NCT01411228

Brief 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 taliglucerase alfa).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Sep 2011

Typical duration for phase_3

Geographic Reach
3 countries

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 4, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 8, 2011

Completed
24 days until next milestone

Study Start

First participant enrolled

September 1, 2011

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2014

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2014

Completed
1.5 years until next milestone

Results Posted

Study results publicly available

January 18, 2016

Completed
Last Updated

September 7, 2018

Status Verified

September 1, 2018

Enrollment Period

2.8 years

First QC Date

August 4, 2011

Results QC Date

November 6, 2015

Last Update Submit

September 5, 2018

Conditions

Gaucher Disease

Keywords

Gaucher diseasepediatrics

Outcome Measures

Primary Outcomes (1)

  • 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.

    Baseline, months 9, 12 and 24

Secondary Outcomes (4)

  • Chitotriosidase

    Baseline, months 9, 12 and 24

  • Spleen Volume

    Baseline, months 12 and 24

  • Platelet Count

    Baseline, months 9, 12, 24 and 33-36

  • Liver Volume

    Baseline, months 12 and 24

Study Arms (2)

60 Units/kg

EXPERIMENTAL
Drug: Taliglucerase alfa

30 Units/kg

EXPERIMENTAL
Drug: Taliglucerase alfa

Interventions

Taliglucerase alfaDRUG

Taliglucerase alfa for infusion every two weeks for 24 months

Also known as: prGCD, plant cell expressed glucocerebrosidase
30 Units/kg60 Units/kg

Eligibility Criteria

Age2 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Shaare Zedek Medical Center

Jerusalem, Israel

Location

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

Barrio Asunción, Paraguay

Location

Morningside Medi-Clinic

Morningside, South Africa

Location

Related Publications (2)

  • 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.

    PMID: 27839981DERIVED

  • 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.

    PMID: 26053270DERIVED

MeSH Terms

Conditions

Gaucher Disease

Interventions

taliglucerase alfa

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Results Point of Contact

Title
Glen Park
Organization
Target Health Inc.
gpark@targethealth.com
212-681-2100

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

August 4, 2011

First Posted

August 8, 2011

Study Start

September 1, 2011

Primary Completion

July 1, 2014

Study Completion

August 1, 2014

Last Updated

September 7, 2018

Results First Posted

January 18, 2016

Record last verified: 2018-09

Locations

PA(1)ZA(1)IL(1)