NCT00430625

Brief Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to this deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the efficacy of every other week dosing of Gene-Activated® Human Glucocerebrosidase (GA-GCB, velaglucerase alfa) at doses of 45 and 60 U/kg in treatment-naïve patients with type 1 Gaucher disease.

Trial Health

93
On Track

Trial Health Score

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

Enrollment
25

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Feb 2007

Geographic Reach
5 countries

5 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

February 1, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 2, 2007

Completed
13 days until next milestone

Study Start

First participant enrolled

February 15, 2007

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2009

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

September 10, 2010

Completed
Last Updated

June 29, 2021

Status Verified

June 1, 2021

Enrollment Period

2.1 years

First QC Date

February 1, 2007

Results QC Date

June 3, 2010

Last Update Submit

June 28, 2021

Conditions

Gaucher Disease, Type 1

Keywords

VPRIV®Enzyme Replacement TherapyGaucher diseaseglucocerebrosidasebeta-glucocerebrosidaseAcid beta-glucocerebrosidaseglucosylceramidaseD-glucosyl-N-acylsphingosine glucohydrolasegene activationhuman

Outcome Measures

Primary Outcomes (1)

  • Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group.

    Efficacy endpoint

    Week 53

Secondary Outcomes (6)

  • Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group

    Week 53

  • Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group.

    Week 53

  • Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)

    Week 51

  • Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI))

    Week 51

  • Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group

    Week 53

  • +1 more secondary outcomes

Study Arms (2)

VPRIV®-45 U/kg, IV, every other week

EXPERIMENTAL

VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB)

Biological: VPRIV ®,

VPRIV®-60 U/kg, IV, every other week

EXPERIMENTAL

VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase,GA-GCB)

Biological: VPRIV ®,

Interventions

VPRIV ®,BIOLOGICAL

Intravenous (IV) infusion, every other week via intravenous infusion for 12 months

Also known as: VPRIV®, velaglucerase alfa, GA-GCB, Gene-activated® human glucocerebrosidase
VPRIV®-45 U/kg, IV, every other weekVPRIV®-60 U/kg, IV, every other week

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patient has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and is willing and able to provide written informed consent prior to initiating any study-related procedures
  • Patient is at least 2 years of age
  • Patient has Gaucher disease-related anemia and
  • Patient has at least moderate splenomegaly or
  • Patient has Gaucher disease-related thrombocytopenia or
  • Patient has a readily palpable enlarged liver
  • Patient has not received treatment for Gaucher disease within 30 months prior to study entry
  • Female patients of child-bearing potential agree to use a medically acceptable method of contraception. Male patients must agree to use a medically acceptable method of birth control.
  • Patient must be sufficiently cooperative to participate in the study as judged by the Investigator.

You may not qualify if:

  • Includes:
  • Patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease
  • Patient is antibody-positive to imiglucerase during screening or has experienced an anaphylactic reaction to imiglucerase
  • Patient has received treatment with any investigational drug or device within the 30 days prior to study entry
  • Patient is Human immunodeficiency virus (HIV) positive
  • Patient is hepatitis positive
  • Patient presents with iron, folic acid and/or vitamin B12 deficiency sustained anemia during screening
  • Patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study
  • Patient has a significant comorbidity(ies)that might affect study data or confound the study results
  • Patient is a pregnant and/or lactating female
  • Patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Hipolito Yrigoyen

Buenos Aires, Argentina

Location

Shaare Zedek Medical Center

Jerusalem, Israel

Location

Sociedad Espanola de Socorros Mutuos

Asunción, Paraguay

Location

National Research Center for Haematology

Moscow, Russia

Location

La Rabta Hospital

Tunis, Tunisia

Location

Related Publications (1)

  • Zimran A, Elstein D, Gonzalez DE, Lukina EA, Qin Y, Dinh Q, Turkia HB. Treatment-naive Gaucher disease patients achieve therapeutic goals and normalization with velaglucerase alfa by 4years in phase 3 trials. Blood Cells Mol Dis. 2018 Feb;68:153-159. doi: 10.1016/j.bcmd.2016.10.007. Epub 2016 Oct 21.

    PMID: 27839979DERIVED

Related Links

MeSH Terms

Conditions

Gaucher Disease

Interventions

Glucosylceramidase

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

Intervention Hierarchy (Ancestors)

GlucosidasesGlycoside HydrolasesHydrolasesEnzymesEnzymes and Coenzymes

Limitations and Caveats

Due to small sample sizes no conclusions could be drawn on Quality of life (QOL).

Results Point of Contact

Title
Study Director
Organization
Shire
ClinicalTransparency@shire.com
+1 866 842 5335

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 1, 2007

First Posted

February 2, 2007

Study Start

February 15, 2007

Primary Completion

April 1, 2009

Study Completion

April 1, 2009

Last Updated

June 29, 2021

Results First Posted

September 10, 2010

Record last verified: 2021-06

Locations

IL(1)TU(1)AR(1)PA(1)RU(1)