duration of the study.
Who cannot join
- Clinically unstable,
- taking or have taken bisphosphonates,
- Gaucher type 2,
- pregnant female,
- or deemed inappropriate for participation by the principal investigator.
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.
- Conditions
- Gaucher Disease Type 1, Gaucher Disease Type 3
- Being tested
- Velaglucerase alfa
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Velaglucerase alfaDrug
Also called VPRIV
Enzyme replacement therapy
| Study group | Role | What they receive |
|---|---|---|
| Gaucher Type 1 or 3 | Other | Velaglucerase alfa IV 60 units/kg every other week for duration of the study. Receives: Drug: Velaglucerase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa.
Measured: Baseline pre-intervention and yearly thereafter for 3 years
Secondary outcomes (4)
Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa.
Measured: Baseline pre intervention and yearly thereafter for 3 years
Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Measured: Baseline pre intervention and yearly thereafter for 3 years
Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Measured: Baseline pre-intervention and yearly thereafter for 3 years
Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Measured: Baseline pre-intervention and yearly thereafter for 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2015 (estimated)
- Study startJul 2015 (estimated)
- Primary completionOct 2017
- Study completionOct 2017
Why it stopped: No subjects accrued and no potential subjects were identified..
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease Type 1Studies of the same condition, open and under way.
- More trials run by Baylor Research InstituteThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Texas Scottish Rite Hospital for Children
- Responsible party
- Sponsor
- Investigators
- Raphael Schiffmann, M.D.,M.H.Sc., Principal investigator, Baylor Research Institute/Institute of Metabolic Disease
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02528617
- Sponsor's protocol ID
- 014-285
- Status last verified
- Feb 2018
- First submitted
- 28 Jul 2015
- Last updated
- 2 Mar 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
- ClinicalTrials.gov ID
- NCT02528617
- Acronym / sponsor ID
- 014-285
- Conditions
- Gaucher Disease Type 1, Gaucher Disease Type 3
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No subjects accrued and no potential subjects were identified..
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Baylor Research InstituteUniversity, hospital or other
- Collaborators
- Texas Scottish Rite Hospital for Children
- Study start
- Jul 2015 (estimated)
- Primary completion
- Oct 2017 (actual)
- Study completion
- Oct 2017 (actual)
- First posted
- 19 Aug 2015 (estimated)
- 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 purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gaucher Type 1 or 3 | Other | Drug: Velaglucerase alfaVelaglucerase alfa IV 60 units/kg every other week for duration of the study. |
Who may qualify
- Ages
- 4 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Enzyme Replacement Therapy naive,
- confirmed diagnosis of Gaucher disease type 1 or 3,
- able to travel to Dallas, Texas 1x per year for baseline plus 3 consecutive years,
- able to tolerate all study procedures,
- skeleton not fully formed as confirmed by DXA and MRI),
Key exclusion criteria (5 listed)
- Clinically unstable,
- taking or have taken bisphosphonates,
- Gaucher type 2,
- pregnant female,
- or deemed inappropriate for participation by the principal investigator.
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 |
|---|---|---|
| Velaglucerase alfaAlso: VPRIVEnzyme replacement therapy | Drug | Gaucher Type 1 or 3 |
Primary outcome
Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa.
Time frame: Baseline pre-intervention and yearly thereafter for 3 years
4 secondary outcomes
Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa.
Time frame: Baseline pre intervention and yearly thereafter for 3 years
Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Time frame: Baseline pre intervention and yearly thereafter for 3 years
Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Time frame: Baseline pre-intervention and yearly thereafter for 3 years
Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Time frame: Baseline pre-intervention and yearly thereafter for 3 years.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2018.
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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.
Raphael Schiffmann, M.D.,M.H.Sc. principal investigator
Baylor Research Institute/Institute of Metabolic Disease
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 Velaglucerase alfa in people with Gaucher Disease Type 1 and Gaucher Disease Type 3, run by Baylor Research Institute 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 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: withdrawn, as of the sponsor's last update on 2 Mar 2018. 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?
The study was withdrawn before enrolling participants, so there are no results.
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 in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa. (time frame: Baseline pre-intervention and yearly thereafter for 3 years). 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 2 Mar 2018; AzTrials last refreshed its copy on 7 Nov 2026.