Gaucher DiseasePhase 3NCT01411228

A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

A Phase 3 study testing Taliglucerase alfa in people with Gaucher Disease, run by Pfizer at 3 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with… Read more about this study

Who is it for?: Ages 2 to 18 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: 15 participants
Where: 3 sitesin 3 countries
Study dates: Sep 2011to Aug 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 2 to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Successful completion of Protocol PB-06-002 or PB-06-005
The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent

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 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.

Country

City, postcode or hospital

3 sites in 3 countries

Israel1 site

Shaare Zedek Medical Center
Jerusalem

Other trials in Israel

Paraguay1 site

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Barrio Asunción

Other trials in Paraguay

South Africa1 site

Morningside Medi-Clinic
Morningside

Other trials in South Africa

About this study

Conditions: Gaucher Disease
Being tested: Taliglucerase alfa
Keywords: Gaucher disease, pediatrics
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 24 months

Study groups in NCT01411228
Study group	Role	What they receive
60 Units/kg	Experimental	Not described Receives: Drug: Taliglucerase alfa
30 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. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Measured: Baseline, months 9, 12 and 24

Secondary outcomes (4)

Chitotriosidase
Chitotriosidase. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Measured: Baseline, months 9, 12 and 24
Spleen Volume
Spleen volume measured by MRI (or ultrasound). Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Measured: Baseline, months 12 and 24
Platelet Count
Platelet count. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Measured: Baseline, months 9, 12, 24 and 33-36
Liver Volume
Liver volume by MRI or Ultrasound. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Measured: Baseline, months 12 and 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2011Aug 2014

First posted8 Aug 2011 (estimated)
Study startSep 2011
Primary completionJul 2014
Study completionAug 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 (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: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 15 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01411228
Sponsor's protocol ID: PB-06-006
Status last verified: Sep 2018
First submitted: 4 Aug 2011
Last updated: 7 Sep 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Expanded access: Available

Publications

Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Cooper PA, Varughese S, Giraldo P, Petakov M, Tan ES, Chertkoff R. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naive or previously treated with imiglucerase. Blood Cells Mol Dis. 2018 Feb;68:163-172. doi: 10.1016/j.bcmd.2016.10.005. Epub 2016 Oct 20.
PubMed 27839981
Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.
PubMed 26053270

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Gaucher Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 7 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
ClinicalTrials.gov ID: NCT01411228
Acronym / sponsor ID: PB-06-006
Conditions: Gaucher Disease
Keywords: Gaucher disease, pediatrics
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 15 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Sep 2011
Primary completion: Jul 2014 (actual)
Study completion: Aug 2014 (actual)
First posted: 8 Aug 2011 (estimated)
Results: Posted 18 Jan 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2

Arm	Type	Receives
60 Units/kg	Experimental	Drug: Taliglucerase alfa
30 Units/kg	Experimental	Drug: Taliglucerase alfa

Who may qualify

Ages: 2 to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (2 listed)

Successful completion of Protocol PB-06-002 or PB-06-005
The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent

Key exclusion criteria (3 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 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 alfaAlso: prGCD, plant cell expressed glucocerebrosidaseTaliglucerase alfa for infusion every two weeks for 24 months	Drug	30 Units/kg, 60 Units/kg

Primary outcome

Hemoglobin
Time frame: Baseline, months 9, 12 and 24
Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.

4 secondary outcomes

Chitotriosidase
Time frame: Baseline, months 9, 12 and 24
Spleen Volume
Time frame: Baseline, months 12 and 24
Platelet Count
Time frame: Baseline, months 9, 12, 24 and 33-36
Liver Volume
Time frame: Baseline, months 12 and 24

Results and safety

Results posted to the registry on 18 Jan 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
60 Units/kg	5	4
30 Units/kg	5	5
Switchover	5	4

Primary outcome: Hemoglobin

Time frame: Baseline, months 9, 12 and 24. Median (Inter-Quartile Range). Unit: g/dL

Measure	60 Units/kg n = 5	30 Units/kg n = 5	Switchover n = 5
Baseline	10.6 (9.1 to 12.3)	11.2 (9.1 to 12.3)	13.5 (12.9 to 14.2)
Month 9	11.9 (10.5 to 13.8)	12.5 (10.1 to 13.7)	13.9 (12.5 to 15.2)
Month 12	12.2 (11.2 to 13.7)	12.8 (11.2 to 14.2)	13.7 (12.0 to 15.1)
Month 24	12.8 (11.0 to 14.2)	13.1 (11.5 to 14.5)	14.2 (12.1 to 16.0)

Population analysed: Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program

4 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
60 Units/kg	1 of 5 (20%)	5 of 5 (100%)	Not reported
30 Units/kg	0 of 5 (0%)	4 of 5 (80%)	Not reported
Switchover	0 of 5 (0%)	5 of 5 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Sep 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 27839981Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Cooper PA, Varughese S, Giraldo P, Petakov M, Tan ES, Chertkoff R. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naive or previously treated with imiglucerase. Blood Cells Mol Dis. 2018 Feb;68:163-172. doi: 10.1016/j.bcmd.2016.10.005. Epub 2016 Oct 20.
Derived from this study: PubMed 26053270Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.

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
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Enrolling by invitationObservational study
Genetic Studies of Lysosomal Storage Disorders
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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 3 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 2 inclusion and 3 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 7 Sep 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 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 18 Jan 2016). 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: Baseline, months 9, 12 and 24). 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 NCT01411228, 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 7 Sep 2018.