Phase 3
N=32
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher Disease
Bottom Line
View on ClinicalTrials.gov: NCT00376168 ↗Enrolled (actual)
32
Serious AEs
0.0%
Results posted
Jul 2012
Primary outcome: Primary: Change From Baseline in Spleen Volume Measured by MRI. — -26.91; -38.01 percentage of change — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Plant cell expressed recombinant glucocerebrosidase (prGCD) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Sep 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Spleen Volume Measured by MRI. |
-26.91; -38.01 | <0.0001 sig |
| SECONDARY Change From Baseline in Liver Volume |
-10.48; -11.11 | 0.0041 sig |
| SECONDARY Change in Hemoglobin |
1.6; 2.2 | 0.0010 sig |
| SECONDARY Change in Platelet Count |
11,427; 41,494 | 0.0460 sig |
Summary
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.
This is the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.
Eligibility Criteria
Inclusion Criteria
- Males and females, 18 years or older
- Confirmed enzymatic diagnosis of Gaucher disease
- Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis
- Female patients of child-bearing potential who agree to use a medically acceptable method of contraception
- Thrombocytopenia (defined as platelet counts below the lower limit of normal) and/or anemia (defined by hemoglobin level at least 1 g/dL below normal range according to sex and age).
- Patients who have not received ERT in the past or patients whoc have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody test.
- Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
- Ability to provide a written informed consent.
Exclusion Criteria
- Currently taking another experimental drug for any condition
- Pregnant or nursing
- Presence of HIV and/or, HBsAg and/or hepatitis C infections
- Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.
- Previous anaphylactoid reaction to Cerezyme® or Ceredase®.
- History of allergy to carrots.
Data sourced from ClinicalTrials.gov (NCT00376168). 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.