Gaucher DiseasePhase 3NCT00705939
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
A Phase 3 study testing Taliglucerase alfa in people with Gaucher Disease, run by Pfizer at 11 sites in 8 countries.
Trial summary
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system. 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
- 45 participants
- Where
- 11 sitesin 8 countries
- Study dates
- Jun 2008to Aug 2013
- 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
- Successful completion of Protocol PB-06-001
- The patient signs informed consent
Who cannot join
- Currently taking another experimental drug for any condition
- Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
- Pregnant or nursing
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study
Trial locations and contacts
11 sites in 8 countries.
11 sites in 8 countries
United States2 sites
Department of Human Genetics, Emory University School of Medicine
Decatur, Georgia, 30033
Neurogenetics, NYU at Rivergate
New York, New York, 10016
Israel2 sites
Rambam Medical Center
Haifa, 31096
Shaare Zedek Medical Center
Jerusalem
United Kingdom2 sites
Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
Cambridge
Royal Free Hospital
London, NW3 2QG
Australia1 site
Bone Marrow Transplant Service, The Royal Melbourne Hospital
Parkville, Victoria
About this study
Official title: A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.
This is an extension trial to Study NCT00376168 and NCT00712348.
Read the detailed description
This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.
- Conditions
- Gaucher Disease
- Being tested
- Taliglucerase alfa
- Keywords
- Gaucher Disease, 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
Also called Plant Cell Expressed Recombinant Human Glucocerebrosidase, prGCD
Intravenous infusion every 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| Naive 30 Units/kg | Experimental | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) Receives: Drug: Taliglucerase alfa |
| Naive 60 Units/kg | Experimental | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) Receives: Drug: Taliglucerase alfa |
| Switchover | Experimental | Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348) 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: Spleen Volume at Baseline and Months 12, 24, and 36
Secondary outcomes (3)
Liver Volume
Liver volume measured by MRI
Measured: Liver volume at Baseline and Months 12, 24 and 36
Hemoglobin
Measured: Hemoglobin at Baseline and Months 12, 24 and 36
Platelet Count
Measured: Platelet count at Baseline and Months 12, 24 and 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2008 (estimated)
- Study startJun 2008
- Primary completionMay 2012
- Study completionAug 2013
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 (2)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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00705939
- Sponsor's protocol ID
- PB-06-003
- Status last verified
- Sep 2018
- First submitted
- 25 Jun 2008
- Last updated
- 4 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Expanded access
- Available
Publications
Zimran A, Duran G, Mehta A, Giraldo P, Rosenbaum H, Giona F, Amato DJ, Petakov M, Munoz ET, Solorio-Meza SE, Cooper PA, Varughese S, Chertkoff R, Brill-Almon E. Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naive patients with Gaucher disease. Am J Hematol. 2016 Jul;91(7):656-60. doi: 10.1002/ajh.24369. Epub 2016 Apr 24.
PubMed 27174694Pastores GM, Shankar SP, Petakov M, Giraldo P, Rosenbaum H, Amato DJ, Szer J, Chertkoff R, Brill-Almon E, Zimran A. Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 18.
PubMed 27102949
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
- ClinicalTrials.gov ID
- NCT00705939
- Acronym / sponsor ID
- PB-06-003
- Conditions
- Gaucher Disease
- Keywords
- Gaucher Disease, Enzyme replacement therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- Jun 2008
- Primary completion
- May 2012 (actual)
- Study completion
- Aug 2013 (actual)
- First posted
- 27 Jun 2008 (estimated)
- Results
- Posted 15 Jul 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.
This is an extension trial to Study NCT00376168 and NCT00712348.
Detailed description
This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Naive 30 Units/kg | Experimental | Drug: Taliglucerase alfaContinue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
| Naive 60 Units/kg | Experimental | Drug: Taliglucerase alfaContinue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
| Switchover | Experimental | Drug: Taliglucerase alfaContinue taliglucerase alfa treatment from PB-06-002 (NCT00712348) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Successful completion of Protocol PB-06-001
- The patient signs informed consent
Key exclusion criteria (4 listed)
- Currently taking another experimental drug for any condition
- Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
- Pregnant or nursing
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient'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 alfaAlso: Plant Cell Expressed Recombinant Human Glucocerebrosidase, prGCDIntravenous infusion every 2 weeks | Drug | Naive 30 Units/kg, Naive 60 Units/kg, Switchover |
Primary outcome
Spleen Volume
Time frame: Spleen Volume at Baseline and Months 12, 24, and 36
Spleen volume measured by MRI
3 secondary outcomes
Liver Volume
Time frame: Liver volume at Baseline and Months 12, 24 and 36
Hemoglobin
Time frame: Hemoglobin at Baseline and Months 12, 24 and 36
Platelet Count
Time frame: Platelet count at Baseline and Months 12, 24 and 36
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 27174694Zimran A, Duran G, Mehta A, Giraldo P, Rosenbaum H, Giona F, Amato DJ, Petakov M, Munoz ET, Solorio-Meza SE, Cooper PA, Varughese S, Chertkoff R, Brill-Almon E. Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naive patients with Gaucher disease. Am J Hematol. 2016 Jul;91(7):656-60. doi: 10.1002/ajh.24369. Epub 2016 Apr 24.
- Derived from this study: PubMed 27102949Pastores GM, Shankar SP, Petakov M, Giraldo P, Rosenbaum H, Amato DJ, Szer J, Chertkoff R, Brill-Almon E, Zimran A. Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 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.
11 sites in 8 countries: see every site and its status.
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 at 11 sites in 8 countries.
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 4 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 4 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Israel, United Kingdom, Australia, Canada and 3 more countries. 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 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 15 Jul 2014). 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: Spleen Volume at Baseline and Months 12, 24, and 36). 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 NCT00705939, which also lists 2 linked publications.
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 4 Oct 2018.