Gaucher DiseasePhase 3NCT01422187
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
A Phase 3 study testing Taliglucerase alfa in people with Gaucher Disease, run by Pfizer.
Trial summary
This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. 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?
- Taliglucerase alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- No sites listed yet
- Study dates
- Aug 2011to Sep 2014
- Phase
- Phase 3
- 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
- Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003
- The subject signs an informed consent
Who cannot join
- Currently taking another investigational drug for any condition.
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.
About this study
This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.
- Conditions
- Gaucher Disease
- Being tested
- Taliglucerase alfa
- Keywords
- enzyme replacement therapy
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Taliglucerase alfaDrug
Taliglucerase infusion every two weeks for 21 months
| Study group | Role | What they receive |
|---|---|---|
| Taliglucerase alfa 30 units/kg | Experimental | Subjects randomized to receive 30 units/kg Receives: Drug: Taliglucerase alfa |
| Taliglucerase alfa 60 units/kg | Experimental | Subjects randomized to 60 units/kg Receives: Drug: Taliglucerase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Spleen Volume
Spleen volume measured by MRI
Measured: 60 months
Secondary outcomes (3)
Liver Volume
Liver volume by MRI
Measured: 60 months
Platelet Count
Platelet count measure annually
Measured: 60 months
Hemoglobin
Hemoglobin measure yearly
Measured: 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2011 (estimated)
- Study startAug 2011
- Primary completionMay 2014
- Study completionSep 2014
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- More trials run by PfizerThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01422187
- Sponsor's protocol ID
- PB-06-007
- Status last verified
- Apr 2023
- First submitted
- 22 Aug 2011
- Last updated
- 19 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Expanded access
- Available
Publications
Zimran A, Duran G, Giraldo P, Rosenbaum H, Giona F, Petakov M, Terreros Munoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R. Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naive patients with Gaucher disease. Blood Cells Mol Dis. 2019 Sep;78:14-21. doi: 10.1016/j.bcmd.2016.07.002. Epub 2016 Jul 18.
PubMed 27499018
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
- ClinicalTrials.gov ID
- NCT01422187
- Acronym / sponsor ID
- PB-06-007
- Conditions
- Gaucher Disease
- Keywords
- enzyme replacement therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- Aug 2011
- Primary completion
- May 2014 (actual)
- Study completion
- Sep 2014 (actual)
- First posted
- 23 Aug 2011 (estimated)
- Results
- Posted 14 Dec 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Taliglucerase alfa 30 units/kg | Experimental | Drug: Taliglucerase alfaSubjects randomized to receive 30 units/kg |
| Taliglucerase alfa 60 units/kg | Experimental | Drug: Taliglucerase alfaSubjects randomized to 60 units/kg |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003
- The subject signs an informed consent
Key exclusion criteria (2 listed)
- Currently taking another investigational drug for any condition.
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.
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 |
|---|---|---|
| Taliglucerase alfaTaliglucerase infusion every two weeks for 21 months | Drug | Taliglucerase alfa 30 units/kg, Taliglucerase alfa 60 units/kg |
Primary outcome
Spleen Volume
Time frame: 60 months
Spleen volume measured by MRI
3 secondary outcomes
Liver Volume
Time frame: 60 months
Platelet Count
Time frame: 60 months
Hemoglobin
Time frame: 60 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 27499018Zimran A, Duran G, Giraldo P, Rosenbaum H, Giona F, Petakov M, Terreros Munoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R. Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naive patients with Gaucher disease. Blood Cells Mol Dis. 2019 Sep;78:14-21. doi: 10.1016/j.bcmd.2016.07.002. Epub 2016 Jul 18.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Taliglucerase alfa in people with Gaucher Disease, run by Pfizer.
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 2 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 19 Apr 2023. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 14 Dec 2015). 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?
Spleen Volume (time frame: 60 months). 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 NCT01422187, 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 19 Apr 2023.