Gaucher DiseasePhase 4NCT01132690
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
A Phase 4 study testing Taliglucerase alfa in people with Gaucher Disease, run by Pfizer at 3 sites in 3 countries.
Trial summary
This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- 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
- 11 participants
- Where
- 3 sitesin 3 countries
- Study dates
- Aug 2010to Jul 2012
- 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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Males and females 2 to <18 years old.
- Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
- Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
- Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
- Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).
Who cannot join
- Currently taking another investigational drug for any condition.
- Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
- Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
- Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
- History of allergy to carrots.
- Presence of HIV, HBsAg or hepatitis C infections.
- Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
- 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.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
Paraguay1 site
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Barrio Sajonia Asunción
About this study
Official title: A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.
- Conditions
- Gaucher Disease
- Being tested
- Taliglucerase alfa
- Keywords
- gaucher disease, pediatric
- 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 prGCD, plant cell expressed glucocerebrosidase
Taliglucerase alfa for infusion every two weeks for 12 months
| Study group | Role | What they receive |
|---|---|---|
| 30 units/kg | Experimental | Not described Receives: Drug: Taliglucerase alfa |
| 60 units/kg | Experimental | Not described Receives: Drug: Taliglucerase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemoglobin
median and interquartile range for change from baseline in haemoglobin
Measured: Every 3 months for 12 months
Secondary outcomes (5)
Chitotriosidase
Percent change from baseline in chitotriosidase
Measured: Every 3 months for 12 months
Spleen Volume
Spleen volume measured by MRI
Measured: Baseline and Month 12
Platelet Count
Mean and standard deviation of platelet count per cubic mm
Measured: Baseline and 12 months
Chemokine (C-C Motif) Ligand 18 (CCL18)
Percent change from baseline in CCL18
Measured: Every 3 months for 12 months
Liver Volume
Liver volume measured by MRI
Measured: Baseline and Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2010 (estimated)
- Study startAug 2010
- Primary completionMay 2012
- Study completionJul 2012
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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01132690
- Sponsor's protocol ID
- PB-06-005
- Status last verified
- Sep 2018
- First submitted
- 26 May 2010
- Last updated
- 5 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Expanded access
- Available
Publications
Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Elstein D, Paz A, Brill-Almon E, Chertkoff R. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease. Blood Cells Mol Dis. 2015 Jan;54(1):9-16. doi: 10.1016/j.bcmd.2014.10.002. Epub 2014 Nov 7.
PubMed 25453586
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
- ClinicalTrials.gov ID
- NCT01132690
- Acronym / sponsor ID
- PB-06-005
- Conditions
- Gaucher Disease
- Keywords
- gaucher disease, pediatric
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- Aug 2010
- Primary completion
- May 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 28 May 2010 (estimated)
- Results
- Posted 11 Nov 2014
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, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 30 units/kg | Experimental | Drug: Taliglucerase alfa |
| 60 units/kg | Experimental | Drug: Taliglucerase alfa |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Males and females 2 to <18 years old.
- Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
- Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
- Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
- Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).
Key exclusion criteria (8 listed)
- Currently taking another investigational drug for any condition.
- Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
- Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
- Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
- History of allergy to carrots.
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: prGCD, plant cell expressed glucocerebrosidaseTaliglucerase alfa for infusion every two weeks for 12 months | Drug | 30 units/kg, 60 units/kg |
Primary outcome
Hemoglobin
Time frame: Every 3 months for 12 months
median and interquartile range for change from baseline in haemoglobin
5 secondary outcomes
Chitotriosidase
Time frame: Every 3 months for 12 months
Spleen Volume
Time frame: Baseline and Month 12
Platelet Count
Time frame: Baseline and 12 months
Chemokine (C-C Motif) Ligand 18 (CCL18)
Time frame: Every 3 months for 12 months
Liver Volume
Time frame: Baseline and Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25453586Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Elstein D, Paz A, Brill-Almon E, Chertkoff R. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease. Blood Cells Mol Dis. 2015 Jan;54(1):9-16. doi: 10.1016/j.bcmd.2014.10.002. Epub 2014 Nov 7.
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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.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Taliglucerase alfa in people with Gaucher Disease, run by Pfizer at 3 sites in 3 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 5 inclusion and 8 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 5 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 3 sites in Israel, Paraguay, South Africa. 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 11 Nov 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?
Hemoglobin (time frame: Every 3 months for 12 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 NCT01132690, 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 5 Oct 2018.