A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 July 2021
• Main ID: NCT00430625
• Date of registration: 01/02/2007
• Prospective Registration: Yes
• Primary sponsor: Shire
• Public title: A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
• Scientific title: A Multicenter, Randomized, Double-Blind, Parallel Group, Two-Dose Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease
• Date of first enrolment: February 15, 2007
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00430625
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 3

Countries of recruitment

Argentina, Israel, Paraguay, Russian Federation, Tunisia, United States

Contacts

• Name: Study Director
• Affiliation: Takeda

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

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



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease, Type 1

Intervention(s)

Biological: VPRIV ®,

Primary Outcome(s)

Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. [Time Frame: Week 53]

Secondary Outcome(s)

Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI) [Time Frame: Week 51]

Percent Change From Baseline to 12 Months in Chemokine (C-C Motif) Ligand 18 (CCL18) [Time Frame: Week 53]

Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group [Time Frame: Week 53]

Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group [Time Frame: Week 53]

Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)) [Time Frame: Week 51]

Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group. [Time Frame: Week 53]

Secondary ID(s)

TKT032

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 10/09/2010
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00430625