An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

May 30, 2021 updated by: Shire

An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease

The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Type 1 Gaucher disease, the most common form,accounts for more than 90% of all cases 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. Gene-Activated® human glucocerebrosidase (GA-GCB,velaglucerase alfa) 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. GA-GCB contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the long-term safety of GA-GCB in men, women, and children with Type 1 Gaucher disease.

Study Type

Interventional

Enrollment (Actual)

95

Phase

  • Phase 3

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

  • Argentina
      • Buenos Aires, Argentina
        • Your Health S.A.
  • India
      • New Delhi, India, 110 029
        • All India Institute of Medical Sciences
      • Pune, India, 411 011
        • KEM hospital
  • Israel
      • Jerusalem, Israel, 91031
        • Shaare Zedek Medical Center
  • Korea, Republic of
    • Gyeongsangnam-do
      • Jinju, Gyeongsangnam-do, Korea, Republic of, 660-702
        • Gyeongsang National University Hospital
  • Paraguay
      • Asuncion, Paraguay
        • Sociedad Espanola de Socorros Mutuos
  • Poland
      • Warszawa, Poland
        • Instytut "Pomnik-Centrum Zdrowia Dziecka"
  • Russian Federation
      • Moscow, Russian Federation, 125167
        • State Institution "Hematology Research Centre RAMS"
  • Spain
      • Zaragoza, Spain, 50008
        • Hospital Universitario Miguel Servet
  • Tunisia
    • Jebbari
      • Tunis, Jebbari, Tunisia, 1007 BS
        • Hospital de La Rabta
  • United Kingdom
      • London, United Kingdom, NM3 2QG
        • Royal Free Hospital
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Los Angeles Medical Center
      • Oakland, California, United States, 94609
        • Children's Hospital Oakland
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University School of Medicine
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Children's Memorial Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Hospitals and Clinics of Minnesota
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • CHILDREN'S MERCY HOSPITALS & CLINICS
    • New York
      • New York, New York, United States, 10016
        • NYU Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Medical Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Children's Hospital of Wisconsin

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

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. The patient has completed study TKT032 or TKT034, or study HGT-GCB-039.
  2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study.
  3. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the investigator.
  4. The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
  5. The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator

Exclusion Criteria:

  1. The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted.
  2. The patient is pregnant or lactating.
  3. The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.
  4. The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.).
  5. The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator

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?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)

This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes patients from the following groups:

VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
Biological: VPRIV®
Intravenous infusion, every other week (EOW)
Other Names:
  • velaglucerase alfa
  • Gene-Activated® Human Glucocerebrosidase(GA-GCB)
Experimental: VPRIV 60 U/kg (Parent study-imiglucerase(60 U/kg) HGT-GCB-039)
imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched 60 U/kg VPRIV in HGT-GCB-044
Biological: VPRIV®
Intravenous infusion, every other week (EOW)
Other Names:
  • velaglucerase alfa
  • Gene-Activated® Human Glucocerebrosidase(GA-GCB)
Experimental: VPRIV 15-60 U/kg (Parent study VPRIV (15-60 U/kg) TKT034)
VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647) and continued in HGT-GCB-044 at the same dose as prescribed in TKT034
Biological: VPRIV®
Intravenous infusion, every other week (EOW)
Other Names:
  • velaglucerase alfa
  • Gene-Activated® Human Glucocerebrosidase(GA-GCB)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Summary of Treatment Emergent Adverse Events
Time Frame: Baseline to termination of study
Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.
Baseline to termination of study

Secondary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group
Time Frame: Baseline to 24 months
Baseline to 24 months
Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group
Time Frame: Baseline to 24 months
Baseline to 24 months
Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group
Time Frame: Baseline to 24 months
Baseline to 24 months
Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group
Time Frame: Baseline to 24 months
Baseline to 24 months

Collaborators and Investigators

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

Sponsor

Shire

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.

General Publications

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 Major Dates

Study Start (Actual)

March 13, 2008

Primary Completion (Actual)

December 28, 2012

Study Completion (Actual)

December 28, 2012

Study Registration Dates

First Submitted

March 6, 2008

First Submitted That Met QC Criteria

March 12, 2008

First Posted (Estimate)

March 13, 2008

Study Record Updates

Last Update Posted (Actual)

June 10, 2021

Last Update Submitted That Met QC Criteria

May 30, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HGT-GCB-044
  • 2008-001965-27 (EudraCT Number)

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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