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.
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
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.
3 sites in 3 countries
Paraguay1 site
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Barrio Asunción
About this study
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 taliglucerase alfa).
- 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 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.
- 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)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
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 27839981Abbas 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