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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


N/A
3 Years
N/A
Not Enrolling
Both
Gaucher Disease, Type 1

Thank you

Trial Information

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


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.


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

Type of Study:

Expanded Access

Study Design:

N/A

Principal Investigator

Gabriel M. Cohn, M.D.

Investigator Role:

Study Director

Investigator Affiliation:

Shire Human Genetic Therapies, Inc.

Authority:

United States: Food and Drug Administration

Study ID:

HGT-GCB-058

NCT ID:

NCT00954460

Start Date:

Completion Date:

Related Keywords:

  • Gaucher Disease, Type 1
  • VPRIV
  • Enzyme Replacement Therapy
  • Gaucher disease
  • glucocerebrosidase
  • beta-glucocerebrosidase
  • Acid beta-glucocerebrosidase
  • glucosylceramidase
  • D-glucosyl-N-acylsphingosine glucohydrolase
  • gene activation
  • human
  • Gaucher Disease

Name

Location

Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
University of Iowa Hospitals and ClinicsIowa City, Iowa  52242
Mount Sinai School of MedicineNew York, New York  10029
Sinai Hospital of BaltimoreBaltimore, Maryland  21225
Southern California Permanente Medical GroupDowney, California  90242
Children's Hospitals and Clinics of MinnesotaSaint Paul, Minnesota  55102
Cincinnati Children's Hospital Medical CenterCincinnati, Ohio  45229-3039
Akron Children's HospitalAkron, Ohio  44308-1062
Rocky Mountain Cancer CentersThornton, Colorado  80260
Yale UniversityNew Haven, Connecticut  06520
New York University School of MedicineNew York, New York  10016
Children's Memorial HospitalChicago, Illinois  60614
Annapolis Oncology CenterAnnapolis, Maryland  21401
University of MassachusettsWorcester, Massachusetts  01655
Gainesville Hematology Oncology AssociatesGainesville, Florida  32605
Duke Medical CenterDurham, North Carolina  
St Joseph's Hospital & Medical CenterPhoenix, Arizona  85013
Tower Hematology OncologyBeverly Hills, California  90211-1850
Rady's Children's Hospital of San DiegoLa Jolla, California  92093
The Permanente Medical GroupSacramento, California  95815
Stanford University Medical GeneticsStanford, California  94305-5208
University Research Foundation for Lysosomal Storage DiseasesCoral Springs, Florida  33065
Adventis Healthcare System dba Florida HospitalOrlando, Florida  32804-4603
East Lake OncologyPalm Harbor, Florida  34685
Emory GeneticsDecatur, Georgia  30033
The University Research Foundation for Lysosomal Storage DiseasesKansas City, Missouri  64108-4619
St. Joseph'sPatterson, New Jersey  07503
Hemophilia Center of Western New York IncorporatedBuffalo, New York  14215
North Shore Hematology/Oncology - ManhassetManhasset, New York  11030
Fullerton GeneticAshville, North Carolina  28801-4420
University of Virginia Health SystemsCharlottesville, Virginia  22908-0386
O & O Alpan, LLCSpringfield, Virginia  22152