Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Information source: Shire Human Genetic Therapies, Inc.
Information obtained from ClinicalTrials.gov on October 19, 2009
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease, Type 1
Intervention: velaglucerase alfa (Drug)
Phase: N/A
Status: Available
Sponsored by: Shire Human Genetic Therapies, Inc. Official(s) and/or principal investigator(s):
Gabriel M. Cohn, M.D., Study Director, Affiliation: Shire Human Genetic Therapies, Inc.
Overall contact:
Dan Madden (One Path), Phone: 1-866-888-0660
Summary
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside
accumulates within macrophages leading to cellular engorgement, organomegaly, and organ
system dysfunction. The purpose of this treatment protocol is to observe the safety of
velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive
(newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy
(ERT) imiglucerase.
Clinical Details
Official title: Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease
Study design: N/A
Detailed description:
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases of
Gaucher disease and does not involve the CNS. Typical manifestations of type 1 Gaucher
disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia,
hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased
quality of life. Velaglucerase alfa (Gene-Activated™ human glucocerebrosidase;GA-GCB) is
produced in a continuous human cell line using proprietary gene-activation technology and
has an identical amino acid sequence to the naturally occurring human enzyme. Velaglucerase
alfa contains terminal mannose residues that target the enzyme to the macrophages-the
primary target cells in Gaucher disease. This treatment protocol will observe the safety of
velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive
(newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy
(ERT) imiglucerase. Patients currently being treated with ERT for their Gaucher disease
will receive the same number of units of velaglucerase alfa per month as their imiglucerase
dose for doses between 30-120 U/kg/month. For patients who experienced dose reductions in
their imiglucerase treatment due to supply constraints the pre-reduction monthly dose may be
used to determine the monthly dose of velaglucerase alfa.
Eligibility
Minimum age: 3 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
1. The patient has a documented diagnosis of type 1 Gaucher disease
2. The patient is > 2 years of age
3. The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction
to another ERT including imiglucerase
4. Women of child-bearing potential must agree to use a medically acceptable method of
contraception at all times during the study; and must have a negative result to a
pregnancy test as required throughout their participation in the study. Male
patients must use a medically acceptable method of birth control throughout their
participation in the study and must report their partner's pregnancy.
5. The patient is sufficiently cooperative to participate in this treatment plan as
judged by the Investigator
6. If the patient is naïve or new to treatment, the patient has one or more of the
following (in absence of the following criteria, please call the sponsor for
treatment justification):
- Gaucher disease-related anemia
- Moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation
- Gaucher disease-related thrombocytopenia
- Gaucher disease-related palpable enlarged liver
Exclusion Criteria: None
Locations and Contacts
Dan Madden (One Path), Phone: 1-866-888-0660
Tower Hematology Oncology, Beverly Hills, California 90211, United States
Southern California Permanente Medical Group, Los Angeles, California 90027, United States
Yale University, New Haven, Connecticut 06510, United States
Gainesville Hematology Oncology Associates, Gainesville, Florida 32605-4218, United States
University Research Foundation for Lysosomal Storage Diseases, Coral Springs, Florida 33065, United States
Annapolis Oncology Center, Annapolis, Maryland 21401, United States
Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota 55404, United States
Children's Mercy Hospital, Kansas City, Missouri 64108, United States
New York University School of Medicine, New York, New York 10016, United States
North Shore Hematology/Oncology - Manhasset, Manhasset, New York 11030, United States
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio 45229, United States
Additional Information
Last updated: October 16, 2009
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