Phase 3 N=25 Randomized Double-blind Treatment

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

Gaucher Disease, Type 1

Bottom Line

View on ClinicalTrials.gov: NCT00430625 ↗

Enrolled (actual)

Serious AEs

4.0%

Results posted

Sep 2010

Primary outcome: Primary: Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. — 2.429 g/dL — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: VPRIV ®, (Biological)
Age: Pediatric, Adult, Older Adult · 2+ yrs
Sex: All
Sponsor: Shire
Primary completion: Apr 2009

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group.	2.429	<0.0001 sig
SECONDARY Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group	2.438	—
SECONDARY Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group.	40.92; 50.88	—
SECONDARY Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)	-0.30; -0.84	—
SECONDARY Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI))	-1.87; -1.92	—
SECONDARY Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group	-61.16; -69.65	—
SECONDARY Percent Change From Baseline to 12 Months in Chemokine (C-C Motif) Ligand 18 (CCL18)	-46.76; -66.02	—

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.

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00430625). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.