Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1 Clinical Trial

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

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT00954460
• Other study ID #: HGT-GCB-058
• Status: Approved for marketing

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type 1

Intervention

Drug:
• velaglucerase alfa
up to 60 U/kg, every other week via intravenous infusion

Locations

Country	Name	City	State
United States	Akron Children's Hospital	Akron	Ohio
United States	Annapolis Oncology Center	Annapolis	Maryland
United States	Fullerton Genetic	Asheville	North Carolina
United States	Sinai Hospital of Baltimore	Baltimore	Maryland
United States	Tower Hematology Oncology	Beverly Hills	California
United States	Hemophilia Center of Western New York Incorporated	Buffalo	New York
United States	University of Virginia Health Systems	Charlottesville	Virginia
United States	Children's Memorial Hospital	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	University Research Foundation for Lysosomal Storage Diseases	Coral Springs	Florida
United States	Emory Genetics	Decatur	Georgia
United States	Rocky Mountain Cancer Centers	Denver	Colorado
United States	Duke Medical Center	Durham	North Carolina
United States	Gainesville Hematology Oncology Associates	Gainesville	Florida
United States	University of Iowa Hospitals and Clinics	Iowa City	Iowa
United States	The University Research Foundation for Lysosomal Storage Diseases	Kansas City	Missouri
United States	Rady's Children's Hospital of San Diego	La Jolla	California
United States	Southern California Permanente Medical Group	Los Angeles	California
United States	North Shore Hematology/Oncology - Manhasset	Manhasset	New York
United States	Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	Yale University	New Haven	Connecticut
United States	Mount Sinai School of Medicine	New York	New York
United States	New York University School of Medicine	New York	New York
United States	Adventis Healthcare System dba Florida Hospital	Orlando	Florida
United States	East Lake Oncology	Palm Harbor	Florida
United States	St. Joseph's	Paterson	New Jersey
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	St Joseph's Hospital & Medical Center	Phoenix	Arizona
United States	The Permanente Medical Group	Sacramento	California
United States	University of Massachusetts	Shrewsbury	Massachusetts
United States	O & O Alpan, LLC	Springfield	Virginia
United States	Stanford University Medical Genetics	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

United States,