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

May 18, 2021 updated by: Shire

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

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.

Study Overview

Status

Approved for marketing

Intervention / Treatment

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.

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Arizona
      • Phoenix, Arizona, United States, 85013
        • St Joseph's Hospital & Medical Center
    • California
      • Beverly Hills, California, United States, 90211-1850
        • Tower Hematology Oncology
      • La Jolla, California, United States, 92093
        • Rady's Children's Hospital of San Diego
      • Los Angeles, California, United States, 90027
        • Southern California Permanente Medical Group
      • Sacramento, California, United States, 95815
        • The Permanente Medical Group
      • Stanford, California, United States, 94305-5208
        • Stanford University Medical Genetics
    • Colorado
      • Denver, Colorado, United States, 80218
        • Rocky Mountain Cancer Centers
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale University
    • Florida
      • Coral Springs, Florida, United States, 33065
        • University Research Foundation for Lysosomal Storage Diseases
      • Gainesville, Florida, United States, 32605-4218
        • Gainesville Hematology Oncology Associates
      • Orlando, Florida, United States, 32804-4603
        • Adventis Healthcare System dba Florida Hospital
      • Palm Harbor, Florida, United States, 34685
        • East Lake Oncology
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Emory Genetics
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Children's Memorial Hospital
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Maryland
      • Annapolis, Maryland, United States, 21401
        • Annapolis Oncology Center
      • Baltimore, Maryland, United States, 21215
        • Sinai Hospital of Baltimore
    • Massachusetts
      • Shrewsbury, Massachusetts, United States, 01545
        • University of Massachusetts
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Hospitals and Clinics of Minnesota
    • Missouri
      • Kansas City, Missouri, United States, 64108-4619
        • The University Research Foundation for Lysosomal Storage Diseases
    • New Jersey
      • Paterson, New Jersey, United States, 07503
        • St. Joseph's
    • New York
      • Buffalo, New York, United States, 14215
        • Hemophilia Center of Western New York Incorporated
      • Manhasset, New York, United States, 11030
        • North Shore Hematology/Oncology - Manhasset
      • New York, New York, United States, 10016
        • New York University School of Medicine
      • New York, New York, United States, 10029-6500
        • Mount Sinai School of Medicine
    • North Carolina
      • Asheville, North Carolina, United States, 28801-4420
        • Fullerton Genetic
      • Durham, North Carolina, United States, 27710
        • Duke Medical Center
    • Ohio
      • Akron, Ohio, United States, 44308
        • Akron Children's Hospital
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Virginia
      • Charlottesville, Virginia, United States, 22908-0386
        • University of Virginia Health Systems
      • Springfield, Virginia, United States, 22152
        • O & O Alpan, LLC

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

August 5, 2009

First Submitted That Met QC Criteria

August 6, 2009

First Posted (Estimate)

August 7, 2009

Study Record Updates

Last Update Posted (Actual)

May 21, 2021

Last Update Submitted That Met QC Criteria

May 18, 2021

Last Verified

May 1, 2021

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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