A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

Completed · Phase 3

Conditions studied: Gaucher Disease

In brief

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.

Key facts

Study ID

NCT00376168

Run by

Pfizer

People needed

32

Starts

2007-08-01

Expected to finish

2009-10-01

Last updated by the study team

2018-10-04

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University Research Foundation for Lysosomal Storage Diseases — Coral Springs, Florida, United States
• Division of Medical Genetics, Emory University School of Medicine — Decatur, Georgia, United States
• New York University Medical Center — New York, New York, United States
• Mount Sinai Hospital — Toronto, Ontario, Canada
• Pontificia Universidad Catolica de Chile — Santiago, Chile
• Rambam Medical Center — Haifa, Israel
• Shaare Zedek Medical Center — Jerusalem, Israel
• Universita "La Sapienza" — Rome, Italy
• Morningside Medi-Clinic — Morningside, South Africa
• Hospital Universitario Miguel Servet — Zaragoza, Spain
• Royal Free Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.